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COG ACNS1422 - A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients
Patients greater than or equal to 3 years of age and < 22 years of age with newly diagnosed type of brain tumor called WNT)/Wingless (WNT)-driven medulloblastoma using reduced craniospinal radiotherapy.
Christopher Moertel, MD
All
3 Years to 21 Years old
Phase 2
STUDY00002501
STUDY00002501
Inclusion Criteria:
Patients must be greater than or equal to 3 years and less than 22 years of age at the time of enrollment
Patients must be newly diagnosed and have:
Eligibility confirmed by rapid central pathology and molecular screening review on APEC14B1 and via the Molecular Characterization Initiative:
Classical histologic type (non LC/A) WNT medulloblastoma
Positive nuclear beta-catenin by immunohistochemistry (IHC)
Positive for CTNNB1 mutation
Negative for MYC and MYCN by fluorescence in situ hybridization (FISH)
Patient must have negative lumbar cerebrospinal fluid (CSF) cytology
Note: CSF cytology for staging should be performed no sooner than 14 days post operatively to avoid false positive CSF; ideally, CSF should be obtained between day 14 and day 21 to allow for final staging status before enrollment onto the study; patients with positive CSF cytology obtained 0 to 14 days after surgery should have cytology repeated to determine eligibility and final CSF status; patients with negative CSF cytology from lumbar puncture obtained 0 to 14 days after surgery do not need cytology repeated; patients with negative CSF cytology from lumbar puncture obtained prior to surgery do not need cytology repeated post-operatively
Patients must have eligibility confirmed by Rapid Central Imaging Review on APEC14B1; patients must have =< 1.5 cm^2 maximal cross-sectional area of residual tumor; whole brain magnetic resonance imaging (MRI) with and without gadolinium and spine MRI with gadolinium must be performed
Patients must be enrolled, and protocol therapy must be projected to begin, no later than 36 days after definitive diagnostic surgery (day 0)
Peripheral absolute neutrophil count (ANC) >= 1000/uL
Platelet count >= 100,000/uL (transfusion independent)
Hemoglobin >= 8.0 g/dL (may receive red blood cell [RBC] transfusions)
Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
3 to < 6 years of age: maximum (max) serum creatinine 0.8 mg/dL (males and females)
6 to < 10 years of age: max serum creatinine 1 mg/dL (males and females)
10 to < 13 years of age: max serum creatinine 1.2 mg/dL (males and females)
13 to < 16 years of age: max serum creatinine 1.5 md/dL (males) and 1.4 md/dL (females)
>= 16 years of age: max serum creatinine 1.7 mg/dL (males) and 1.4 mg/dL (females)
The threshold creatinine values were derived from the Schwartz formula for estimating GFR utilizing child length and stature data published by the Centers for Disease Control and Prevention (CDC)
Total or direct bilirubin =< 1.5 x upper limit of normal (ULN) for age, and
Serum glutamate pyruvate (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L (3x ULN); for the purpose of this study, the ULN for SGPT is 45 U/L
Central nervous system function defined as:
Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled
Patients must not be in status epilepticus, a coma or on assisted ventilation at the time of study enrollment
Patients must have receptive and expressive language skills in English, French, or Spanish to complete the QoL and neurocognitive assessments; if a patient meets these criteria but the parent/guardian speaks a language other than English, French, or Spanish, the patient may still be enrolled and tested, and the parent-report measures should be omitted
All patients and/or their parents or legal guardians must sign a written informed consent; assent, when appropriate, will be obtained according to institutional guidelines
All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Exclusion Criteria:
Patients with metastatic disease by either MRI evaluation (brain and spine) or lumbar CSF cytology are not eligible; patients who are unable to undergo a lumbar puncture for assessment of CSF cytology are ineligible
Patients must not have received any prior radiation therapy or chemotherapy (tumor-directed therapy) other than surgical intervention and/or corticosteroids
Pregnancy and Breast Feeding
Female patients who are pregnant are ineligible due to risks of fetal and teratogenic adverse events as seen in animal/human studies
Lactating females are not eligible unless they have agreed not to breastfeed their infants
Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained
Sexually active patients of reproductive potential are not eligible unless they have agreed to use an effective contraceptive method for the duration of their study participation
Patients with a history of moderate to profound intellectual disability (i.e., intelligence quotient [Q)]=< 55) are not eligible for enrollment; PLEASE NOTE: Children with a prior history of attention deficit hyperactivity disorder (ADHD) or a specific learning disability (e.g., dyslexia) are eligible for this study. Children with posterior fossa syndrome (also known as cerebellar mutism) are eligible for this study Cisplatin, Cyclophosphamide, Laboratory Biomarker Analysis, Lomustine, Magnetic Resonance Imaging, Radiation Therapy, Vincristine, Vincristine Sulfate, Cisplatin, Cyclophosphamide, Laboratory Biomarker Analysis, Lomustine, Magnetic Resonance Imaging, Radiation Therapy, Vincristine, Vincristine Sulfate
Medulloblastoma
Synergistic Enteral Regimen for Treatment of the Gangliosidoses (SYNER-G) (Syner-G)
The Syner-G regimen research study seeks to evaluate the use of a combination of a medication called miglustat and a ketogenic diet for treatment of the gangliosidoses to learn if this combination will provide improved clinical outcomes compared to what we currently know about the natural course of the disease.
Jeanine Jarnes
All
to 204 Months old
Phase 4
1311M46101
1311M46101
Inclusion Criteria:
Subjects must have a documented infantile or juvenile gangliosidosis disease.
Age: 17 years or less at time of enrollment
Subjects and their caregivers must be willing to work with a ketogenic diet team for management of the subject's ketogenic diet.
Exclusion Criteria:
A desire to not participate
Patients who are older than 17 years will not be enrolled in this study.
Children with severe renal impairment will not be enrolled in this study.
Post-pubertal females who are pregnant, or who are unwilling to use highly-effective methods to prevent pregnancy, will be excluded from this study.
Breast-feeding females will be excluded from this study.
Subjects who have an allergy to miglustat or any of the components within the drug product will be excluded from this study. miglustat, Ketogenic Diet, miglustat, Ketogenic Diet
GM1 Gangliosidoses, GM2 Gangliosidoses, Tay-Sachs Disease, Sandhoff Disease
infantile Tay-Sachs disease, juvenile Tay-Sachs disease, infantile GM1 gangliosidosis, juvenile GM1 gangliosidosis, infantile GM2 gangliosidosis, juvenile GM2 gangliosidosis, Sandhoff disease, gangliosidoses, miglustat, ketogenic diet, SYNER-G regimen, Syner-G, Zavesca, Tay-Sachs disease, Tay Sachs disease
Neoadjuvant therapy for patients with high risk stage III melanoma: a pilot clinical trial (NeoACTIVATE)
To estimate the percentage of patients with stage III BRAFm melanoma that achieves a pathologic complete response after 12 weeks of neoadjuvant vemurafenib/cobimetinib/atezolizumab. To estimate the percentage of patients with stage III BRAFwt melanoma that achieves a pathologic complete response after 12 weeks of neoadjuvant cobimetinib/atezolizumab. Adjuvant phase primary objectives: To assess recurrence-free survival (RFS) in patients with stage III BRAFm melanoma after neoadjuvant vemurafenib/cobimetinib/atezolizumab, surgery, and adjuvant atezolizumab. To assess RFS in patients with stage III BRAFwt melanoma after neoadjuvant cobimetinib/atezolizumab, surgery, and adjuvant atezolizumab.
Evidio Domingo Musibay
All
18 Years to old
Phase 2
STUDY00004666
STUDY00004666
Inclusion Criteria:
PRE-REGISTRATION: High-risk stage III melanoma, defined as (any of the following):
Recurrent nodal metastasis, or
Clinically detectable nodal metastasis, or
Metastatic involvement of more than one nodal basin
NOTE: For the purpose of pre-registration, high-risk stage III melanoma is defined based on clinical and imaging assessment (positron emission tomography/computed tomography [PET/CT], CT, or magnetic resonance imaging [MRI]). Histologic confirmation of nodal metastatic disease is not needed at the time of pre-registration, provided there is histologic confirmation of primary melanoma or a prior lymph node metastasis.
PRE-REGISTRATION: Willing to submit archival tissue from a lymph node biopsy or undergo a needle biopsy (with clip placement) for BRAF testing and for research purposes.
PRE-REGISTRATION: Willing to forego anticancer treatments or investigational agents during pre-registration period.
PRE-REGISTRATION: The following laboratory values obtained =< 28 days prior to pre-registration:
Only for patients receiving therapeutic anticoagulation: stable anticoagulant regimen and stable international normalized ratio (INR).
REGISTRATION: Histologic confirmation of stage III melanoma, as defined by the American Joint Committee on Cancer, 8th revised edition.
REGISTRATION: Documentation of BRAFV600 mutation status in melanoma tumor tissue (archival or newly obtained) through use of a Clinical Laboratory Improvement Amendments (CLIA)-approved clinical mutation test.
REGISTRATION: Surgically resectable disease, as determined by a melanoma surgical oncologist.
REGISTRATION: Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
REGISTRATION: Life expectancy >= 26 weeks.
REGISTRATION: Absolute neutrophil count (ANC) >= 1500/mm^3 obtained =< 14 days prior to registration.
REGISTRATION: Platelet count >= 100,000/mm^3 obtained =< 14 days prior to registration.
REGISTRATION: Hemoglobin >= 9.0 g/dL obtained =< 14 days prior to registration.
REGISTRATION: Direct bilirubin =< institutional upper limit of normal (ULN) obtained =< 14 days prior to registration.
REGISTRATION: Aspartate transaminase (AST) and alanine transaminase (ALT) =< 2 x ULN obtained =< 14 days prior to registration.
REGISTRATION: Alkaline phosphatase < 2.5 x ULN obtained =< 14 days prior to registration.
REGISTRATION: Creatinine =< 1.5 x ULN or creatinine clearance (CrCl) >= 45 mL/min on the basis of measured CrCl from a 24-hour urine collection or Cockcroft-Gault glomerular filtration rate estimation obtained =< 14 days prior to registration.
REGISTRATION: Arms A and B only: Left ventricular ejection fraction (LVEF) >= 50% or institutional lower limit of normal (LLN) =< 6 months prior to registration.
REGISTRATION: Arms A and B only: Average corrected QT interval (QTc) =< 450 ms on triplicate 12 lead electrocardiography (ECG) =< 28 days prior to registration.
NOTE: QTc intervals will be corrected using Fridericia's formula.
REGISTRATION: Negative pregnancy test done =< 7 days prior to registration, for persons of childbearing potential only.
REGISTRATION: For persons of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use a contraceptive method with a failure rate of < 1% per year during the treatment period and for 6 months after the last dose of study treatment.
REGISTRATION: For persons able to father a child: agreement to remain abstinent (refrain from heterosexual intercourse with a person of childbearing potential) or use contraceptive measures, and agreement to refrain from donating sperm during the treatment period and for 6 months after the last dose of study treatment.
REGISTRATION: Provide written informed consent.
REGISTRATION: Willing to return to enrolling institution for follow-up (during the active monitoring phase of the study).
REGISTRATION: Willing to provide tissue, blood, and stool samples for correlative research purposes.
REGISTRATION: Arm C Only: Negative serology for acute Epstein-Barr virus (EBV) infection (negative EBV viral capsid antigen [VCA] immunoglobulin M [IgM]).
Exclusion Criteria:
PRE-REGISTRATION: Prior systemic anti-cancer therapy for melanoma (e.g., chemotherapy, hormonal therapy, targeted therapy, immunotherapy including anti-PD-1, anti-PDL1 agents, or other biologic therapies), with the following exceptions: adjuvant treatment with interferon, IL-2, granulocyte-macrophage colony-stimulating factor (GM-CSF) or vaccine therapies are allowed, if discontinued >= 28 days prior to pre-registration.
PRE-REGISTRATION: Receiving any other investigational agent which would be considered as a treatment for the primary neoplasm.
PRE-REGISTRATION: For patients with concurrent diagnosis of primary melanoma with nodal involvement, major surgical procedure other than lymph node biopsy or wide local excision of primary melanoma =< 4 weeks prior to pre-registration, or anticipation of need for a major surgical procedure for reasons other than melanoma during the course of the study.
PRE-REGISTRATION: For patients with nodal recurrence, surgical procedure or anti-cancer therapy for this recurrence (other than lymph node biopsy) or anticipation of need for a major surgical procedure for reasons other than melanoma during the course of the study.
PRE-REGISTRATION: Prior radiotherapy for melanoma.
PRE-REGISTRATION: History of non-nodal melanoma metastasis or central nervous system (CNS) lesion(s) proven or clinically suspected to be metastasis.
PRE-REGISTRATION: Active malignancy (other than melanoma) or malignancy =< 3 years prior to pre-registration.
NOTE: Exceptions: Asymptomatic papillary thyroid cancer (not requiring treatment), resected basal cell carcinoma (BCC), resected cutaneous squamous cell carcinoma (SCC), resected carcinoma in situ of the cervix, resected carcinoma in situ of the breast, in situ prostate cancer, non-muscle-invasive bladder cancer, Stage I uterine cancer, or other curatively treated malignancies from which the patient has been disease-free for at least 3 years prior to pre registration.
PRE-REGISTRATION: Prior allogeneic stem cell or solid organ transplantation.
PRE-REGISTRATION: History of idiopathic pulmonary fibrosis, organizing pneumonia (e.g., bronchiolitis obliterans), drug-induced pneumonitis, or idiopathic pneumonitis, or evidence of active pneumonitis on screening chest CT scan.
PRE-REGISTRATION: History of autoimmune disease requiring systemic immunosuppressive or immune-modulatory therapy =< 5 years prior to pre-registration.
NOTE: Exceptions are allowed for hypothyroidism on thyroid replacement therapy; or Type 1 diabetes on insulin regimen.
PRE-REGISTRATION: Active psoriasis requiring therapy (systemic or topical).
PRE-REGISTRATION: Known clinically significant liver disease, including alcoholism, cirrhosis, fatty liver, and other inherited liver disease as well as active viral disease.
PRE-REGISTRATION: Arms A and B only: History of or evidence of retinal pathology on ophthalmologic examination including but not limited to:
Neurosensory retinal detachment
Central serous chorioretinopathy
Retinal vein occlusion (RVO)
Neovascular macular degeneration
PRE-REGISTRATION: Immunocompromised patients and patients known to be human immunodeficiency virus (HIV) positive and currently receiving antiretroviral therapy.
NOTE: Patients known to be HIV positive, but without clinical evidence of an immunocompromised state, are eligible for this trial.
PRE-REGISTRATION: Uncontrolled intercurrent illness including, but not limited to:
Ongoing or active infection (including but not limited to tuberculosis)
Clinically significant cardiac dysfunction including:
Symptomatic congestive heart failure defined as New York Heart Association class II or higher
Unstable angina pectoris or new-onset angina =< 3 months prior to pre-registration
Unstable cardiac arrhythmia
Myocardial infarction =< 3 months prior to pre-registration
Congenital long QT syndrome
Clinically significant stroke, reversible ischemic neurological defect, or transient ischemic attack =< 6 months prior to pre-registration
Any grade 3 hemorrhage or bleeding event =< 4 weeks prior to pre-registration
Uncontrolled diabetes or symptomatic hyperglycemia
Psychiatric illness/social situations that, in the judgement of the investigator, would: a) limit compliance with study requirements, or b) make the patient inappropriate for entry into this study, or c) interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens.
PRE-REGISTRATION: Known hypersensitivity to biopharmaceutical agents produced in Chinese hamster ovary cells (example [ex]: recombinant follicle-stimulating hormone [FSH]).
PRE-REGISTRATION: Known hypersensitivity to any components of the atezolizumab (all arms), tiragolumab (Arm C only), cobimetinib (Arms A and B only), or vemurafenib (Arms A and B only) formulations.
PRE-REGISTRATION: History of severe allergic, anaphylactic or other hypersensitivity reactions to chimeric or humanized antibodies or fusion proteins.
REGISTRATION: Received anticancer treatments or investigational agents during pre-registration period.
REGISTRATION: Clinically suspected non-nodal metastatic melanoma.
REGISTRATION: Arm A only: For BRAF-mutant patients only: anticipated use of any concomitant medication =< 7 days prior to registration that is known to cause QT prolongation (which may lead to torsade de pointes).
REGISTRATION: Arms A and B only: History of malabsorption or other clinically significant metabolic dysfunction that may interfere with absorption of oral study treatment or inability or unwillingness to swallow oral medication.
REGISTRATION: Signs or symptoms of infection or has received antibiotics =< 14 days prior to registration.
NOTE: Patients receiving prophylactic antibiotics (e.g., to prevent a urinary tract infection or chronic obstructive pulmonary disease exacerbation) are eligible for the study.
REGISTRATION: Any of the following because this study involves investigational agents whose genotoxic, mutagenic and teratogenic effects on the developing fetus and newborn are unknown:
Pregnant persons
Nursing persons
Persons of childbearing potential who are unwilling to employ adequate contraception
REGISTRATION: Treatment with a live, attenuated vaccine =< 4 weeks prior to registration, or anticipation of need for such a vaccine during the course of the study.
REGISTRATION: Treatment with systemic immunosuppressive medication (including, but not limited to, prednisone, cyclophosphamide, azathioprine, methotrexate, thalidomide, and anti-tumor necrosis factor (TNF)-alpha agents) =< 2 weeks prior to registration, or anticipation of need for systemic immunosuppressive medication during the course of the study.
NOTE: Patients who have received acute, low-dose systemic steroids (=< 10 mg/day oral prednisone or equivalent) prior to registration or a one-time pulse dose of systemic immunosuppressant medication (e.g., 48 hours of corticosteroids for a contrast allergy) are eligible for the study.
NOTE: The use of inhaled corticosteroids for chronic obstructive pulmonary disease or asthma, mineralocorticoids (e.g., fludrocortisone), or low-dose corticosteroids for patients with orthostatic hypotension or adrenocortical insufficiency is allowed.
REGISTRATION: Requirement for concomitant therapy or food that is prohibited during the study.
REGISTRATION: Arms A and B only: Inability to abstain from alcohol during neoadjuvant phase.
REGISTRATION: Arm C only: Known Epstein-Barr virus (EBV) infection.
NOTE: Patients with symptoms such as splenomegaly, fever, sore throat, non-malignant cervical lymphadenopathy, and/or tonsillar exudate, should undergo an EBV polymerase chain reaction (PCR) test to screen for acute infection or suspected chronic active infection. Patients with a positive EBV PCR test are excluded. Atezolizumab, Cobimetinib, Tiragolumab, Vemurafenib, Atezolizumab, Cobimetinib, Tiragolumab, Vemurafenib
Clinical Stage III Cutaneous Melanoma AJCC v8, Pathologic Stage III Cutaneous Melanoma AJCC v8, Pathologic Stage IIIA Cutaneous Melanoma AJCC v8, Pathologic Stage IIIB Cutaneous Melanoma AJCC v8, Pathologic Stage IIIC Cutaneous Melanoma AJCC v8, Pathologic Stage IIID Cutaneous Melanoma AJCC v8
Clinics and Surgery Center (CSC)
An Adaptive Algorithm-Based Approach to Treatment for Adolescent Depression
The purpose of the current study is to evaluate the effectiveness of two adaptive treatment strategies (ATSs) for adolescent depression. The ATSs include delivery of an evidence-based psychotherapy for adolescent depression (interpersonal psychotherapy, IPT-A), systematic symptom monitoring, and an empirically-derived algorithm that specifies whether, when, and how to augment IPT-A. Two hundred depressed adolescents (age 12-18) will be recruited to participate in a 16-week SMART conducted in an outpatient community mental health clinic. Adolescents will be randomized to the IPT-A ATS condition (N=134) or the community clinic’s usual care (UC) (N=66). The aims of this R01 are to (1) evaluate the effectiveness of the ATSs embedded in this trial, (2) evaluate adolescents’ interpersonal functioning as a treatment target of IPT-A, (3) evaluate moderators of initial treatment and treatment augmentation strategies, and (4) conduct a process evaluation to identify barriers and facilitators that influenced ATS implementation.
Meredith Gunlicks-Stoessel
All
12 Years to 18 Years old
Phase II
STUDY00000460
STUDY00000460
Inclusion Criteria:
Meet DSM-V criteria for a primary diagnosis of Major Depressive Disorder, Persistent Depressive Disorder, or Depressive Disorder NEC
Current significant depressive symptoms (based on Children's Depression Rating Scale - Revised [CDRS-R] & Beck Depression Inventory-II [BDI-II])
Current impairment in psychosocial functioning (based on Children's Global Assessment Scale [CGAS])
Exclusion Criteria:
Non English-speaking
Meet DSM-V criteria for bipolar disorder, psychosis, anorexia nervosa, substance use disorder, autism spectrum disorder, or intellectual disability disorder.
Adolescents who are actively suicidal with a plan and/or intent who are assessed to need a higher level of care than outpatient treatment due to safety risk will be referred for appropriate level of stabilization. Once stabilized, the adolescent can be re-evaluated for eligibility to participate in the study.
Currently taking medication for a psychiatric diagnosis other than ADHD
Females who are pregnant, breastfeeding, or having unprotected sexual intercourse, due to the possibility of randomization to treatment with an SSRI. Interpersonal Psychotherapy for Depressed Adolescents, Selective Serotonin Reuptake Inhibitor, Usual Care, Interpersonal Psychotherapy for Depressed Adolescents, Selective Serotonin Reuptake Inhibitor, Usual Care
Depressive Disorder
Low sulfur fecal transplant for ulcerative colitis
This study is a pilot randomized controlled clinical trial examining how fecal microbiota transplant (FMT) given by capsules can change the bacteria and inflammation in people with active ulcerative colitis (UC). We will look at global changes of bacterial composition while on FMT versus those not on FMT. We are examining some specific groups of bacteria that are related to sulfate reduction. Will will measure the changes of sulfate reducing bacteria over time and among those who get better and those who don't. Overall, we aim to determine if we can alter the microbiota in UC towards a healthy, more diverse microbiota resembling the donor using capsule FMT material.
Byron Vaughn
All
18 Years to 89 Years old
Phase 1
STUDY00005279
STUDY00005279
Inclusion Criteria:
Able and willing to provide consent
English speaking
Diagnosis of ulcerative colitis based on typical clinical-histopathic diagnosis
Diagnosis of ulcerative colitis > 3 months
Active disease on endoscopy (endoscopic Mayo subscore ≥ 1)
Evidence of inflammation extending beyond a minimum of 20cm
Any ongoing ulcerative colitis therapy must be at stable doses for 4 weeks prior to study and remain stable over the course of the study
Exclusion Criteria:
Extensive bowel resection
Presence of ileostomy or colostomy
Suspicion of ischemic colitis, radiation colitis or microscopic colitis
Diagnosis of Crohn's disease
Diagnosis of per-anal fistula or abscess
Adenomatous polyps that have not been removed
Use of pre or probiotics within 30 days of randomization
Pregnancy
Severe food allergies
End stage liver disease or cirrhosis
An absolute neutrophil count < 500 cell/µL
Life expectancy < 6 months Fecal microbiota, Placebo, Fecal microbiota, Placebo
Digestive & Liver Health
inflammatory bowel disease
Enhanced Spatial Targeting in ECT Utilizing Focally Electrically-administered Seizure Therapy (FEAST)
The purpose of this study is to look at a different type of electroconvulsive therapy (ECT) that may reduce negative side effects while still providing relief from symptoms of major depression.
Ziad Nahas
All
22 Years to 90 Years old
N/A
STUDY00006734
STUDY00006734
Inclusion Criteria:
Diagnosis of major depressive disorder using mini-7 to derive RDC; DSM-IV
Pretreatment HRSC score greater than or equal to 18
ECT indicated by physician evaluation
Willing and capable of providing informed consent as determined by physician evaluation
Exclusion Criteria:
History of schizophrenia, schizoaffective disorder, other functional psychosis, or rapid cycling bipolar disorder as determined by mini-7; rapid cycling defined as greater than or equal to four episodes in past year
History of neurological illness or insult other than conditions associated with psychotropic exposure (e.g., tardive dyskinesia) determined by physician evaluation and medical history
Alcohol or substance abuse or dependence in the past year (RDC) determined by physician evaluation
Secondary diagnosis of a delirium, dementia, or amnestic disorder (DSM-IV), pregnancy, or epilepsy determined by physician evaluation
Requires especially rapid antidepressant response due to suicidality, psychosis, inanition, psychosocial obligations, etc. determined by physician evaluation
ECT in the past six months determined by physician evaluation and medical history
Pregnancy as determined by urine pregnancy test and clinical interview FEAST, FEAST RP, FEAST RC, FEAST, FEAST RP, FEAST RC
Treatment Resistant Depression
Bupropion for the Prevention of Postpartum Smoking Relapse
Sharon Allen, PhD
Female
18 Years to 40 Years old
Phase 4
STUDY00007684
STUDY00007684
Inclusion Criteria:
Ability to provide informed consent
Age 18 to 40 years old
Stable health
7-day point prevalence abstinence demonstrated at randomization
Lifetime history of at least 100 cigarettes smoked
Quit smoking during the current pregnancy
Self-report of intention to remain abstinent after delivery ≥ 7 on a 10 point Likert-type scale
Uncomplicated delivery
Denies plans to become pregnant again during the trial.
Full-term delivery ≥ 37 weeks gestation
Home within 10 days of delivery
Exclusion Criteria:
Current use of other forms of tobacco or nicotine (e-cigs, chew, snuff, etc.)
Current use of cessation aids (e.g., varenicline, NRT)
Current use of illicit drugs or alcohol dependence
Current use of antidepressant medication
Bipolar disorder, eating disorder, or psychotic disorder based on the Structured Clinical Interview
Medications & conditions that may increase the risk of taking bupropion (e.g., current or history of pulmonary embolus, stroke, heart disease, kidney disease, glaucoma, diabetes, seizure disorder, traumatic head injury, use of medications metabolized by CYP2D6)
Family history of seizures or seizure disorder
Maternal use of medications that lower seizure threshold
Newborn with an elevated risk of seizure Bupropion Extended Release Oral Tablet, Placebo oral tablet, Bupropion Extended Release Oral Tablet, Placebo oral tablet
Postpartum Smoking Relapse
MT2017-45 :Chimeric Antigen Receptor (CAR)-T Cell Therapy for Patients with Hematologic Malignancies
This is a phase II study of FDA-approved CAR-T products for patients with hematologic malignancies. The study provides criteria for consistent treatment and management according to FDA labelling of CAR-T products and does not contain experimental components. Patients will be assigned to Arms A B and C based on age, CAR-T product and diagnosis. Overall remission rate, safety events and other endpoints will be calculated for Arms A B and C separately.
Veronika Bachanova, MD
All
STUDY00004096
STUDY00004096
ARM A (Kymriah) and Arm G (Tecartus) :Refractory/relapsed B-cell acute lymphoblastic leukemia expressing CD19
Inclusion Criteria:
Age and Disease Status
Must be age 0-25 years (for Arm A Kymriah) or >18 years (Arm G Tecartus)
Disease status: Relapsed and refractory pediatric B-cell ALL defined by one of these:
Primary induction failure with no complete remission after ≥2 cycles of induction chemotherapy, or
Patients with persistent minimal residual disease (MRD >0.01% by flow cytometry or persistent by cytogenetic or molecular assays) after ≥2 cycles of consolidation chemotherapy, or
Patients in 2nd or greater relapse of B-ALL or
Patients with persistent CNS leukemia, or
Down Syndrome or other congenital diseases assuming that they fit the criteria for second or greater relapse or refractory leukemia, or
Patients with Ph+ ALL are eligible if theywho have failed or are intolerant to two lines of TKI assuming they fit the criteria for second or greater relapse or are considered refractory.
Performance Status
* Arm A: Karnofsky (age ≥16 years) or Lansky (age < 16 years) performance status ≥ 50% at screening; Arm G: ECOG 0, 1 or 2
Organ Function
Renal function defined as:
A serum creatinine of ≤1.5 x ULN OR
eGFR ≥ 50 mL/min/1.73 m2
Liver function defined as:
** ALT ≤ 5 times the ULN for age (unless due to disease)
** Bilirubin ≤ 2.0 mg/dl with the exception of patients with Gilbert syndrome; may be included if their total bilirubin is ≤ 3.0 x ULN and direct bilirubin ≤ 1.5 x ULN
Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and pulse oxygenation SpO2 > 91% on room air
Hemodynamically stable and LVEF ≥ 45% confirmed by echocardiogram or MUGA
Other Inclusion Criteria
Life expectancy ≥12 weeks
Women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment.
Written voluntary consent (adults) or parental/guardian consent (minors or adults with diminished capacity) prior to the performance of any research related tests or procedures.
Exclusion Criteria:
Pregnant or breastfeeding - Females of childbearing potential must have a blood test or urine study within 14 days prior to registration to rule out pregnancy.
Patients with Burkitt's lymphoma/leukemia (i.e. patients with mature B-cell ALL, leukemia with B-cell [sIg positive and kappa or lambda restricted positivity] ALL, with FAB L3 morphology and /or a MYC translocation)
CNS 2A
CAR-T is not indicated for the treatment of patients with primary central nervous system lymphoma.
Presence of Grade 2 to 4 acute or extensive chronic graft-versus-host disease (GVHD). All GVHD medication must be stopped 2 weeks prior to apheresis.
Uncontrolled active hepatitis B or hepatitis C
Active HIV infection
Uncontrolled acute life threatening bacterial, viral or fungal infection (e.g. blood culture positive ≤ 72 hours prior to infusion)
Unstable angina and/or myocardial infarction within 1 month prior to CAR-T infusion
Investigational medicinal product within the last 7 days prior to apheresis or CAR-T infusion
Intolerance to the excipients of the CAR-T cell product
Any immunosuppressive medication must be stopped ≥ 2 weeks prior to enrollment.
Patient has taken one of the prohibited concomitant medications within the timeframe outlined in section 6.1
ARM B: Yescarta for Relapsed or Refractory diffuse large B cell lymphoma
Inclusion Criteria:
Age and Disease Status
Adult patients (age ≥ 18 years)Patients must be ≥18 years of age
One of the following histologies and expression of CD19 by tumor cells:
** diffuse large B-cell lymphoma (DLBCL) not otherwise specified, or
** primary mediastinal large B-cell lymphoma, or
** high grade B-cell lymphoma, or
** DLBCL arising from follicular lymphoma
Disease status:
** Chemotherapy refractory disease after ≥2 lines of chemotherapy, or
** Relapsed with no remission after ≥1 lines of salvage chemotherapy, or
** Relapsed following autologous HCT (and failed at least 2 prior lines of therapy including high dose chemotherapy). If salvage therapy is given post autoHCT, the subject must have no response or relapse after the last line of therapy
Measurable disease at time of apheresis: Nodal lesions or extranodal lesion
ECOG performance status 0-2
ALC >/=100/uL at screening (prior to apheresis)
Renal function defined as:
** A serum creatinine of ≤1.5 x ULN OR
** eGFR ≥ 50 mL/min/1.73 m2
Liver function defined as:
ALT ≤ 5 times the ULN for age (unless due to disease)
Bilirubin ≤ 2.0 mg/dl with the exception of patients with Gilbert syndrome; may be included if their total bilirubin is ≤ 3.0 x ULN and direct bilirubin ≤ 1.5 x ULN
Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and pulse oxygenation SpO2 > 91% on room air
Hemodynamically stable and LVEF ≥ 45% confirmed by echocardiogram or MUGA
Adequate bone marrow reserve (unless marrow infiltrated by disease) defined as :
Absolute neutrophil count (ANC) > 1.000/mm3 (only for NHL)
Platelets ≥ 50.000/mm3 (transfusion support can be provided)
Hemoglobin >8.0 mg/dl (transfusion support can be provided)
Life expectancy ≥12 weeks
Women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment.
Written voluntary consent (adults) or parental/guardian consent (minors or adults with diminished capacity) prior to the performance of any research related tests or procedures.
Exclusion Criteria:
Pregnant or breastfeeding - Females of childbearing potential must have a blood test or urine study within 14 days prior to registration to rule out pregnancy.
Active CNS involvement by malignancy (no evidence of disease in CSF by flow cytometry) CAR-T is not indicated for the treatment of patients with primary central nervous system lymphoma.
Presence of Grade 2 to 4 acute or extensive chronic graft-versus-host disease (GVHD). All GVHD medication must be stopped 2 weeks prior to apheresis.
Uncontrolled active hepatitis B or hepatitis C
Active HIV infection (controlled HIV is permissible)
Uncontrolled acute life threatening bacterial, viral or fungal infection (e.g. blood culture positive ≤ 72 hours prior to infusion)
Unstable angina and/or myocardial infarction within 1 month prior to CAR-T infusion
Investigational medicinal product within the last 7 days prior to apheresis or CAR-T infusion
Intolerance to the excipients of the CAR-T cell product
Any immunosuppressive medication must be stopped ≥ 2 weeks prior to apheresis.
Patient has taken one of the prohibited concomitant medications within the timeframe.
ARM C: Kymriah for rRelapsed or rRefractory diffuse large B cell lymphoma
Inclusion Criteria:
Age and Disease Status
Adult patients (age ≥ 18 years)
with relapsed or refractory (r/r) large B-cell lymphoma, including
diffuse large B-cell lymphoma (DLBCL) not otherwise specified,
high grade B-cell lymphoma
and DLBCL arising from follicular lymphoma.
Disease status:
after two or more lines of systemic therapy or
relapse after autologous HCT
Performance Status
ECOG performance status 0-2
ALC >/=100/uL at screening (prior to apheresis)
Organ Function
Renal function defined as:
A serum creatinine of ≤1.5 x ULN OR
eGFR ≥ 50 mL/min/1.73 m^2
Liver function defined as:
ALT ≤ 5 times the ULN for age (unless due to disease)
Bilirubin ≤ 2.0 mg/dl with the exception of patients with Gilbert syndrome; may be included if their total bilirubin is ≤ 3.0 x ULN and direct bilirubin ≤ 1.5 x ULN
Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and pulse oxygenation SpO2 > 91% on room air
Hemodynamically stable and LVEF ≥ 45% confirmed by echocardiogram or MUGA
Adequate bone marrow reserve (unless marrow infiltrated by disease) defined as :
Absolute neutrophil count (ANC) > 1.000/mm3 (only for NHL)
Platelets ≥ 50.000/mm3 (transfusion support can be provided)
Hemoglobin >8.0 mg/dl (transfusion support can be provided)
Other Inclusion Criteria
Life expectancy ≥12 weeks
Women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment.
Written voluntary consent (adults) or parental/guardian consent (minors or adults with diminished capacity) prior to the performance of any research related tests or procedures.
Exclusion Criteria:
Pregnant or breastfeeding - Females of childbearing potential must have a blood test or urine study within 14 days prior to registration to rule out pregnancy.
Active CNS involvement by malignancy (no evidence of disease in CSF by flow cytometry) CAR-T is not indicated for the treatment of patients with primary central nervous system lymphoma.
Presence of Grade 2 to 4 acute or extensive chronic graft-versus-host disease (GVHD). All GVHD medication must be stopped 2 weeks prior to apheresis.
Uncontrolled active hepatitis B or hepatitis C
Active or inactive HIV infection
Uncontrolled acute life threatening bacterial, viral or fungal infection (e.g. blood culture positive ≤ 72 hours prior to infusion)
Unstable angina and/or myocardial infarction within 1 month prior to CAR-T infusion
Investigational medicinal product within the last 7 days prior to apheresis or CAR-T infusion
Intolerance to the excipients of the CAR-T cell product
Any immunosuppressive medication must be stopped ≥ 2 weeks prior to apheresis.
Patient has taken one of the prohibited concomitant medications within the timeframe
ARM D: Tecartus (Brexucabtagene Autoleucel) for relapsed or refractory mantle cell lymphoma
Inclusion Criteria:
Age and Disease Status
* with relapsed or refractory (r/r) mantle cell lymphoma, including
prior anthracycline or Bendamustine containing therapy
prior Rituximab or other CD20 directed antibody (or inability to treat with CD20 MoAb)
not a candidate or relapse after autologous HCT
active disease at enrollment
Performance Status
*ECOG performance status 0-1
Organ Function
Renal function defined as:
A serum creatinine of ≤1.5 x ULN OR
eGFR ≥ 50 mL/min/1.73 m2
Liver function defined as:
ALT ≤ 5 times the ULN for age (unless due to disease)
Bilirubin ≤ 2.0 mg/dl with the exception of patients with Gilbert syndrome; may be included if their total bilirubin is ≤ 3.0 x ULN and direct bilirubin ≤ 1.5 x ULN
Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and pulse oxygenation SpO2 > 91% on room air
Hemodynamically stable and LVEF ≥ 45% confirmed by echocardiogram or MUGA
Adequate bone marrow reserve (unless marrow infiltrated by disease) defined as:
Absolute neutrophil count (ANC) > 1,000/mm^3 (only for NHL)
Platelets ≥ 50,000/mm^3 (transfusion support can be provided)
Hemoglobin >8.0 mg/dl (transfusion support can be provided)
Other Inclusion Criteria:
Life expectancy ≥12 weeks
Women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment. See section 4.5 for definitions of child bearing potential and section 4.6 for definitions of adequate birth control.
Written voluntary consent (adults) or parental/guardian consent (minors or adults with diminished capacity) prior to the performance of any research related tests or procedures.
Exclusion Criteria:
Pregnant or breastfeeding - Females of childbearing potential must have a blood test or urine study within 14 days prior to registration to rule out pregnancy.
Active CNS involvement by malignancy (no evidence of disease in CSF by flow cytometry) CAR-T is not indicated for the treatment of patients with primary central nervous system lymphoma.
Presence of Grade 2 to 4 acute or extensive chronic graft-versus-host disease (GVHD). All GVHD medication must be stopped 2 weeks prior to apheresis.
Uncontrolled active hepatitis B or hepatitis C
Active HIV infection
Uncontrolled acute life threatening bacterial, viral or fungal infection (e.g. blood culture positive ≤ 72 hours prior to infusion)
Unstable angina and/or myocardial infarction within 1 month prior to CAR-T infusion
Investigational medicinal product within the last 7 days prior to apheresis or CAR-T infusion
Intolerance to the excipients of the CAR-T cell product
Any immunosuppressive medication must be stopped ≥ 2 weeks prior to apheresis (steroids must be stopped >72 hours prior to apheresis).
Patient has taken one of the prohibited concomitant medications within the timeframe
ARM E: Breyanzi "lisocabtagene maraleucel" for relapsed or refractory large B-cell lymphoma
Inclusion Criteria:
Age and Disease Status
Adult patients (age ≥ 18 years)
with relapsed or refractory disease after two or more lines of systemic therapy, including
diffuse large B-cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma),
high-grade B-cell lymphoma,
primary mediastinal large B-cell lymphoma,
follicular lymphoma grade 3B
Performance Status
*ECOG performance status 0-2
Organ Function
Renal function defined as:
A serum creatinine of ≤1.5 x ULN OR
eGFR ≥ 30 mL/min/1.73 m2
Liver function defined as:
ALT ≤ 5 times the ULN for age (unless due to disease)
Bilirubin ≤ 2.0 mg/dl with the exception of patients with Gilbert syndrome; may be included if their total bilirubin is ≤ 3.0 x ULN and direct bilirubin ≤ 1.5 x ULN
Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and pulse oxygenation SpO2 > 91% on room air
Hemodynamically stable and LVEF ≥ 40% confirmed by echocardiogram or MUGA
Adequate bone marrow reserve (unless marrow infiltrated by disease) defined as:
Absolute neutrophil count (ANC) > 1,000/mm^3 (only for NHL)
Platelets ≥ 50,000/mm^3 (transfusion support can be provided)
Hemoglobin >8.0 mg/dl (transfusion support can be provided)
Other Inclusion Criteria:
Life expectancy ≥12 weeks
Women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment. See section 4.5 for definitions of child bearing potential and section 4.6 for definitions of adequate birth control.
Written voluntary consent (adults) or parental/guardian consent (minors or adults with diminished capacity) prior to the performance of any research related tests or procedures.
Exclusion Criteria:
Pregnant or breastfeeding - Females of childbearing potential must have a blood test or urine study within 14 days prior to registration to rule out pregnancy.
Active CNS involvement by malignancy (no evidence of disease in CSF by flow cytometry) CAR-T is not indicated for the treatment of patients with primary central nervous system lymphoma.
Presence of Grade 2 to 4 acute or extensive chronic graft-versus-host disease (GVHD). All GVHD medication must be stopped 2 weeks prior to apheresis.
Uncontrolled active hepatitis B or hepatitis C
Active HIV infection
Uncontrolled acute life threatening bacterial, viral or fungal infection (e.g. blood culture positive ≤ 72 hours prior to infusion)
Unstable angina and/or myocardial infarction within 1 month prior to CAR-T infusion
Investigational medicinal product within the last 7 days prior to apheresis or CAR-T infusion
Intolerance to the excipients of the CAR-T cell product
Any immunosuppressive medication must be stopped ≥ 2 weeks prior to apheresis (steroids must be stopped >72 hours prior to apheresis).
Patient has taken one of the prohibited concomitant medications within the timeframe
ARM F: Abecma "Idecabtagene Vicleucel" for relapsed or refractory multiple myeloma
Inclusion Criteria:
Age and Disease Status
Adult patients (age ≥ 18 years)
Relapsed (progression after prior partial or complete remission) or refractory multiple myeloma
Evidence of active disease (medullary or extramedullary)
Prior therapy (Failure or intolerance to) with an immunomodulatory agent, a proteasome inhibitor, and an antiCD38 monoclonal antibody
Performance Status
*ECOG performance status 0-1
Organ Function
Renal function defined as:
A serum creatinine of ≤2 x ULN OR
eGFR ≥ 50 mL/min/1.73 m2
Liver function defined as:
ALT ≤ 5 times the ULN for age (unless due to disease)
Bilirubin ≤ 2.0 mg/dl with the exception of patients with Gilbert syndrome; may be included if their total bilirubin is ≤ 3.0 x ULN and direct bilirubin ≤ 1.5 x ULN
Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and pulse oxygenation SpO2 > 91% on room air
Hemodynamically stable and LVEF ≥ 45% confirmed by echocardiogram or MUGA
Adequate bone marrow reserve (unless marrow infiltrated by disease) defined as:
Absolute neutrophil count (ANC) > 1,000/mm^3 (only for NHL)
Platelets ≥ 50,000/mm^3 (transfusion support can be provided)
Hemoglobin >8.0 mg/dl (transfusion support can be provided)
Other Inclusion Criteria:
Life expectancy ≥12 weeks
Women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment. See section 4.5 for definitions of child bearing potential and section 4.6 for definitions of adequate birth control.
Written voluntary consent (adults) or parental/guardian consent (minors or adults with diminished capacity) prior to the performance of any research related tests or procedures.
Exclusion Criteria:
Pregnant or breastfeeding - Females of childbearing potential must have a blood test or urine study within 14 days prior to registration to rule out pregnancy.
Active CNS involvement by malignancy (no evidence of disease in CSF by flow cytometry) CAR-T is not indicated for the treatment of patients with primary central nervous system lymphoma.
Presence of Grade 2 to 4 acute or extensive chronic graft-versus-host disease (GVHD). All GVHD medication must be stopped 2 weeks prior to apheresis.
Uncontrolled active hepatitis B or hepatitis C
Active HIV infection
Uncontrolled acute life threatening bacterial, viral or fungal infection (e.g. blood culture positive ≤ 72 hours prior to infusion)
Unstable angina and/or myocardial infarction within 1 month prior to CAR-T infusion
Investigational medicinal product within the last 7 days prior to apheresis or CAR-T infusion
Intolerance to the excipients of the CAR-T cell product
Any immunosuppressive medication must be stopped ≥ 2 weeks prior to apheresis (steroids must be stopped >72 hours prior to apheresis).
Patient has taken one of the prohibited concomitant medications within the timeframe KYMRIAH, YESCARTA, Fludarabine 30mg/m2 4 doses, Cyclophosphamide 500 mg/m2, 2 doses, Fludarabine 30mg/m2 3 doses, Cyclophosphamide 500 mg/m2, 3 doses, Fludarabine 25mg/m2 3 days, Cyclophosphamide 250 mg/m2, 3 days, Tecartus, Abecma, Intravenous Suspension, Cyclophosphamide 900 mg/m2, 1 day, Breyanzi Injectable Product, KYMRIAH, YESCARTA, Fludarabine 30mg/m2 4 doses, Cyclophosphamide 500 mg/m2, 2 doses, Fludarabine 30mg/m2 3 doses, Cyclophosphamide 500 mg/m2, 3 doses, Fludarabine 25mg/m2 3 days, Cyclophosphamide 250 mg/m2, 3 days, Tecartus, Abecma, Intravenous Suspension, Cyclophosphamide 900 mg/m2, 1 day, Breyanzi Injectable Product
Acute Lymphoblastic Leukemia, Large B-cell Lymphoma
Clinics and Surgery Center (CSC)
MT2017-17: Alpha/Beta T Cell Depleted (alpha/beta TCD) Hematopoietic Cell Transplantation in Patients with Fanconi Anemia
The primary objective is to determine the incidence of grade II-IV acute graft versus host disease (GVHD) by Day 100 using an alpha/beta T cell depleted peripheral blood stem cells (PBSC) and without routine GVHD prophylaxis.
Margaret MacMillan, MD
STUDY00003182
STUDY00003182
Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1), Fludarabine (FLU), Methylprednisolone (MP), Donor mobilized PBSC infusion, G-CSF, Cyclophosphamide (CY) (Plan 2), Rituximab, Busulfan, Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1), Fludarabine (FLU), Methylprednisolone (MP), Donor mobilized PBSC infusion, G-CSF, Cyclophosphamide (CY) (Plan 2), Rituximab, Busulfan
Fanconi Anemia, Severe Aplastic Anemia, Myelodysplastic Syndromes
Clinics and Surgery Center (CSC)
Role of Pharmacotherapy in Counteracting Weight Regain in Adolescents with Severe Obesity
In this study we want to find out more about weight loss and how diet and medications can affect weight loss. This study will last for up to 58 weeks. There are two phases to the study: - A weight loss phase with prescribe meals that lasts 6 weeks. - A study medication/placebo phase that lasts up 52 weeks. You will not know if you are receiving the medication or the placebo.
Aaron Kelly
All
12 Years to 18 Years old
Phase 2
STUDY00008743
STUDY00008743
Inclusion Criteria:
Severe obesity (BMI >/= 120% of the 95th percentile or BMI >/= 35 kg/m2)
Age 12 to < 18 years of age at enrollment (screening) and Tanner stage >/= 2 - Female participants who are sexually active with males and who are able to get pregnant must agree to use two forms of contraception throughout the trial
Exclusion Criteria:
Diabetes (type 1 or 2)
Current or recent (< six months prior to enrollment) use of anti-obesity medication(s) defined as orlistat, phentermine, topiramate, combination phentermine/topiramate, liraglutide, and/or combination naltrexone/bupropion (monotherapy use of naltrexone or bupropion is not an exclusion)
Previous metabolic/bariatric surgery
Current use of a stimulant medication
History of glaucoma
Current or recent (<14 days) use of monoamine oxidase inhibitor
Known hypersensitivity to sympathomimetic amines
Any history of treatment with growth hormone
Any history of bulimia nervosa
Major psychiatric disorder as determined by the local medical monitor
Unstable and clinically-diagnosed (defined as documented in the medical record, if available) depression
Any history of active suicide attempt
History of suicidal ideation or self-harm within the previous 30 days
PHQ-9 score >15
Current pregnancy or plans to become pregnant during study participation
Current tobacco use
ALT or AST >/= 3 times the upper limit of normal
Bicarbonate <18 mmol/L
Creatinine > 1.2 mg/dL
Any history of seizures
Uncontrolled hypertension as determined by the local medical monitor
History of structural heart defect or clinically significant arrhythmia
Diagnosed monogenic obesity
Any history of cholelithiasis
Any history of nephrolithiasis
Clinically diagnosed hyperthyroidism
Untreated thyroid disorder
Any disorder, unwillingness, or inability, not covered by any other exclusion criteria, which in the investigator's opinion, might jeopardize the subject's safety or compliance with the protocol Phentermine-Topiramate, Meal Replacement Therapy, Placebo, Phentermine-Topiramate, Meal Replacement Therapy, Placebo
Obesity
Clinics and Surgery Center (CSC)
Clinical Trial of Two Study Drinks in Detoxification of Environmental Toxicants and Carcinogens
The goal of this research is to determine if consuming one of the two study drinks will help enhance the detoxification of multiple environmental toxicants and cancer causing agents. If our research supports this idea, this drink could be an inexpensive dietary component, which could promote good health.
Dorothy Hatsukami
All
18 Years to old
Phase 2
STUDY00003508
STUDY00003508
Inclusion Criteria:
Adult Male or female. Participants can be smokers or non-smokers
In good physical health
In stable and good mental health
Not using any medications that may affect the Nrf2 pathway
Women who are not pregnant or nursing or planning to become pregnant
Participants have provided written informed consent to participate in the study
Exclusion Criteria:
Significant immune system disorders, respiratory diseases, kidney or liver diseases or any other medical disorders that may affect biomarker data as determined by the licensed medical professional
Vital signs outside of the allotted range
Not willing to abstain from eating cruciferous vegetables during the course of the study Freeze dried Powder, Placebo Preparation, Freeze dried Powder, Placebo Preparation
Healthy
A Randomized Double Blind Phase II Trial of Restorative Microbiota Therapy (RMT) or Placebo in Combination with Durvalumab (MEDI4736) and Tremelimumab With Chemotherapy in Treatment Naive Advanced or Metastatic Adenocarcinoma Non-Small Cell Lung Cancer
This is a randomized, active-controlled, parallel-group, double-blind Phase II trial, of oral restorative microbiota therapy (RMT) or placebo combined with intravenous (IV) durvalumab (MEDI4736) plus tremelimumab and chemotherapy in patients with treatment naïve advanced or metastatic adenocarcinoma non-small cell lung cancer (NSCLC). The primary objectives include: -To evaluate the efficacy of restorative microbiota therapy (RMT) in combination with durvalumab and tremelimumab plus chemotherapy compared with placebo in combination with durvalumab and tremelimumab plus chemotherapy using PFS per RECIST 1.1 as assessed by the investigator -To evaluate the safety and feasibility of restorative microbiota therapy (RMT) in combination with durvalumab and tremelimumab plus chemotherapy in patients with untreated advanced or metastatic adenocarcinoma non-small cell lung cancer (NSCLC)
Amit Kulkarni
All
18 Years to old
Phase 2
STUDY00007800
STUDY00007800
Inclusion Criteria:
Histologically or cytologically confirmed adenocarcinoma of the lung that is unresectable stage IIIB/C or stage IV, does not have an EGFR sensitizing (activating) mutation or ALK or ROS1 translocation. BRAF, RET, NTRK, MET ex 14 splice site mutation
Measurable disease based on RECIST 1.1
Tumor sample requirements
Mandatory provision of an unstained, archived tumor tissue sample in a quantity sufficient to allow for biomarker and genomic analysis. A minimum of 10 unstained slides should be available for evaluation.
Known PD-L1 TC expression status assayed by Ventana SP263. Patients who have known PDL-1 as assayed by PharmDx 22C3 assay may be eligible; however, available archival tissue will be used to assay with Ventana SP263 test.
Prior chemotherapy or immunotherapy as adjuvant therapy for lung cancer is permitted as long as it has been >6 months from last dose at the time of enrollment. Local treatment of isolated lesions, excluding target lesions, for palliative intent is acceptable (e.g. by local surgery or radiotherapy). Prior systemic therapy for advanced/metastatic NSCLC makes the patient ineligible for this study.
Patients with treated brain metastasis are eligible as long as they have stable symptoms, are more than 2 weeks from completion of therapy, and do not require more than 10mg of daily prednisone or equivalent.
ECOG Performance status of 0 or 1
Body weight of >30 kg
Adequate organ function within 14 days of study enrollment defined as:
Hemoglobin ≥ 9.0 g/dL
Absolute neutrophil count ≥1,500/mcL
Platelets ≥ 100,000/mcL
Total bilirubin ≤1.5x upper limit of normal (ULN) - this will not apply to patients with confirmed Gilbert's syndrome (persistent or recurrent hyperbilirubinemia that is predominantly unconjugated in the absence of hemolysis or hepatic pathology), who will be allowed only in consultation with their physician.
AST (SGOT) and ALT (SGPT) ≤2.5 x institutional ULN unless liver metastases are present, in which case it must be ≤5x ULN
Measured creatinine clearance (CL) >40 mL/min or calculated creatinine CL>40 mL/min by the Cockcroft-Gault formula (Cockcroft and Gault 1976) or by 24-hour urine collection for determination of creatinine clearance:
Expected life expectancy of at least 12 weeks in the opinion of the enrolling investigator as documented in the medical record
Women of childbearing potential and men with partners of child-bearing potential must agree to use effective contraception for the time of screening to the duration of treatment and 3 months after the last dose of study drug
Provide voluntary written consent prior to the performance of any research related tests or procedures.
Exclusion Criteria:
Not pregnant or breast feeding. Evidence of post-menopausal status, or negative urine or serum pregnancy test for female pre-menopausal patients. Women will be considered postmenopausal if they have amenorrhea for 12 months without an alternative medical explanation
Dysphagia or inability to swallow medications
Squamous cell, large cell, or NSCLC NOS (not otherwise specified) histology or mixed tumors
Has untreated brain metastasis or active leptomeningeal carcinomatosis
Has a known sensitivity to any component of therapeutic agents used in this study
Receipt of any immunotherapy or investigational drug within 4 weeks prior to the first dose of study drug; and in the case of monoclonal antibodies 6 weeks prior to the first dose of study drug
Prior treatment with any other anti-PD-1, or PD-L1, including durvalumab or an anti-PD-L2 agent or an antibody or a small molecule targeting other immuno-regulatory receptors except as adjuvant therapy for NSCLC so long as it has been greater than six months since the last treatment
Current or prior use of immunosuppressive medication within 28 days before the first dose of study drug, with the exceptions of intranasal and inhaled corticosteroids or systemic corticosteroids at physiological doses, which are not to exceed 10 mg/day of prednisone, or an equivalent corticosteroid
Recent major surgery within 4 weeks prior to entry into the study (excluding the placement of vascular access) that would prevent administration of study drug
Active or prior documented autoimmune disease within the past 2 years requiring systemic treatment (i.e. with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Patients with vitiligo, Grave's disease, or psoriasis not requiring systemic treatment (within the past 2 years) are not excluded. Replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment.
Active documented inflammatory bowel disease (eg, Crohn's disease, ulcerative colitis) who require immunosuppression or patients who exceed 10 mg/day of prednisone, or an equivalent corticosteroid are excluded
History of primary immunodeficiency
History of organ transplant that requires therapeutic immunosuppression
Taking daily probiotics (patients with last probiotic > 4weeks prior to first dose of RMT are eligible)
History of Human Immunodeficiency Virus (HIV) (known HIV 1/2 antibodies positive) who are on anti-retroviral treatment for < 6 months and absolute CD4 count<500 (patients with HIV not meeting these criteria are eligible)
Has known active Hepatitis B or C. Active Hepatitis B is defined as a known positive HBsAg, and positive hepatitis B virus quantification assay (patients with history of Hepatitis B who have seroconversion i.e. Hepatitis B core antibody positive and Hepatitis B surface antibody positive are eligible). Active Hepatitis C is defined by a known positive Hep C Ab result and positive quantitative HCV RNA results (Patients with Hepatitis C who are on anti-viral suppressive therapy and negative quantitative HCV RNA results are eligible)
Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, uncontrolled hypertension, unstable angina pectoris, cardiac arrhythmia, active peptic ulcer disease or gastritis, active bleeding diatheses
Myocardial infarction or stroke within 3 months prior to enrollment
History of systolic or diastolic heart failure with New York Heart Association (NYHA) class III or IV symptoms (refer to Appendix II)
Has active or prior history of (non-infectious) pneumonitis that required steroids or patients with interstitial lung disease
Psychiatric illness/social situations that would limit compliance with study requirements or compromise the ability of the patient to give written informed consent
Known history of active tuberculosis. Patients with prior history of latent TB could be included if they have been treated previously with isoniazid.
Patients who are on chronic systemic antibiotic therapy (antibiotics for ≥60 consecutive days within 12 weeks of enrollment). Patients who receive systemic antibiotics between enrollment and start of RMT are eligible
Receipt of live attenuated vaccination within 30 days prior to study entry or within 30 days of receiving RMT
History of another primary malignancy (excluding non-melanoma skin cancer) within 5 years prior to starting RMT, except if the patient has undergone potentially curative therapy with no evidence of disease recurrence for 5 years since initiation of that therapy
Any condition that, in the opinion of the investigator, would interfere with evaluation of the study drug or interpretation of patient safety or study results Oral Restorative Microbiota Therapy (RMT) Capsules, Durvalumab 1500 mg IV, Cisplatin/pemetrexed or Carboplatin/pemetrexed, Placebo, Oral Restorative Microbiota Therapy (RMT) Capsules, Durvalumab 1500 mg IV, Cisplatin/pemetrexed or Carboplatin/pemetrexed, Placebo
Adenocarcinoma of Lung, Lung Cancer
Clinics and Surgery Center (CSC)
Prefrontal Cortical Stimulation in Severe Treatment Resistant Depression
This study looks at the use of an implanted brain stimulator for people who have treatment resistant depression. The change in brain function by EEG and symptoms of depression will be examined. This study is open to people 22-55 years old with Medicare or Medicare Advantage insurance.
Ziad Nahas
All
22 Years to 55 Years old
N/A
STUDY00006945
STUDY00006945
Inclusion Criteria:
Participant must be able to provide written informed consent.
Participant has a diagnosis of chronic (greater than or equal to 2 years) depressive episode as defined by DSM V criteria
Participant has not had an adequate response to three or more adequate antidepressant treatments from at least two different antidepressant treatment categories in the current depressive episode according to the Antidepressant Treatment History Form (ATHF)
Participant must have had ECT (Electroconvulsive Therapy) or refused to undergo ECT if clinically indicated to them
Participant must have HRSD greater than or equal to 20
Participant must be able to complete the evaluations needed for this study including the functional imaging scans and the EEG Bayesian optimization sessions
Participant must be under the care of a licensed psychiatrist, undergoing regular care evaluations, and inform study team of any change to care team during study participation
Participant must agree to allow all forms of communication between investigators and study staff and any health care provider (current or having provided service within two years of enrollment)
Participant must provide name and contact information for at least two people greater than or equal to 22 in age who reside within a 30 minute drive of the participant's residents and whom staff may contact as necessary during study participation
Participant must be enrolled in a Medicare program
Exclusion Criteria:
The PCS would (in the investigator's judgment) pose an unacceptable surgical or medical risk for the participant
Participant is unable to undergo required full body magnetic resonance imaging (MRI) during the clinical study
Participant is judged by the investigator to be acutely suicidal (e.g. within the 30 days prior to the PCS implant the participant has made a suicide attempt or gesture or has made specific plans or preparation to commit suicide or scores 21 or higher on the MSSI)
In addition to the acute suicidal risks mentioned above, participant meets any of the following:
Has made a suicide attempt within the previous 12 months that required medical treatment
Has made greater than or equal to two suicide attempts in the past 12 months
Has a clear-cut plan for suicide that states that she/he cannot guarantee that she/he will call her/his regular psychiatrist or the Investigator if the impulse to implement the plan becomes substantial during the study
Is likely to attempt suicide within the next six months, in the Investigator's opinion
Participant has a history of schizophrenia, schizoaffective disorder, or other psychotic disorder, or a current major depressive episode that includes psychotic features (commonly referred to as psychotic depression) according to the DSM V criteria
Participant with a diagnosis of dementia with a Mini-Mental State Exam (MMSE) less than or equal to 23
Participant with a positive urine pregnancy test
Participant with a positive urine drug screen
Participant with DBS (Deep Brain Stimulator)
Participant with VNS (Vagus Nerve Stimulator) if the device was active in the last 6 months prior to study enrollment
Participant with history of seizures Prefrontal Cortical Stimulation (PCS), Prefrontal Cortical Stimulation (PCS)
Treatment Resistant Depression
Clinics and Surgery Center (CSC)
Efficacy of Belimumab and Rituximab Compared to Rituximab Alone for the Treatment of Primary Membranous Nephropathy (REBOOT)
This trial is a two-part study (Part A and Part B) of adults with primary membranous nephropathy, ages 18-75 inclusive. Part A is an open-label, PK phase to compare belimumab exposure between participants who have “low” proteinuria (≥ 4 to < 8 g/day) and “high” proteinuria (≥ 8 g/day) at Visit -1. Part B is a prospective, randomized, phase II, double-blind, placebo-controlled, multicenter clinical trial in adults with primary MN. Part B will commence after the analysis of the PK data in Part A.
Patrick Nachman
All
18 Years to 75 Years old
Phase 2
STUDY00006831
STUDY00006831
Inclusion Criteria:
Subjects must meet all of the following criteria to be eligible for this study-
Diagnosis of one of the following:
Primary membranous nephropathy (MN):
Confirmed by kidney biopsy obtained in the past 5 years, or
If relapsing following a complete remission or partial remission, confirmed with a kidney biopsy obtained in the past 7 years
Nephrotic syndrome, and a contraindication to kidney biopsy (e.g., anti-coagulation, solitary kidney, body habitus that increases the risk of biopsy, or other contraindication in the opinion of the investigator).
Serum anti-PLA2R positive;
Estimated Glomerular Filtration Rate (eGFR) ≥ 30 mL/min/1.73m^2 while on maximally tolerated renin-angiotensin system (RAS) blockade;
Proteinuria:
≥4 and < 8 g/day that has been present for ≥ 3 months while on while on maximally tolerated RAS blockade, or
≥8 g/day while on maximally tolerated RAS blockade.
Blood pressure while on maximally tolerated RAS blockade:
Systolic blood pressure ≤ 140 mmHg, and
Diastolic blood pressure ≤ 90 mmHg
Exclusion Criteria:
Subjects meeting any of the following criteria will not be eligible for this study-
Secondary cause of membranous nephropathy (MN) (e.g., systemic lupus erythematosus (SLE), drug, infection, malignancy) suggested by review of the subject's medical history and/or clinical presentation;
Rituximab use within the previous 12 months;
Rituximab use > 12 months ago:
With an undetectable CD19 B cell count, or
Did not result in a complete remission (CR) or partial remission (PR) with rituximab treatment alone (e.g., without other immunosuppressive or immunomodulatory therapy).
Use of anti-B cell therapy other than rituximab within the previous 12 months (or 5 half-lives, whichever is greater);
Cyclophosphamide use within the past 3 months;
Use of other immunosuppressive medications, such as cyclosporine or tacrolimus, within the past 30 days;
Use of systemic corticosteroids within the past 30 days;
Use of any biologic investigational agent, defined as any drug not approved for sale in the country it is used, in the previous 12 months;
Use of any non-biologic investigational agent in the past 30 days (or 5 half-lives, which ever is greater);
Poorly controlled diabetes mellitus defined as hemoglobin A1c (HbA1c) ≥ 9.0%
Patients with diabetic glomerulopathy on renal biopsy that is:
Greater than Class I diabetic glomerulopathy, or
Class I diabetic glomerulopathy with a history of poor diabetic control (e.g., HbA1c ≥ 9.0%) since time of biopsy;
Unstable kidney function defined as > 15% decrease in the Estimated Glomerular Filtration Rate (eGFR) during the previous 3 months;
Decrease in proteinuria by 50% or more during the previous 12 months;
White blood cell (WBC) count < 3.0 x 10^3/µl;
Absolute neutrophil count < 1.5 x 10^3/µl;
Moderately severe anemia (hemoglobin <9mg/dL);
History of primary immunodeficiency;
Serum immunoglobulin A (IgA) < 10 mg/dL;
Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) = ≥2 times the upper limit of normal (ULN);
Positive human immunodeficiency virus (HIV) serology;
Positive hepatitis C virus (HCV) serology, unless treated with anti-viral therapy with achievement of a sustained virologic response (undetectable viral load 24 weeks after cessation of therapy);
Evidence of current or prior infection with hepatitis B, as indicated by a positive HBsAg, positive HBcAb, or positive HBsAb serology without history of vaccination;
Positive QuantiFERON - tuberculosis (TB) Gold test results,
--Note: Tuberculin Purified Protein Derivative (PPD) test may be substituted for QuantiFERON - TB Gold test.
History of lung disease with FVC < 70% predicted, DLCO < 70% predicted, or requiring supplemental oxygen;
History of malignant neoplasm within the last 5 years,
--Exception: basal cell or squamous cell carcinoma of the skin treated with local resection only, or carcinoma in situ of the uterine cervix treated locally and with no evidence of metastatic disease for 3 years.
Absence of individualized, age-appropriate cancer screening;
Women of child-bearing potential who are pregnant, nursing, or unwilling to be sexually inactive or use FDA-approved contraception until study week 104;
Acute or chronic infection, including:
current use of suppressive therapy for chronic infection,
hospitalization for treatment of infection in the past 60 days, or
parenteral anti-microbial (including anti-bacterial, anti-viral, or anti-fungal agents) use in the past 60 days for infection.
History of anaphylactic reaction to parenteral administration of contrast agents, human or murine proteins or monoclonal antibodies, including:
rituximab, or
belimumab.
Evidence of serious suicide risk, including:
any history of suicidal behavior in the last 6 months,
any suicidal ideation in the last 2 months, or
who, in the investigator's judgment, pose a significant suicide risk.
Evidence of current drug or alcohol abuse or dependence, or a history of drug or alcohol abuse or dependence in the past 12 months;
Vaccination with a live vaccine within the past 30 days;
Other diseases or conditions which, in the opinion of the investigator, would put the subject at risk or confound the results of the study; or
Inability to comply with study and follow-up procedures. Belimumab, Placebo for Belimumab, Rituximab, Belimumab, Placebo for Belimumab, Rituximab
Membranous Nephropathy, Nephrotic Syndrome
Clinics and Surgery Center (CSC)
A Phase II Study of Pembrolizumab Monotherapy in Recurrent Ovarian Cancer of the Immunoreactive Subtype determined by NanoString Gene Expression Profiling
To evaluate clinical anti-tumor activity of pembrolizumab monotherapy based on ORR as assessed by the investigator per irRECIST in patients with ROC whose tumors show an immunoreactive gene expression signature
Boris Winterhoff
Female
18 Years to old
Phase 2
STUDY00006054
STUDY00006054
Inclusion Criteria:
Be willing and able to provide written informed consent/assent for the trial
Have received 1-5 prior lines for treating ROC (i.e. 2-6 total prior lines counting the front line) and must have a platinum-free interval (PFI) or a treatment-free interval (TFI) >= 3 months based on the last regimen received
Have measurable disease based on Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
Note: Tumor lesions situated in a previously irradiated area are considered measurable if progression has been demonstrated in such lesions
Have a performance status of 0 or 1 on the Eastern Cooperative Oncology Group (ECOG) performance scale
Have histologically diagnosed recurrent epithelial ovarian, fallopian or primary peritoneal ovarian cancer
Have provided a tumor tissue sample either collected from a newly obtained tumor tissue biopsy or an archival tissue specimen. Subjects for whom newly obtained samples cannot be provided (e.g. inaccessible or subject safety concern) may submit an archived biopsy specimen. Formalin-fixed paraffin embedded (FFPE) block specimens are preferred to 20 unstained slides. Additional samples may be requested if tumor tissue provided is not adequate for quality and/or quantity as assessed by the central laboratory
Performed within 10 days of treatment initiation: absolute neutrophil count (ANC) >= 1,500/mcL
Performed within 10 days of treatment initiation: platelets >= 100,000/mcL
Performed within 10 days of treatment initiation: hemoglobin >= 9 g/dL or >= 5.6 mmol/L without transfusion or erythropoietin (EPO) dependency (within 7 days of assessment)
Performed within 10 days of treatment initiation: serum creatinine OR measured or calculated creatinine clearance (CrCl) (glomerular filtration rate [GFR] can also be used in place of creatinine or CrCl) =< 1.5 x upper limit of normal (ULN) OR >= 45 mL/min for subject with creatinine levels > 1.5 x institutional ULN
Creatinine clearance should be calculated per institutional standard
Performed within 10 days of treatment initiation: serum total bilirubin =<1.5 x ULN OR direct bilirubin =< ULN for subjects with total bilirubin levels > 1.5 ULN
Performed within 10 days of treatment initiation: aspartate aminotransferase (AST) (serum glutamate pyruvate transaminase [SGOT]) and aspartate aminotransferase (ALT) (serum glutamic-oxaloacetic transaminase [SGPT]) =< 2.5 x ULN OR =< 5 x ULN for subjects with liver metastases
Female subject of childbearing potential should have a negative urine or serum pregnancy within 72 hours prior to receiving the first dose of study medication. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required
Have received front line platinum-based chemotherapy (preoperative chemotherapy is allowed)
Female subjects of childbearing potential must be willing to use an adequate method of contraception for the course of the study through 120 days after the last dose of study medication
Note: Abstinence is acceptable if this is the usual lifestyle and preferred contraception for the subject
Exclusion Criteria:
Is currently participating and receiving study therapy or has participated in a study of an investigational agent and received study therapy or used an investigational device within 2 weeks of the first dose of treatment
Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to the first dose of trial treatment
Has a known history of active TB (Bacillus Tuberculosis)
Hypersensitivity to pembrolizumab or any of its excipients
Has had a prior anti-cancer monoclonal antibody (mAb) within 4 weeks prior to study day 1 or who has not recovered (i.e., =< grade 1 or at baseline) from adverse events due to agents administered more than 4 weeks earlier
Has had prior chemotherapy, targeted small molecule therapy, or radiation therapy within 2 weeks prior to study day 1 or who has not recovered (i.e., =< grade 1 or at baseline) from adverse events due to a previously administered agent
Note: Subjects with =< grade 2 neuropathy are an exception to this criterion and may qualify for the study
Note: If subject received major surgery, they must have recovered adequately from the toxicity and/or complications from the intervention prior to starting therapy
Has a known additional malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer
Has known active central nervous system (CNS) metastases and/or carcinomatous meningitis. Subjects with previously treated brain metastases may participate provided they are stable (without evidence of progression by imaging for at least four weeks prior to the first dose of trial treatment and any neurologic symptoms have returned to baseline), have no evidence of new or enlarging brain metastases, and are not using steroids for at least 7 days prior to trial treatment. This exception does not include carcinomatous meningitis which is excluded regardless of clinical stability
Has active autoimmune disease that has required systemic treatment in the past 2 years (i.e. with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (eg., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment
Has known history of, or any evidence of active, non-infectious pneumonitis
Has an active infection requiring systemic therapy
Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the subject?s participation for the full duration of the trial, or is not in the best interest of the subject to participate, in the opinion of the treating investigator
Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial
Is pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the trial, starting with the pre-screening or screening visit through 120 days after the last dose of trial treatment
Has received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 agent
Has a known history of human immunodeficiency virus (HIV) (HIV 1/2 antibodies)
Has known active hepatitis B (e.g., hepatitis B surface antigen [HBsAg] reactive) or hepatitis C (e.g., hepatitis C virus [HCV] ribonucleic acid [RNA] [qualitative] is detected)
Has received a live vaccine within 30 days of planned start of study therapy
Note: Seasonal influenza vaccines for injection are generally inactivated flu vaccines and are allowed; however intranasal influenza vaccines (e.g., Flu-Mist) are live attenuated vaccines, and are not allowed Pembrolizumab, Pembrolizumab
Recurrent Fallopian Tube Carcinoma, Recurrent Ovarian Carcinoma, Recurrent Primary Peritoneal Carcinoma
Clinics and Surgery Center (CSC)
Smart Use of Medication for the Treatment of Adolescent Severe Obesity (SMART)
We are studying the best time to add weight loss medication to diet and exercise for helping adolescents who carry extra weight. All participants start with a lifestyle modification program and some participants may also receive study medication. Participants must be 12-17 years of age and carry extra weight. The program will last for 48 weeks.
Claudia Fox
Male or Female
up to 18 Years old
Phase 2
STUDY00006824
STUDY00006824
Inclusion Criteria:
Provision of signed and dated informed assent form;
Provision of signed and dated informed parental consent form from at least 1 legal parent/guardian;
Stated willingness to comply with all study procedures and availability for the duration of the study;
BMI >/= 1.2 times the 95th percentile or BMI >/= 35 Kg/m2, whichever is lower;
Tanner stage >/= 2;
Male or female, aged 12-17 at time of consenting;
For females of reproductive potential: when sexually active, agreement to use highly effective contraception (oral contraceptive pill, intra-uterine device (IUD), or implant) during study participation;
For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner.
Exclusion Criteria:
Contraindications to phentermine or topiramate use according to package inserts, including: history of glaucoma; current or recent (< 14 days) use of monoamine oxidase inhibitor; known hypersensitivity to sympathomimetic amines; current pregnancy, plans to become pregnant, or if sexually active refusal to use 2 forms of birth control; history of cardiac disease including coronary artery disease; clinically significant cardiac arrhythmias; heart failure or uncontrolled hypertension;
Diabetes (type 1 or 2);
Presence of cardiac pacemaker;
Current or recent (<6 months prior to enrollment) use of weight loss medication(s);
Current use of weight-altering medication(s) (e.g., atypical antipsychotic, metformin) unless dose has been stable for past 6 months;
Current use of other sympathomimetic amine such as attention-deficit hyperactivity disorder (ADHD) stimulants;
Seizure disorder (other than infantile febrile seizure);
Previous bariatric surgery;
Recent initiation of change in dose (< 3 months prior to enrollment) of anti-hypertensive or lipid medication(s);
Tobacco use
History of or current diagnosis of schizophrenia, psychosis, mania, chemical dependency;
Unstable depression or anxiety that has required hospitalization in the past year;
Any history of suicide attempt;
Suicidal ideation or self-harm within 12 months prior to enrollment;
Bicarbonate < 18 mmol/L;
Creatinine > 1.2 mg/dL;
History of cholelithiasis;
History of nephrolithiasis;
Untreated thyroid disorder;
Hyperthyroidism;
Breastfeeding Lifestyle Modification Therapy (LSMT), Phentermine Pill, Topiramate Pill, Lifestyle Modification Therapy (LSMT), Phentermine Pill, Topiramate Pill
Children's Health, Diabetes & Endocrine
Weight management, Clinics and Surgery Center (CSC)
Real-Time Feedback (RTFB) to Improve Colonoscopy
To test whether real-time feedback during the withdrawal phase of colonoscopy improves quality of colonoscopy and the adenoma detection rate.
Piet de Groen
All
18 Years and over
N/A
STUDY00007271
STUDY00007271
Inclusion Criteria:
• Any endoscopist willing to parcipate and performing routine colonscopy
Exclusion Criteria:
• No exclusion criteria
AI program for colonoscopy, AI program for colonoscopy
Digestive & Liver Health
Clinics and Surgery Center (CSC)
MT2019-41: A Phase 2 Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects with Fanconi Anemia Subtype A
The objective of this study is to assess the therapeutic efficacy of a hematopoietic gene therapy consisting of autologous CD34+ enriched cells transduced with a LV carrying the FANCA gene in subjects with FA-A.
Margaret MacMillan, MD
All
1 Year to old
Phase 2
STUDY00008719
STUDY00008719
Inclusion Criteria:
Fanconi anemia as diagnosed by chromosomal fragility assay of cultured lymphocytes in the presence of DEB or a similar DNA-crosslinking agent
Patients of the complementation group FA-A
Minimum age: 1 year and a minimum weight of 8 kg
At least 30 CD34+ cells/μL are determined in one bone marrow (BM) aspiration within 3 months prior to CD34+ cell collection OR (see subsequent criterion)
If the number of CD34+ cells/ μL in BM is in the range of 10-29, peripheral blood (PB) parameters should meet two of the three following criteria:
Hemoglobin: ≥11g/dL
Neutrophils: ≥900 cells/μL
Platelets: ≥60,000 cells/μL
Provide informed consent in accordance with current legislation
Women of childbearing age must have a negative urine pregnancy test at the baseline visit, and accept the use of an effective contraception method during participation in the trial
Exclusion Criteria:
Subjects with an available and medically eligible HLA-identical sibling donor.
Evidence of myelodysplastic syndrome or leukemia, or cytogenetic abnormalities other than those reported as variant(s) of normal in BM aspirate analysis. This assessment should be made by valid studies conducted within the 3 months before the subject commences the stem cell mobilization/collection procedures of the clinical trial.
Subjects with somatic mosaicism associated with stable or improved counts in all PB cell lineages. (If T-lymphocyte chromosomal fragility analysis indicates potential mosaicism, a medically significant decrease (≥1 NCI CTCAE grade) in at least one blood lineage over time must be documented to enable eligibility, as should <5% resistance of bone marrow colony forming cells (CFCs) to 10nM MMC; whenever possible potential mosaicism should also be evaluated by gene sequencing of MMC-resistant CFCs).
Lansky performance status ≤60%.
Any concomitant disease or condition that, in the opinion of the Principal Investigator, renders the subject unfit to participate in the study.
Pre-existing sensory or motor impairment ≥grade 2 according to the criteria of the NCI.
Pregnant or breastfeeding women.
Hepatic dysfunction as defined by either:
Bilirubin >3.0 × the upper limit of normal (ULN) or
Alanine aminotransferase (ALT) > 5.0 × ULN or
Aspartate aminotransferase (AST) > 5.0 × ULN
For subjects with bilirubin, ALT or AST above ULN, a workup to identify the etiology of liver abnormality should be conducted prior to confirmation of eligibility as stipulated in exclusion criterion 5, including evaluation of viral hepatitis, iron overload, drug injury or other causes.
Renal dysfunction requiring either hemodialysis or peritoneal dialysis.
Pulmonary dysfunction as defined by either:
Need for supplemental oxygen during the prior 2 weeks in absence of acute infection or
Oxygen saturation by pulse oximetry <90%.
Evidence of active metastatic or locoregionally advanced malignancy for which survival is anticipated to be less than 3 years.
Subject is receiving androgens (i.e. danazol, oxymetholone).
Subject is receiving other investigational therapy for treatment/prevention of FA-associated bone marrow failure. RP-L102, RP-L102
Fanconi Anemia Complementation Group A
anemia, bone marrow failure, gene therapy
A Phase II, Open Label, Two Arm Study of Therapeutic Iobenguane (131I) as Single Agent or in Combination with Vorinostat for Recurrent or Progressive High-Risk Neuroblastoma Subjects (OPTIMUM Trial) Protocol Number: MIBG 2014-01 (OPTIMUM)
This will be a Phase II, two-arm, nonrandomized, non-comparative, open-label study in participants ≥ 1 year of age with iobenguane avid, recurrent or progressive high-risk neuroblastoma. Participants not eligible for vorinostat treatment may receive 131I-MIBG as monotherapy.
Emily Greengard
All
1 Year to old
Phase 2
STUDY00005792
STUDY00005792
Inclusion Criteria:
Subjects with a diagnosis of iobenguane avid, high-risk neuroblastoma based on Revised INRC criteria at the time of study enrollment with recurrent or progressive disease at any time prior to enrollment, regardless of overall response to frontline therapy, where frontline therapy includes a minimum of 4 cycles of induction therapy at any time prior to enrollment.
May have had prior 131I-MIBG therapy, provided:
It has been at least 6 months from the date of last 131I-MIBG ;
Response was other than progressive disease on first restaging after 131I-MIBG ;
Prior 131I-MIBG was given as monotherapy and not in combination with systemic anticancer agents;
Cumulative lifetime dose of 131I-MIBG at enrollment does not exceed 18 mCi/kg.
All soft tissue lesions identified on CT/MRI scans must be iobenguane avid lesions on an (123I)-iobenguane scan, or
any progressive non-iobenguane avid lesion is proven by biopsy to be a non-neuroblastoma lesion.
any other non-avid lesion is comprised of a fibrotic or scarred mass as shown by routine imaging and confirmed by the investigator.
Adequate cryopreserved autologous peripheral blood stem cells or bone marrow (at least 2 aliquots of 2.0 × 10exp6 CD34/kg at the time of study enrollment).
If a male, must agree to use an adequate contraception method as deemed appropriate by the Investigator (e.g., vasectomy, condoms) or partner using effective contraception and to not donate sperm during the study and for 90 days after receiving the last dose of study drug.
If a female of childbearing potential, have a negative serum pregnancy test result prior to each dosing and, if sexually active, be practicing an effective method of birth control [e.g., intrauterine device, double-barrier method (i.e., diaphragm, or a cervical cap) with intravaginal spermicidal foam, cream or gel], or male partner sterilization throughout the study.
Age at study entry ≥1 year.
Previous platelet transfusions are permitted, as long as the subject has a platelet count ≥50,000/μL without transfusion support for at least 1 week.
Subjects must have a minimum pulse oximetry measurement of at least 94% at baseline.
An absolute neutrophil count ≥750/μL without growth factor for 5 days.
Liver function parameter results: total bilirubin ≤2 × upper limit of normal for age, and Serum alanine aminotransferase (glutamic-pyruvic transaminase) and serum aspartate aminotransferase (glutamic-oxaloacetic transaminase) ≤ 10 times the upper limit of normal (for all sites, the upper limit of normal for alanine aminotransferase is defined as 45 U/L).
Normal thyroid function as measured by T4 or TSH or have abnormal results that are not considered clinically important by the Investigator or may be receiving levothyroxine.
Cardiac Function: shortening fraction of ≥ 27% by echocardiogram or ejection fraction ≥ 50% documented by echocardiogram or radionuclide angiogram within 1 month prior to Visit 1 (Baseline).
Karnofsky Performance Status (for subjects >16 years of age) or the Lansky Performance Status Performance Status (for subjects 1 to 16 years of age) ≥50%.
Full recovery from the toxic effects of any prior therapy.
Coagulation Function:
International Normalized Ratio (INR) < 1.5
Partial thromboplastin time (PTT) < 1.5 times upper limit of normal.
Exclusion Criteria:
Subjects within 5 half-lives after any antibody-based immunotherapy, or have not recovered from effects of any biologic therapy.
Subjects <12 weeks after myeloablative therapy with autologous stem cell transplant.
Subjects who have had an allogeneic stem cell treatment less than 4 months from Visit 1 are excluded. Those who have received allogeneic stem cell treatment more than 4 months from Visit 1 must have recovered and have no active graft versus host disease (GVHD) to be eligible.
Subjects must not have received radiation for a minimum of 2 weeks prior to study enrollment. Subjects whose only site(s) of disease have been radiated are eligible as long as the subject has MIBG avidity 2 weeks after completion of radiation. A minimum of 12 weeks prior to study enrollment is required following prior large field radiation therapy (ie, craniospinal, whole abdominal, total lung, > 50% marrow space)
History of total body irradiation.
Subjects do not have adequate renal function defined as GFR ≥ 70 mL/min/1.73 m2 either by creatinine clearance or radioisotope direct measurement or by calculation with the Schwartz formula
Subjects who are on hemodialysis.
Pregnancy or breastfeeding.
Significant active infections including active hepatitis B, or hepatitis C infection, or known infection with human immunodeficiency virus (HIV) (testing for HIV is not required prior to study entry).
Clinically important cardiac, pulmonary, and hepatic impairment.
Vorinostat treatment exclusion criteria (subjects, who meet any one of these criteria and otherwise meet eligibility criteria, are still eligible for 131I-MIBG monotherapy)
Since valproic acid has HDAC inhibitory activity, patients must not have received valproic acid within 30 days of study entry.
Since vorinostat may prolong the QT interval, patients must not be receiving other medications known to prolong the QT interval at the time of study entry . Pentamidine must not have been received within 1 week of study enrollment.
Patients with a history of deep venous thrombosis that was not associated with the presence of a central venous catheter.
Patients who are receiving Coumadin. 131I-MIBG, 131-MIBG + Vorinostat, 131I-MIBG, 131-MIBG + Vorinostat
Neuroblastoma, Neuroectodermal Tumors, Neoplasms
Iobenguane Avid High-risk Neuroblastoma, 3-Iodobenzylguanidine, Radiopharmaceutical
A Multicenter, 6-Month, Randomized, Open-Label, Active Control, Parallel Arm, Phase 2b Study of Daily Oral LUM-201 in Naive-to-Treatment, Prepubertal Children with Growth Hormone Deficiency (GHD) (OraGrowtH210)
This research study is studying LUM-201 as a possible treatment for Growth Hormone Deficiency (GHD) in pre-pubertal (before puberty) children. Lumos Pharma is sponsoring this research study. Your child is being asked to be in this study because she or he has been diagnosed to have growth hormone deficiency; and your child’s study doctor thinks that your child might be a good candidate for this study. Growth hormone deficiency can result in growth failure. One of the most visible signs of growth failure is a height that is much shorter than most other children of the same age. This is called short stature. However, some children can have growth failure even if they do not have short stature. The standard treatment for growth hormone deficiency is daily injections under the skin of recombinant human growth hormone (rhGH). This study seeks to see if oral LUM-201 at various doses may achieve similar catch-up growth compared to rhGH and provide a safe and effective oral treatment alternative to daily injections. LUM-201 is to be taken by mouth and it is thought that it can increase the body’s ability to release growth hormone.
Brad Miller, MD, PhD
All
3 Years to 12 Years old
Phase II
STUDY00011599
STUDY00011599
Inclusion Criteria:
Have an established diagnosis of idiopathic PGHD as determined by standard diagnostic criteria. Eligible subjects must be naïve-to-treatment and be prepubertal.
Morning cortisol ≥ 7 µg/dL or stimulated cortisol ≥ 14 µg/dL.
At Screening, be ≥ 3.0 years and ≤ 11.0 years for girls and ≤ 12.0 years for boys.
Have HT-SDS ≤ -2.0 or HT-SDS ≥ 2 SD below mean parental HT-SDS.
Have a baseline height velocity < 5.5 cm/year based on at least 6 months of growth.
Have a bone age delayed by ≥ 6 months with respect to chronological age.
Have prepubertal status as evidenced by Tanner Stage I breast development in girls and testicular volume < 4.0 mL in boys.
In girls, have genetic testing results to rule out Turner syndrome. If SHOX genetic testing results are available, they need to be negative.
Have normal thyroid function. Subjects diagnosed with hypothyroidism must have documented successful treatment for at least 30 days prior to Day 1.
Exclusion Criteria:
Any medical or genetic condition which, in the opinion of the Investigator or Medical Monitor (MM), can be an independent cause of short stature and/or limit the response to exogenous growth factor treatment. (Examples: diabetes, idiopathic short stature).
A medical or genetic condition that, in the opinion of the Investigator and/or MM, adds unwarranted risk to use of LUM-201 or rhGH.
Use of any medication that, in the opinion of the Investigator and/or MM, can independently cause short stature or limit the response to exogenous growth factors (Example: glucocorticoids).
Evidence or history of an intracranial mass (e.g., pituitary tumor, craniopharyngioma).
Suspicion of absent pituitary function as evidenced by a maximal stimulated GH ≤ 3 ng/mL on two prior standard of care GH stimulation tests, or pituitary deficiencies beyond GH and thyroid function.
Malnutrition as evidenced by medical history or a body weight < 3rdth percentile for current height.
BMI > 95th percentile.
Gestational age-adjusted birth weight < 5th percentile (small for gestational age).
History of spinal, cranial, or total body irradiation.
Treatment with medications known to act as moderate or strong inhibitors or strong inducers of CYP3A/4, or with medications known to act as strong inhibitors of P-glycoprotein (P-gp) or potent substrates of P-gp or Multidrug and toxin extrusion protein 1 (MATE1). LUM-201, rhGH Norditropin® pen (34 µg/kg), LUM-201, rhGH Norditropin® pen (34 µg/kg)
Growth Hormone Deficiency
Catch-up growth, GHD, Growth hormone secretagogue, Height, LUM-201, Oral, PEM, PGHD, Predictive Enrichment Marker
A Phase 2, Open-Label, Basket Study of Atrasentan in Patients with Proteinuric Glomerular Diseases (AFFINITY)
The purpose of the research is to find out if atrasentan delays worsening of kidney function in IgAN, FSGS, and Alport Syndrome
Michelle Rheault
Male or Female
Not specified
Phase 2
STUDY00012146
STUDY00012146
Inclusion Criteria:
Age 18 years and older for patients in the IgAN, FSGS, and Alport Syndrome cohorts
Age 18-70 years for patients in the DKD cohort
Receiving a maximally tolerated dose of RAS inhibitor therapy (ACEi or ARB) that has been stable for at least 12 weeks.
For patients enrolling in IgAN Cohort:
Biopsy-proven IgA nephropathy
UPCR between 0.5 to less than 1.0 g/g
Screening eGFR ≥ 30 mL/min/1.73 m2
For patients enrolling in FSGS Cohort:
Biopsy-proven FSGS or documented genetic mutation in a podocyte protein associated with FSGS
UPCR > 1.0 g/g
Screening eGFR ≥ 30 mL/min/1.73 m2
Subjects receiving systemic corticosteroids or other immunosuppressants must be on a stable dose for at least 12 weeks.
BMI ≤ 40 kg/m2
For patients enrolling in Alport syndrome Cohort:
Diagnosis of Alport syndrome by genetic testing
UPCR > 0.5 g/g
Screening eGFR ≥ 30 mL/min/1.73 m2
For patients enrolling in DKD Cohort:
Diagnosis of type 2 diabetes mellitus
UACR ≥ 0.5 g/g
Screening eGFR ≥ 45 mL/min/1.73 m2
Receiving a stable dose of SGLT2 inhibitor for at least 12 weeks
Willing and able to provide informed consent and comply with all study requirements
Exclusion Criteria:
Current diagnosis of another cause of chronic kidney disease or another primary glomerulopathy.
History of kidney transplantation or other organ transplantation.
Except for FSGS patients, use of systemic immunosuppressant medications, such as steroids, for more than 2 weeks in the past 3 months.
Blood pressure above 150 mmHg systolic or 95 mmHg diastolic as evaluated by the Investigator.
History of heart failure or a previous hospital admission for fluid overload.
Clinically significant history of liver disease as assessed by the Investigator.
Hemoglobin below 9 g/dL as measured by the Investigator or blood transfusion for anemia within the past 3 months.
Clinical diagnosis of nephrotic syndrome
Malignancy within the past 5 years. Exception to the criteria include nonmelanoma skin cancer and curatively treated cervical carcinoma in situ.
For women, pregnant, breastfeeding, or intent to become pregnant during the study.
For men, intent to father a child or donate sperm during the study.
Recently received an investigational agent.
Clinically significant unstable or uncontrolled medical condition as assessed by the Investigator. Atrasentan, Atrasentan
Kidney, Prostate & Urinary
Glomerular Disease, Alport Syndrome, IgAN, FSGS
COG AREN1921 - Treatment of Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT)
This phase II trial studies how well combination chemotherapy works in treating patients (≤ 30 years old) with newly diagnosed stage II-IV diffuse anaplastic Wilms tumors (DAWT) or favorable histology Wilms tumors (FHWT) that have come back (relapsed).This trial may help doctors find out what effects, good and/or bad, regimen UH-3 (vincristine, doxorubicin, cyclophosphamide, carboplatin, etoposide, and irinotecan) has on patients with newly diagnosed DAWT and standard risk relapsed FHWT (those treated with only 2 drugs for the initial WT)and regimen ICE/Cyclo/Topo (ifosfamide, carboplatin, etoposide, cyclophosphamide, and topotecan) has on patients with high and very high risk relapsed FHWT (those treated with 3 or more drugs for the initial WT).
Emily Greengard
All
to 30 Years old
Phase 2
STUDY00011385
STUDY00011385
Inclusion Criteria:
Patients with newly diagnosed stages 2 - 4 diffuse anaplastic Wilms tumor must be enrolled on AREN03B2 and have received an initial risk assignment showing DAWT (if anaplasia first identified at diagnostic, pre-treatment nephrectomy or biopsy) or a delayed nephrectomy classification showing DAWT (if anaplasia first noted at delayed nephrectomy) prior to enrollment on AREN1921. Prior enrollment on AREN03B2 is not an eligibility requirement for patients with relapsed favorable histology Wilms tumor.
Patients must be =< 30 years old at study enrollment
Patients with the following diagnoses are eligible for this study:
Newly diagnosed stages 2 - 4 diffuse anaplastic Wilms tumor as confirmed by central review
Favorable histology Wilms tumor at first relapse. Relapsed FHWT patients must have previously achieved remission for their initial FHWT diagnosis to be eligible for this study. The relapse risk groups are defined as follows, regardless of radiation therapy:
Standard-Risk relapse: Patients who received two chemotherapy agents for frontline therapy; primarily actinomycin D and vincristine
High-Risk relapse: Patients who received three chemotherapy agents for frontline therapy; primarily vincristine, actinomycin D and doxorubicin or vincristine, actinomycin D and irinotecan
Very High-Risk relapse: Patients who received four or more chemotherapy agents as part of initial therapy; primarily regimen M or its variations
Patients with newly diagnosed DAWT must have had histologic verification of the malignancy. For relapsed FHWT patients, biopsy to prove recurrence is encouraged, but not required
Note: For relapsed FHWT patients, an institutional pathology report confirming favorable histology Wilms tumor (from relapse, if available, or from original diagnosis) must be available for upload prior to initiation of protocol therapy
Patients with newly diagnosed Stages 2 - 4 diffuse anaplastic Wilms tumor must be enrolled on AREN1921 within 2 weeks of the tumor-directed surgery or biopsy procedure that first confirms a diagnosis of DAWT, whether at initial diagnostic procedure or delayed nephrectomy (such surgery/biopsy is day 0). For patients who received prior therapy for presumed favorable histology Wilms tumor, later confirmed to have diffuse anaplastic Wilms tumor at subsequent review of the initial biopsy
Patients with newly diagnosed DAWT who undergo upfront nephrectomy must have at least 1 lymph node sampled prior to study enrollment
Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2. Use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age
Patients must have a life expectancy of >= 8 weeks
Diffuse Anaplastic Wilms Tumor: Patients with diffuse anaplastic histology must have had no prior systemic therapy, except in the following situations:
Patients with diffuse anaplastic Wilms tumor who received no more than 12 weeks of pre nephrectomy chemotherapy for what was originally presumed to be favorable histology Wilms tumor, subsequently confirmed to be diffuse anaplastic Wilms tumor at delayed nephrectomy
Patients with diffuse anaplastic Wilms tumor who received no more than 6 weeks of chemotherapy following upfront biopsy, initiated within 14 days of biopsy, for presumed favorable histology Wilms tumor based on institutional review, but subsequently corrected to diffuse anaplastic Wilms tumor based on the AREN03B2 initial risk assignment results (if available per current version of AREN03B2)
Treatment consisting of vincristine/doxorubicin/cyclophosphamide initiated on an emergent basis and within allowed timing as described
Note: Patients who received prior therapy for presumed favorable histology Wilms tumor, later identified to have diffuse anaplastic Wilms tumor as per above, must begin study treatment starting at cycle 3 (week 7) of regimen UH 3. Patients who received emergency radiation to preserve organ function are eligible as noted. Patients who received radiation as part of standard of care for presumed newly diagnosed favorable histology Wilms tumor, along with chemotherapy as noted above, prior to identification of diffuse anaplasia, are also eligible
Relapsed Favorable Histology Wilms Tumor: Patients must not have received prior chemotherapy for their relapsed favorable histology Wilms tumor diagnosis. In addition, patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study
Myelosuppressive chemotherapy: Must not have received within 2 weeks of entry onto this study
Radiation therapy (RT): >= 2 weeks (wks) must have elapsed for local palliative RT (small port); >= 6 months must have elapsed if prior craniospinal RT or if >= 50% radiation of pelvis; >= 6 wks must have elapsed if other substantial bone marrow (BM) radiation. Patients with relapsed favorable histology Wilms tumor who received emergency radiation to preserve organ function are eligible and do not need to washout with the above criteria
Patients may not be receiving any other investigational agents (within 4 weeks prior to study enrollment)
Peripheral absolute neutrophil count (ANC) >= 750/uL (performed within 7 days prior to enrollment)
Platelet count >= 75,000/uL (transfusion independent) (performed within 7 days prior to enrollment)
Hemoglobin >= 8.0 g/dL (may receive red blood cell [RBC] transfusions) (performed within 7 days prior to enrollment)
Patients with high-risk or very high-risk relapsed FHWT who will be treated with regimen ICE/Cyclo/Topo, must have renal function assessed by creatinine clearance or radioisotope glomerular filtration rate (GFR) and meet the following requirement:
Creatinine clearance or radioisotope GFR >= 60 mL/min/1.73 m^2 (performed within 7 days prior to enrollment)
Patients diagnosed with stage 2-4 DAWT or standard risk relapsed FHWT, who will be treated with regimen UH 3, may either obtain a creatinine clearance, radioisotope GFR (meeting the above criteria of GFR >= 60 mL/min/1.73 m^2), or an adequate serum creatinine as per the following table:
Age: Maximum Serum Creatinine (mg/dL)
1 month to < 6 months: 0.4 (male and female)
6 months to < 1 year: 0.5 (male and female)
1 to < 2 years: 0.6 (male and female)
2 to < 6 years: 0.8 (male and female)
6 to < 10 years: 1 (male and female)
10 to < 13 years: 1.2 (male and female)
13 to < 16 years: 1.5 (male), 1.4 (female)
>= 16 years: 1.7 (male), 1.4 (female)
Total bilirubin =< 1.5 x upper limit of normal (ULN) for age or direct bilirubin =< ULN for patients whose total bilirubin > 1.5 x ULN (performed within 7 days prior to enrollment)
Serum glutamic-oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 2.5 x upper limit of normal (ULN) for age or =< 5 x ULN for patients with liver metastases (performed within 7 days prior to enrollment)
Shortening fraction of >= 27% by echocardiogram, or ejection fraction of >= 50% by radionuclide angiogram (obtained within 21 days prior to enrollment and start of protocol therapy)
Exclusion Criteria:
Patients with a history of bilateral Wilms tumor (synchronous or metachronous)
Patients with any uncontrolled, intercurrent illness including, but not limited to, ongoing or active infection, or symptomatic congestive heart failure (defined as grade 2 or higher heart failure per Common Terminology Criteria for Adverse Events [CTCAE] version 5.0)
Relapsed FHWT patients who did not receive frontline chemotherapy (e.g., very low risk FHWT initially observed without chemotherapy) or received only one chemotherapy agent for frontline therapy
For patients with high-risk or very high-risk relapsed FHWT:
Patients with renal tubular acidosis (RTA) as evidenced by serum bicarbonate < 16 mmol/L and serum phosphate =< 2 mg/dL (or < 0.8 mmol/L) without supplementation
For stages 2-4 DAWT and standard-risk relapsed FHWT patients:
Chronic inflammatory bowel disease and/or bowel obstruction
Concomitant use of St. John's wort, which cannot be stopped prior to the start of trial treatment
Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential
Lactating females who plan to breastfeed their infants
Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation Biopsy, Biospecimen Collection, Bone Scan, Carboplatin, Computed Tomography, Cyclophosphamide, Doxorubicin, Etoposide, Ifosfamide, Irinotecan, Magnetic Resonance Imaging, Positron Emission Tomography, Radiation Therapy, Surgical Procedure, Topotecan, Transabdominal Ultrasound, Vincristine, X-Ray Imaging, Biopsy, Biospecimen Collection, Bone Scan, Carboplatin, Computed Tomography, Cyclophosphamide, Doxorubicin, Etoposide, Ifosfamide, Irinotecan, Magnetic Resonance Imaging, Positron Emission Tomography, Radiation Therapy, Surgical Procedure, Topotecan, Transabdominal Ultrasound, Vincristine, X-Ray Imaging
Anaplastic Kidney Wilms Tumor, Recurrent Kidney Wilms Tumor, Stage II Kidney Wilms Tumor, Stage III Kidney Wilms Tumor, Stage IV Kidney Wilms Tumor
A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Reducing Supraphysiologic Glucocorticoid Use in Adult Subjects with Classic Congenital Adrenal Hyperplasia
SPR001-204 will be a randomized, double-blind, placebo-controlled study that will evaluate the potential of tildacerfont to reduce GC burden in adult subjects with classic CAH who have lower limit of detection (LLD) ≤ A4 ≤ 1.5x upper limit of normal (ULN) and are on supraphysiologic doses of GC therapy. SPR001-204 will be the first study of tildacerfont to evaluate GC dose reduction. In addition, Study SPR001-204 will characterize clinical outcomes after up to 52 weeks of treatment with tildacerfont.
Kyriakie Sarafoglou
Male or Female
18 Years and over
Phase II
STUDY00011764
STUDY00011764
Inclusion Criteria:
Male and female subjects over 18 years old, inclusive
Has a documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or documented elevated 17-OHP and currently treatment with HC, HC acetate, prednisone, prednisolone, methylprednisolone (or a combination of the aforementioned GCs)
Has been on a stable, supraphysiologic dose of GC replacement for ≥1 month before screening.
For subjects with the salt-wasting form of CAH, subject has been on a stable dose of mineralocorticoid replacement for ≥1 month before screening
Exclusion Criteria:
Has a known or suspected diagnosis of any other known form of classic CAH (not due to 21-hydroxylase deficiency)
Has a history that includes bilateral adrenalectomy or hypopituitarism
Has a history of allergy or hypersensitivity to tildacerfont, any of its excipients, or any other CRF1 receptor antagonist
Shows clinical signs or symptoms of adrenal insufficiency Tildacerfont/Placebo, Tildacerfont/Placebo
Diabetes & Endocrine
congenital adrenal hyperplasia
The efficacy of incobotulinum toxin A injections for treatment of tinnitus: a randomized controlled trial
This study is a double blinded crossover clinical trial evaluating the safety and effectiveness of incobotulinum toxin A or a placebo (before crossover) injections for the treatment of tinnitus.
Stephanie Standal
STUDY00011665
STUDY00011665
Ear, Nose & Throat
Lifestyle Counseling and Medication for Adolescent Weight Management (QUEST)
This study will compare the effectiveness and durability of intensive behavioral counseling vs. medical management plus low-intensity behavioral counseling on BMI, body fat, cardiometabolic risk factors, and quality of life in adolescents with severe obesity. We hypothesize that liraglutide plus low-intensity behavioral counseling will elicit superior reductions in BMI (primary efficacy endpoint) and body fat and greater improvements in cardiometabolic risk factors and quality of life compared to intensive behavioral counseling at 56 weeks.
Aaron Kelly
All
12 Years to 17 Years old
Phase 2
STUDY00012932
STUDY00012932
Inclusion Criteria:
Severe obesity (Body Mass Index (BMI) >/= 120% of the 95th percentile or BMI >/= 35 kg/m2)
Age 12 to < 18 years old and Tanner stage >1
Exclusion Criteria:
Diabetes (type 1 or 2)
Current or recent (< 6 months prior to enrollment) use of anti-obesity medication(s) defined as orlistat, phentermine, topiramate, combination phentermine/topiramate, liraglutide (or other GLP-1RA) and/or combination naltrexone/bupropion (monotherapy use of naltrexone or bupropion is not an exclusion)
Previous bariatric surgery
Any history of treatment with growth hormone
Medically-documented history of bulimia nervosa
Major psychiatric disorder as determined by the local medical monitor
Unstable depression requiring hospitalization within the previous 6 month
Any history of suicide attempt
History of suicidal ideation or self-harm within the previous 30 days
Current pregnancy or plans to become pregnant
ALT or AST >/= 5 times the upper limit of normal
Creatinine > 1.2 mg/dL
Uncontrolled hypertension as determined by the local medical monitor
Diagnosed and medically-documented monogenic obesity
Medically-documented history of cholelithiasis
Untreated thyroid disorder
Medically documented history of pancreatitis
Personal or family history of medullary thyroid carcinoma or multiple endocrine neoplasia syndrome type 2
Clinically significant heart disease as determined by the local medical monitor
Personal history of malignant neoplasms within the past five years
Hypersensitivity to any component of semaglutide Intensive Behavioral Program, Semaglutide and Behavioral Program, Intensive Behavioral Program, Semaglutide and Behavioral Program
Obesity, Childhood
Phase II Randomized, Intervention Versus Non-Intervention, Multi-Center Study of the Effects of Thyroid Hormone (T3) on Extravascular Lung Water (EVLW) in Subjects with Acute Respiratory Distress Syndrome (ARDS) (ARDS+T3)
Study objective: To determine the safety and tolerability of Thyroid Hormone (T3) delivery into the lungs of Acute Respiratory Distress Syndrome (ARDS) patients, and to measure the effect of T3 on extravascular lung water in ARDS patients.
Ronald Reilkoff
All
18 Years to old
Phase 1/Phase 2
STUDY00007410
STUDY00007410
Inclusion Criteria:
Clinical diagnosis of ARDS:
Chest x-ray: bilateral pulmonary infiltrates
Hypoxemia: PaO2:FIO2 ratio <200
Volume status: wedge and CVP<18
Main inclusion criteria:
Adults (≥18 years of age), non-pregnant
On mechanical ventilatory support
Exclusion Criteria:
Inadequate medical history for determining inclusion/exclusion criteria, as determined by the Principal Investigator and/or Sub-Investigators.
Unlikely to complete the protocol with clinic follow-up after discharge, as determined by the Principal Investigator and/or Sub-Investigators or hospice status.
Active drug/alcohol use with positive drug screen or alcohol level on admission.
Prior history of thyroid cancer or hyperthyroidism, per thorough patient/family interviews, review of past medical history, medication list, laboratory test.
Prior history of cardiovascular disease including:
Hypertensive crisis in the past 3 months (systolic >200, or diastolic >120 mmHg),
Sustained ventricular arrhythmia in the past 3 months (duration > 30 seconds)
Coronary artery disease (documented >50% occlusion in any coronary vessel)
Cardiac-related angina pectoris (> 2 episodes in the past 3 months)
Myocardial infarction with ischemia on ECG (i.e., new ST-elevation/depression of >1mm in contiguous leads), or positive cardiac enzymes (Ratio of CK-MB: Total CK > 3.5).
Peripheral vascular disease (documented >50% occlusion in any peripheral vessel).
Moderate or severe ischemic/non-ischemic cardiomyopathy (documented ejection fraction < 40%).
Decompensated or symptomatic heart failure (i.e., hospitalized for CHF exacerbation, or a change in CHF medications within two weeks prior)
Currently pregnant or breastfeeding.
Currently taking tricyclic antidepressants, glycosides, ketamine, or vasopressors with ongoing evidence of myocardial ischemia.
Known allergy to study drug. Liothyronine Sodium (T3) (modified formulation), Liothyronine Sodium (T3) (modified formulation)
ARDS, Human, Lung, Wet, Thyroid, Pulmonary Edema, Lung Inflammation
COG AOST2121 - An Open-Label Phase 2 Study of Maintenance Therapy with OST31-164 After Resection of Recurrent Osteosarcoma (OST-164-01)
This phase II trial studies OST31-164 as a single agent every 3 weeks for 48 weeks, with 4 doses constituting 1 treatment cycle (12 weeks per cycle). Each patient will receive treatment at a dose of 1x109 CFU until Week 48 or until disease progression, unacceptable toxicity, or the patient meets any other treatment discontinuation criteria. Following treatment discontinuation, all patients will enter a 3-year survival follow-up period. The primary endpoints are disease control during the first 12 months after enrollment and safety assessments (adverse events [AEs], physical examinations, clinical laboratory tests, vital sign measurements, performance status, and tests to monitor for the persistence of Lm).
Emily Greengard
All
12 Years to 39 Years old
Phase 2
STUDY00013667
STUDY00013667
Inclusion Criteria:
Note: Patients enrolled on AOST2031 are eligible for enrollment in the present study.
Patients are eligible to be included in the study only if all the following criteria apply:
Age and Weight
Between 12 years of age and 39 years of age at the time the Informed Consent/ Assent form is signed.
Weight at least 40 kg. Diagnosis
Has histologic confirmation of osteosarcoma at diagnosis.
Has at least one episode of disease recurrence in the lungs without limitation on the number of episodes of recurrence as long as the following criteria are met:
Surgical resection of all possible sites of suspected pulmonary metastases to achieve a complete remission within 8 weeks prior to study enrollment
Pathological confirmation of osteosarcoma from at least one resected tumor.
Patients will not require radiographic confirmation of complete remission for enrollment. However, a postoperative CT chest scan is required as a baseline for future comparisons.
https://members.childrensoncologygroup.org/files/Disc/surgery/handbooks/OsteoBoneHandbook.pdf) Performance Status
Patient must have a performance status corresponding to ECOG scores of 0, 1, or 2. Use Karnofsky scale for patients > 16 years of age and Lansky scale for patients < years of age Prior Therapy
Patient must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, radiotherapy, or surgery prior to entering this study.
Organ Function Requirements
Patient has adequate organ function as defined below:
a. Hematological: i. Absolute neutrophil count (ANC) is at least 1,000/µL without transfusion or growth factor support. ii. Platelet count ≥ 50,000/µL without transfusion or growth factor support. b. Adequate renal function defined as: i. Creatine clearance or radioisotope glomerular filtration rate (GFR) > 70 mL/min/1.73 m2 or ii. A serum creatine based on age/gender as follows:
Maximum Serum Creatinine (mg/dL)
Age: 12 to < 13 years Male :1.2 Female:1.2 Age:13 to < 16 years Male :1.5 Female:1.4 Age: ≥ 16 years Male :1.7 Female:1.4 Note: the threshold for creatinine values in this table were derived from the Schwartz formula for estimating GFR.
c. Adequate liver function defined as: i. Total bilirubin < 1.5 x upper limit of normal (ULN) for age ii. Serum glutamic-pyruvic transaminase (SGPT) / alanine aminotransferase (ALT) < 110 U/L (for the purpose of this study the ULN for SGPT is 45 U/L) iii. Serum albumin > 2 g/dL
d. Adequate coagulation i. International normalized ratio (INR) or prothrombin time (PT) < 1.5 x ULN unless patient is receiving anticoagulant therapy as long as PT or INR is within therapeutic range of intended use of anticoagulants.
ii. Activated partial thromboplastin time (aPTT) < 1.5 x ULN unless patient is receiving anticoagulant therapy as long as aPTT is within therapeutic range of intended use of anticoagulants.
e. Adequate cardiac function defined as: i. Shortening fraction of > 27% by echocardiogram, or ii. Ejection fraction of > 50% by radionuclide angiogram or echocardiogram f. Adequate pulmonary function defined as: i. No evidence of dyspnea at rest, no history of exercise intolerance, and a pulse oximetry of > 94%.
g. Central nervous system (CNS) function defined as: i. Patients with a known seizure disorder may be enrolled if on anticonvulsants and/or are well-controlled.
ii. CNS toxicity including peripheral neuropathy < Grade 2.
Patient and/or patient's parent or legal guardian must be capable of understanding the investigational nature, potential risks, and benefits of the study. The patient and/or the parent or legal guardian must sign a written informed consent. Age-appropriate assent will be obtained per institutional guidelines.
Contraception:
Female patients :
A female patient is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:
Not a woman of childbearing potential (WOCBP) as defined in the protocol OR
A WOCBP who agrees to follow the contraceptive guidance in the protocol during the treatment period and for at least 120 days after the last dose of study treatment.
A female patient of childbearing potential must have a negative urine or serum pregnancy test within 72 hours prior to receiving any dose of study treatment. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required.
Male patients:
A male patient is eligible to participate if he agrees to follow the contraceptive guidance in the protocol during the study treatment period and for at least 120 days after the last dose of study treatment.
Exclusion Criteria:
Has clinically evident metastatic or recurrent disease.
Has concurrent pulmonary recurrence and local recurrence at the primary tumor site.
Has primary refractory disease with progression of the primary tumor on initial-therapy.
Has CNS or any extrapulmonary disease involvement at the time of the most recent episode of disease recurrence proceeding enrollment.
Has active infection requiring systemic therapy or is dependent on or is currently receiving systemic antibiotics that cannot be discontinued before dosing. (Note: Patients who discontinue an antibiotic prior to dosing must wait at least 5 half-lives after the last dose of antibiotic before receiving any OST31-164 infusion). Inhaled prophylactic PJP (pneumocystis jiroveci pneumonia) treatment is acceptable per Investigator discretion.
Is currently dependent on or has received corticosteroids within the past 4 weeks (topical corticosteroids and occasional inhaled corticosteroids are allowed).
Is currently participating in or has participated in a study of an investigational agent or is using an investigational device within 4 weeks of the first dose of treatment.
Has a history of other active malignancy for < 2 years prior to enrollment. Basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or in situ cervical cancer that has undergone potentially curative therapy or is felt by the Investigator to be at low risk for recurrence is allowed.
Has an active autoimmune disease requiring systemic treatment within the past 3 months or a documented history of clinically severe autoimmune disease, or a syndrome that requires systemic steroids or immunosuppressive agents. Replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency) is not considered a form of systemic treatment. Patients with vitiligo or resolved childhood asthma/atopy would be an exception to this rule. Patients who require intermittent use of bronchodilators or local steroid injections will not be excluded from the study. Patients with hypothyroidism stable on hormone replacement or Sjogren's syndrome will not be excluded from the study.
Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.
Is pregnant or breastfeeding or expecting to conceive or father children within the projected duration of the trial, starting with the pre-screening or screening visit through 120 days after the last dose of study treatment.
Has a known allergy to any component of the study treatment(s) formulations.
Has a contraindication (e.g., sensitivity/allergy) to both trimethoprim/ sulfamethoxazole and amoxicillin.
Has contraindication to administration of NSAIDs.
Is currently receiving or will be receiving any chemotherapy, including PI3K inhibitors, during the treatment phase.
Has had a prior monoclonal antibody therapy within 2 weeks prior to study Day 1.
Requires or anticipates requiring tumor necrosis factor (TNF) blocking agent (e.g., infliximab) therapy for diagnosis of rheumatologic disease or inflammatory bowel disease (e.g., ankylosing spondylitis, Crohn's disease, plaque psoriasis, psoriatic arthritis, rheumatoid arthritis, or ulcerative colitis).
Has previous history of listeriosis.
Has a known history of human immunodeficiency virus (HIV) (HIV 1/2 antibodies).
Has known active hepatitis B (e.g., HBsAg reactive) or hepatitis C (e.g., HCV RNA [qualitative] is detected).
Has received a live vaccine within 30 days prior to Study Day 1.
Patient is or has an immediate family member (spouse, children, or parent) who is directly involved with this study or is employed by the investigational site or Sponsor, unless prospective Institutional Review Board (IRB) approval (by chair or designee) is given allowing exception to this criterion for a specific patient.
Regulatory Requirements:
All patients and/or their parents or legal guardians must sign a written informed consent.
All institutional, FDA, and NCI requirements for human studies must be met. OST31-164, OST31-164
Bone Cancer
Osteosarcoma, Bone Cancer
ARACOG: A Randomized Phase II Study of Androgen Receptor Directed Therapy on COGnitive Function in Patients Treated with Darolutamide or Enzalutamide (ARACOG)
To compare the effects of treatment with enzalutamide (ENZ) versus darolutamide (DARO) on the cognitive function of men with non-metastatic and metastatic castration-resistant prostate cancer (mCRPC) by comparing the change in the maximally changed cognitive domain from baseline in patients in each study arm by 24 weeks.
Stuart Bloom
Male
18 Years to old
Phase 2
STUDY00010912
STUDY00010912
Key inclusion criteria include:
Histologically or cytologically confirmed adenocarcinoma of the prostate without neuroendocrine differentiation or small cell features
Progressive disease per PCWG3 criteria
Metastatic CRPC or non-metastatic CRPC (M0CRPC)
Castration-resistant prostate cancer demonstrated during continuous ADT, defined as 3 PSA rises at least 1 week apart, with the last PSA >2 ng/mL.
For mCRPC: metastatic disease documented by standard or novel imaging techniques OR for M0CPRC: no evidence of metastatic disease on standard imaging.
Surgically or medically castrated, with testosterone levels of <50 ng/dL. If the patient is medically castrated, continuous dosing with GnRHa must have been initiated at least 4 weeks prior to randomization and must be continued throughout the study.
Eastern Cooperative Oncology Group (ECOG) performance status ≤2
Able to complete cognitive testing and patient reported outcome surveys in English.
Ability to swallow study tablets whole.
Able to provide informed consent.
Key exclusion criteria include:
Prior chemotherapy for treatment of CRPC. Patients who received chemotherapy for castrate-sensitive prostate cancer are still eligible provided chemotherapy was completed >6 months prior to study entry.
Use of investigational agents for the treatment of prostate cancer within 4 weeks of study entry.
Prior usage of ENZ or DARO.
Prior use of apalutamide
Prior use of investigational agents that act on the androgen axis.
Progression during treatment with abiraterone (PSA or radiographic progression). Must have < 12 weeks of abiraterone exposure prior to enrollment if given for treatment of CRPC. If used with radiation for high risk localized hormone sensitive disease, can enroll if no progression of disease during treatment with abiraterone (PSA or radiographic) and >6 months since last exposure to abiraterone.
Planned radiation treatment > 21 days during enrollment in the study.
Any active, or prior history of, brain metastasis that have not been treated and stabilized.
Active or history of seizures or seizure disorder.
Prior diagnosis of dementia or other neurologic impairment.
Use of chronic opiates (other than stable doses of opioids that in the view of the patient and investigator do not affect cognition).
Clinically significant history of falls or risk of falls at baseline (timed up-and-go (TUG) test time of >12 seconds).
Darolutamide, Enzalutamide, Darolutamide, Enzalutamide
Metastatic Prostate Cancer, Prostate Cancer Metastatic, Prostate Cancer, Castrate Resistant Prostate Cancer
Clinics and Surgery Center (CSC)
State Representation in Early Psychosis (STEP)
Our Center will focus on the unifying hypothesis that processes underlying state representation dysfunction are relevant to psychosis, providing a window into pathophysiologic heterogeneity and precision treatment. Our Center will study three species (nonhuman primates, mice, and humans) using eight methodologies (genetic manipulations, slice physiology, ensemble recordings, LFP, behavior, EEG, fMRI, cognitive training). We will use a central computational perspective to translate and integrate across species and methodologies: Changes in neural information processing that affect parameters underlying attractor dynamics and influence state representation processes. Such changes create observable effects in behavior and neurophysiology, which we will study through the lens of attractor network models to inform our understanding of pathophysiologic heterogeneity, clinical trajectories, and precision treatment.
Sophia Vinogradov
Male or Female
Not specified
N/A
STUDY00009964
STUDY00009964
Inclusion Criteria:
English proficiency, as determined by staff observation and participant self-report
Estimated IQ at or above 70, as estimated by the cognitive assessments
Additional Inclusion Criteria for Early Psychosis Participants:
Clinical diagnosis of schizophrenia, schizoaffective disorder, schizophreniform disorder, psychosis NOS, bipolar disorder with psychosis, or major depressive disorder with psychosis, with onset of psychotic symptoms within the previous 5 years
Achieved clinical stability, defined as outpatient status for at least one month prior to study participation plus clinically stable doses of psychiatry medications for at least one month prior to study participation
Exclusion Criteria:
Unable or unwilling to provide informed consent
The participant is unable to demonstrate adequate decisional capacity, in the judgment of the consenting study staff member, to make a choice about participating in the research study
Participant is pregnant
Participant is illiterate
Cannot pass the CMRR Subject Safety Screen due to MRI contraindications
Presence of a major neurological disorder
Previous clinically significant head injury or prolonged unconsciousness, as determined by the PI/Co-Is
Meets criteria for substance or alcohol dependence within 3 months of enrollment
The presence of any major medical condition that, in the opinion of the PI/Co-Is, would impede participation in the study or would put the participant at additional risk by participating
Additional Exclusion Criteria for Early Psychosis Participants:
Has participated in significant formal cognitive training programs, as determined by the PI/Co-Is
Meets criteria for clinical risk of suicidal behavior, as defined by:
Clinician judgement
A suicide attempt within 6 months of enrollment
Active suicidal ideation at screening or baseline, as indicated by the C-SSRS
Previous intent to act on suicidal ideation with a specific plan and/or preparatory acts within 6 months of enrollment, as indicated by the C-SSRS
Additional Exclusion Criteria for Control Participants:
Meets DSM-5 criteria for psychotic, bipolar, or autism spectrum disorder
Has a family history (1st degree relative) of psychotic, bipolar, or autism spectrum disorder Computerized Cognitive Training, Computerized Cognitive Training
Mental Health & Addiction
A PHASE 2, OPEN-LABEL, SINGLE-ARM, COHORT STUDY TO EVALUATE THE SAFETY, EFFICACY, AND PHARMACOKINETICS OF SPARSENTAN TREATMENT IN PEDIATRIC SUBJECTS WITH SELECTED PROTEINURIC GLOMERULAR DISEASES (EPPIK) (EPPIK)
Currently, there are no approved treatment options for pediatric subjects with proteinuric kidney conditions. The study will look at the safety, efficacy, and pharmacokinetic (PK) trial in children ≥1 to <18 years treated for up to 108 weeks with the drug sparsentan.
Michelle Rheault
Male or Female
up to 18 Years old
STUDY00013216
STUDY00013216
Inclusion Criteria:
• Child 1 to 18 years old
• Diagnosed by biopsy with specific types of glomerular disease & protein in the urine
• Blood pressure is within normal range for age
• Maintained on a stable dose of immunosuppressive medications
Exclusion Criteria:
• Weight less than 7.3 kg at screening.
• Disease due to to viral infections, drug toxicities, or cancer.
• Kidney function is below the minimum required
Sparsentan, Sparsentan, Sparsentan, Sparsentan
Rare Diseases, Children's Health, Kidney, Prostate & Urinary
Glomerulosclerosis, Immunoglobulin A Nephropathy, IgA Vasculitis, Alport Syndrome