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COG AGCT1531 - A Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients with Germ Cell Tumors
This partially randomized phase III trial studies how well active surveillance, bleomycin, carboplatin, etoposide, or cisplatin work in treating pediatric and adult patients with germ cell tumors. Active surveillance may help doctors to monitor subjects with low risk germ cell tumors after their tumor is removed. Drugs used in chemotherapy, such as bleomycin, carboplatin, etoposide, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
Emily Greengard
Not specified
SITE00000295
Inclusion Criteria:
• newly diagnosed with a Stage I germ cell tumor or metastatic germ cell tumor
• see link to clinicaltrials.gov for detailed inclusion criteria
Exclusion Criteria:
• patients must have had no prior systemic therapy for the current cancer diagnosis
• patients must have had no prior radiation therapy (exception of CNS irradiation of brain metastases for standard risk 1 patients)
• female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs
• lactating females who plan to breastfeed their infants
• there are additional exclusion criteria (study staff will review)
Cancer
Germ Cell Tumor, Malignant Germ Cell Tumor
ALX-HPP-501: An Observational,Longitudinal Prospective, Long-term Registry of Patients with Hypophosphatasia
This is a long-term registry is designed to collect data on hypophosphatasia (HPP) to better understand the condition and learn more about the disease, how patients feel about living with HPP and effect of HPP on the patients wellbeing and health. The study will look at participant’s medical records and health questionnaires about the health status of patients. This study collects observational data from clinical care and does not involve any treatment for HPP or administration of medication for HPP.
Kyriakie Sarafoglou
Not specified
STUDY00004936
Inclusion Criteria:
• confirmed diagnosis of HPP.
• documented alkaline phosphatase (ALP) activity below the lower limit of normal for age and sex, or a documented ALPL gene mutation.
• able to read and speak English
Exclusion Criteria:
• currently participating in an Alexion-sponsored clinical trial
Rare Diseases
HPP, Hypophosphatasia
COG AALL2121: A Phase 2 study of SNDX-5613 in combination with chemotherapy for patients with relapsed or refractory KMT2A-rearranged infant leukemia
This phase II trial tests the safety and best dose of revumenib when given together with chemotherapy, and how well the treatment regimen works for infants and young children with leukemia that has come back (relapsed) or does not respond to treatment (refractory) and is associated with a KMT2A (MLL) gene rearrangement (KMT2A-R). Revumenib is an oral medicine that directly targets the changes that occur in a cell with a KMT2A rearrangement and has been shown to specifically kill these leukemia cells in test tubes and animals. Drugs used in chemotherapy, such as vincristine, prednisone, asparaginase, fludarabine and cytarabine work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. This trial is being done to find out if the combination of revumenib and chemotherapy may help to treat the cancer cells better than either treatment alone.
Peter Gordon
Up to 18 years old
STUDY00021176
Inclusion Criteria:
• Age: Patients must be 1 month to less than 6 years old at the time of study enrollment and must have had initial diagnosis of leukemia less than 2 years old.
• Diagnosis: Patients must have KMT2A-rearranged acute lymphoblastic leukemia (ALL), acute leukemia of ambiguous lineage (ALAL), or mixed phenotype acute leukemia (MPAL), which is determined to be refractory or in first marrow relapse.
• Disease status: First relapse, refractory or failure to achieve remission
Exclusion Criteria:
• Patients with isolated extramedullary leukemia.
• Patients diagnosed with Down syndrome.
Cancer
ALAL, ALL, leukemia, MPAL, relapse
INHALE-1: A 26-week Primary Treatment Phase, with 26-week Extension, Open-label, Randomized Clinical Trial Evaluating the Efficacy and Safety of Afrezza? Versus Rapid-acting Insulin Analog Injections, Both in Combination with a Basal Insulin, in Pediatric Subjects with Type 1 or Type 2 Diabetes Mellitus (INHALE-1)
To assess the safety of Afrezza in a pediatric population when compared to the usual standard of care insulin.
Muna Sunni
Up to 18 years old
SITE00001625
Inclusion Criteria:
• 4 years to under 18 years old
• diagnosis of type 1 diabetes mellitus (T1DM) or type 2 diabetes mellitus (T2DM)
• using insulin for at least 6 months for T1DM, or at least 3 months for T2DM
• treated with basal-bolus insulin therapy delivered by multiple daily injections for at least 2 weeks
• bolus insulins are restricted to the RAAs insulin lispro, insulin aspart or insulin glulisine, including biosimilar products
• basal insulins are restricted to insulin glargine, insulin degludec or insulin detemir, including biosimilar products
• HbA1c between 7.0% and 11%
• average prandial dose of insulin 2 or more units per meal
• used CGM for at least 70% of the time over a consecutive 14-day period before starting the study
• access to stable WiFi connection
Exclusion Criteria:
• history of recent blood transfusions (within previous 3 months)
• recent history of asthma (defined as using any medications to treat within the last year) or any other clinically important lung disease
• history of serious complications of diabetes
• any other illness that isn't stable (study staff will review)
• uncontrolled eating disorder (e.g., anorexia or bulimia nervosa)
• current drug or alcohol abuse or a history of drug or alcohol abuse
• smoking (includes cigarettes, cigars, pipes, marijuana, and vaping devices) for the preceding 6 months
Diabetes & Endocrine
diabetes, insulin, type 1 diabetes, type 2 diabetes
ONC201 for the Treatment of Newly Diagnosed H3 K27M-mutant Diffuse Glioma Following Completion of Radiotherapy: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study (ACTION)
The study will test dordaviprone (also known as ONC201), a new drug that is being developed for treating people with glioma. ONC201 may help to remove tumor cells without affecting normal cells of the body. ONC201 is a first-in-class drug, which means it is a new drug having unique action.
Elizabeth Neil
Not specified
STUDY00021033
Inclusion Criteria:
• body weight at least 10 kg (22 pounds)
• new diagnosis of H3 K27M-mutant diffuse glioma
• started radiotherapy within 12 weeks from the initial diagnosis
• completed radiotherapy within 2 to 6 weeks prior to starting the study treatment
• see link to clinicaltrials.gov for complete Inclusion and Exclusion criteria
Exclusion Criteria:
• primary spinal tumor
• diffuse intrinsic pontine glioma (DIPG)
• evidence of leptomeningeal spread of disease or cerebrospinal fluid dissemination
• received whole-brain radiotherapy or proton therapy
• study staff will review additional criteria
Cancer
Clinics and Surgery Center (CSC), Brain Cancer, Glioma, H3 K27M
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
Up to 18 years old
1311M46101
Inclusion Criteria:
• no more than 17 years old
• documented infantile or juvenile gangliosidosis disease
Exclusion Criteria:
• severe kidney disease
• females who are pregnant or breast feeding
• females who are post puberty who are unwilling to use highly effective birth control
Rare Diseases
GM1 Gangliosidoses, GM2 Gangliosidoses, Sandhoff Disease, Tay-Sachs Disease
MT2013-09C : Umbilical Cord Blood Transplantation Using a Myeloablative Preparative Regimen for the Treatment of Hematological Diseases
This is a treatment protocol for an unrelated umbilical cord blood transplant (UCBT) using a myeloablative preparative regimen for the treatment of hematological diseases, including, but not limited to acute leukemias. There is no research element except the collection of routine clinical data.
Margaret MacMillan, MD
Not specified
1305M34181
Inclusion Criteria:
• up to 55 years old
• see link to clinicaltrials.gov for inclusion criteria specific to each type of leukemia
Exclusion Criteria:
• Radiation Oncology will evaluate all patients who have had previous radiation therapy
• pregnant or breastfeeding
• HIV positive
• study staff will review additional exclusion criteria
Cancer
Clinics and Surgery Center (CSC), Acute Lymphocytic Leukemia (ALL), Acute Myeloid Leukemia (AML), Chronic Myelogenous Leukemia
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
1 year and over
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.A Phase 1/2a Open-Label Dose-Ranging and Observer-Blind Placebo-Controlled, Safety and Immunogenicity Study of mRNA-1647 Cytomegalovirus Vaccine in Female and Male Participants 9 to 15 Years of Age; mRNA-1647-P104
This study it to test an investigational vaccine called mRNA-1647 that is being developed for preventing cytomegalovirus (CMV) infection in people. CMV is a common virus that can spread easily through an infected person’s saliva or other body fluids such as blood, urine, and breast milk. We want see if the trial vaccine can prevent CMV infection in participants who have not been previously infected, to understand the safety (how many side effects you may have) of the trial vaccine, and to see if the trial vaccine results in participants making antibodies to CMV.
Mark Schleiss
Up to 18 years old
SITE00001871
Inclusion Criteria:
• female or male 9 to 15 years of age
• in good general health
• BMI requirements ( study staff will review)
• female participants of childbearing potential: negative pregnancy test and adequate contraception for at least 28 days prior to receiving vaccine through 3 months following vaccine administration
Exclusion Criteria:
• received, or plans to receive, any nonstudy vaccine less than 28 days prior to or after any study medication
• any diagnosis or condition requiring significant changes in management or medication within the 2 months before starting the study
• contact study staff for review of additional exclusion criteria
Children's Health, Infectious Diseases
CMV, cytomegalovirus, vaccine
MT2022-54 A MULTINATIONAL, MULTICENTER, DOSE ESCALATION STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PRELIMINARY ACTIVITY OF FP-045 IN PATIENTS WITH FANCONI ANEMIA (FuschiA Study)
The purpose of this research study is to determine the best dose of FP-045 for Fanconi anemia pediatric and adolescent participants. The study will look at whether the participants have any side effects and if there are any possible changes in something called “biomarkers,” which are blood proteins that will be checked to see if they change when taking FP-045 and that may indicate if FP-045 can delay or prevent disease symptoms. Every participant will receive FP-045.
Meera Srikanthan
Not specified
SITE00001887
Inclusion Criteria:
• 3 to 25 years old
• documented Fanconi anemia by chromosome breakage analysis
• women of child-bearing potential and males required to use highly effective birth control
Exclusion Criteria:
• history of any cancer except squamous cell or basal cell carcinoma of the skin or carcinoma in situ of cervix
• myelodysplastic syndrome or acute leukemia
• history of any significant medical conditions
• history of bone marrow or stem cell transplant
• see link to clinicaltrials.gov for complete criteria
Blood Disorders, Rare Diseases
Fanconi Anemia
Longitudinal Study of Porphyrias
The objective is to conduct a longitudinal investigation of the natural history, complications, and therapeutic outcomes in people with acute and cutaneous porphyria.
Marshall Mazepa, MD
Not specified
SITE00000892
Inclusion Criteria:
• patient of any age
• diagnosis of a porphyria
• biochemical findings, as documented by laboratory reports of porphyria-specific testing performed after 1980
Exclusion Criteria:
• elevations of porphyrins in urine, plasma or erythrocytes due to other diseases
Rare Diseases
Acute Porphyrias, Cutaneous Porphyrias
COG ACNS1833 - A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) not associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)
The overall goal of this phase 3 non-inferiority study is to assess if selumetinib works as well as the standard treatment using carboplatin and vincristine (called CV) for subjects with low-grade glioma (LGG).
Christopher Moertel, MD
2 years to 21 years old
SITE00000799
Inclusion Criteria:
Patients must be >= 2 years and =< 21 years at the time of enrollment
Patients must have a body surface area (BSA) of >= 0.5 m^2 at enrollment
Patients must have non-neurofibromatosis type 1 (non-NF1) low-grade glioma (LGG) without a BRAFV600E mutation as confirmed by Rapid Central Pathology and Molecular Screening Reviews performed on APEC14B1 (NCT02402244) and that has not been treated with any modality besides surgery. Note: Patients may be newly-diagnosed OR previously diagnosed, and there is no required time frame between biopsy/surgery and treatment initiation.
Patients with residual tumor after resection or progressive tumor after initial diagnosis (with or without surgery) who have not received treatment (chemotherapy and/or radiation) are eligible
Patients must have two-dimensional measurable tumor >= 1 cm^2 to be eligible
Eligible histologies will include all tumors considered low-grade glioma or low-grade astrocytoma (World Health Organization [WHO] grade I and II) by 5th edition WHO classification of central nervous system (CNS) tumors with the exception of subependymal giant cell astrocytoma
Patients with metastatic disease or multiple independent primary LGG are eligible
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 (performed within 7 days prior to enrollment):
Age: Maximum Serum Creatinine (mg/dL)
2 to < 6 years: 0.8 mg/dL (male); 0.8 mg/dL (female)
6 to < 10 years: 1 mg/dL (male); 1 mg/dL (female)
10 to < 13 years: 1.2 mg/dL (male); 1.2 mg/dL (female)
13 to < 16 years: 1.5 mg/dL (male); 1.4 mg/dL (female)
>= 16 years: 1.7 mg/dL (male); 1.4 mg/dL (female)
Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (performed within 7 days prior to enrollment) (children with a diagnosis of Gilbert's syndrome will be allowed on study regardless of their total and indirect [unconjugated] bilirubin levels as long as their direct [conjugated] bilirubin is < 3.1 mg/dL)
Serum glutamic pyruvic transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L (performed within 7 days prior to enrollment). For the purpose of this study, the ULN for SGPT is 45 U/L
Albumin >= 2 g/dL (performed within 7 days prior to enrollment)
Left ventricular ejection fraction (LVEF) >= 53% (or institutional normal; if the LVEF result is given as a range of values, then the upper value of the range will be used) by echocardiogram (performed within 4 weeks prior to enrollment)
Corrected QT (QTc) interval =< 450 msec by electrocardiography (EKG) (performed within 4 weeks prior to enrollment)
Absolute neutrophil count >= 1,000/uL (unsupported) (performed within 7 days prior to enrollment)
Platelets >= 100,000/uL (unsupported) (performed within 7 days prior to enrollment)
Hemoglobin >= 8 g/dL (may be supported) (performed within 7 days prior to enrollment)
Patients with a known seizure disorder should be stable and should not have experienced a significant increase in seizure frequency within 2 weeks prior to enrollment
Patients 2-17 years of age must have a blood pressure that is =< 95th percentile for age, height, and gender at the time of enrollment (with or without the use of anti-hypertensive medications)
Patients >= 18 years of age must have a blood pressure =< 130/80 mmHg at the time of enrollment (with or without the use of anti-hypertensive medications)
Note for patients of all ages: Adequate blood pressure can be achieved using medication for the treatment of hypertension
All patients must have ophthalmology toxicity assessments performed within 4 weeks prior to enrollment
For all patients, a magnetic resonance imaging (MRI) of the brain (with orbital cuts for optic pathway tumors) and/or spine (depending on the site(s) of primary disease) with and without contrast must be performed within 4 weeks prior to 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 the ability to swallow whole capsules
All patients have signed an appropriate consent form and Health Insurance Portability and Accountability Act (HIPAA) authorization form (if applicable)
All patients and/or their parents or legal guardians must sign a written informed consent
All patients have been consented and enrolled on APEC14B1 (NCT02402244) followed by enrollment on the ACNS1833 Pre-Enrollment Eligibility Screening (Step 0) on the same day to complete the Rapid Central Review
All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Exclusion Criteria:
Patients must not have received any prior tumor-directed therapy including chemotherapy, radiation therapy, immunotherapy, or bone marrow transplant. Prior surgical intervention is permitted
Patients with a concurrent malignancy or history of treatment (other than surgery) for another tumor within the last year are ineligible
Patients with diffuse intrinsic pontine tumors as seen on MRI (> 2/3 of pons involvement on imaging) are not eligible even if biopsy reveals grade I/II histology
Patients may not be receiving any other investigational agents
Patients with any serious medical or psychiatric illness/condition, including substance use disorders or ophthalmological conditions, likely in the judgment of the investigator to interfere or limit compliance with study requirements/treatment
Patients who, in the opinion of the investigator, are not able to comply with the study procedures are not eligible
Female patients who are pregnant are not eligible 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 are not eligible
Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation and for 12 weeks after stopping study therapy are not eligible.
Note: Women of child-bearing potential and males with sexual partners who are pregnant or who could become pregnant (i.e., women of child-bearing potential) should use effective methods of contraception for the duration of the study and for 12 weeks after stopping study therapy to avoid pregnancy and/or potential adverse effects on the developing embryo
Known genetic disorder that increases risk for coronary artery disease. Note: The presence of dyslipidemia in a family with a history of myocardial infarction is not in itself an exclusion unless there is a known genetic disorder documented
Symptomatic heart failure
New York Health Association (NYHA) class II-IV prior or current cardiomyopathy
Severe valvular heart disease
History of atrial fibrillation
Current or past history of central serous retinopathy
Current or past history of retinal vein occlusion or retinal detachment
Patients with uncontrolled glaucoma
If checking pressure is clinically indicated, patients with intraocular pressure (IOP) > 22 mmHg or ULN adjusted by age are not eligible
Supplementation with vitamin E greater than 100% of the daily recommended dose. Any multivitamin containing vitamin E must be stopped prior to study enrollment even if less than 100% of the daily recommended dosing for vitamin E
Surgery within 2 weeks prior to enrollment, with the exception of surgical biopsy, placement of a vascular access device or cerebral spinal fluid (CSF) diverting procedure such as endoscopic third ventriculostomy (ETV) and ventriculoperitoneal (VP) shunt.
Note: Patients must have healed from any prior surgery
Patients who have an uncontrolled infection are not eligibleA Randomized, Double-Blind, Placebo-Controlled Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Fragile X Syndrome - RECONNECT (RECONNECT)
The purpose of this study is to investigate how effective and safe ZYN002, a transdermal gel, is in participants with FXS. The drug product ZYN002 is a pharmaceutically manufactured CBD. It is being developed as a clear gel that can be applied to the skin (called transdermal delivery), to provide consistent, controlled levels of CBD in the blood when it is given twice a day. Participants will be assigned by chance to get one of the following study treatments: Active study drug – ZYN002 or placebo. Assigning study drug by chance is called “randomization,” and it is an important part of testing an experimental study drug. Participants will be randomly assigned to study treatment according to a computer program and will have 1 in 2 chance of receiving the active study drug.
Amy Esler
3 years to 23 years old
SITE00001338
Inclusion Criteria:
• ages 3 to less than 23 years
• resides with caregiver who will continue to provide consistent care throughout the study
• diagnosis of Fragile X Syndrome (FXS) through molecular documentation
• body mass index between 12-30 kg/m2
• in generally good health based upon the results of medical history, physical exam, 12-lead ECG and clinical laboratory test results
• contact study staff for additional requirements
Exclusion Criteria:
• women who are pregnant, nursing or planning a pregnancy
• has transitioned to independent living or living in a residential facility such as a university setting or congregate care
• use of cannabis or any THC or CBD-containing product within 3 months first study visit or during the study
• positive drug screen, including ethanol, cocaine, THC, barbiturates, amphetamines (unless prescribed), benzodiazepines (except midazolam or comparable administered for blood draws and ECG collection), and opiates
• additional medical or mental health diagnosis (study staff will review)
Rare Diseases
Fragile X Syndrome
MT2015-29 : Myeloablative Allogeneic Hematopoietic Cell Transplantation Using a Related or Adult Unrelated Donor for the Treatment of Hematological Disorders
Punita Grover
Up to 60 years old
STUDY00001087
-
Inclusion Criteria:
Age: ≤ 60 years of age
Performance Status: Karnofsky ≥ 70%, Lansky play score ≥ 70
Consent: Voluntary written consent (adult or legally authorized representative; or parental/guardian)
Adequate Organ Function:
Renal: Creatinine <2x upper limit of normal. Patients above this limit must have creatinine clearance ≥ 40 ml/min/1.73m2 as determined by an age-appropriate method, such as cystatin C GFR.
Hepatic: Bilirubin, AST, alkaline phosphatase <4 times the upper limit of institutional normal
Pulmonary: Diffusion capacity of oxygen, corrected for hemoglobin, > 50% of predicted. For pediatric patients not able to undergo PFTs or diffusion testing: O2 sat of >95% on room air
Cardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 45%. For children not able to cooperate with MUGA or echocardiography, such should be clearly stated in the physician's documentation
HIV Status: HIV infection with undetectable viral load. All HIV+ patients must be evaluated by Infectious Disease (ID) and a HIV management plan establish prior to transplantation
Other Inclusion Criteria:
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.
Donor Availability: Patients considered for transplantation must have a sufficient graft as based on current criteria of the University of Minnesota Blood and Marrow Transplantation Program
Eligible Diseases and Status: Patients are eligible unless their treatment is to be guided by a higher priority protocol.
Acute Leukemias: Must be in remission by morphology (≤5% blasts). Also a small percentage of blasts that is equivocal between marrow regeneration vs. early relapse are acceptable provided there are no associated cytogenetic markers consistent with relapse.
Acute Myeloid Leukemia (AML) and related precursor neoplasms: 2nd or greater complete remission (CR); first complete remission (CR1) in patients > 60 years old; CR1 in ≤ 60 years old that is NOT considered as favorable-risk.
Favorable risk AML is defined as having one of the following:
t(8,21) without cKIT mutation
inv(16) or t(16;16) without cKIT mutation
Normal karyotype with mutated NPM1 and wild type FLT-ITD
Normal karyotype with double mutated CEBPA
Acute prolymphocytic leukemia (APL) in first molecular remission at the end of consolidation
Very high risk pediatric patients with AML: Patients <21 years, however, are eligible with (M2 marrow) with < 25% blasts in marrow after having failed one or more cycles of chemotherapy.
Acute lymphoblastic leukemia (ALL)/lymphoma: second or greater CR; CR1 unable to tolerate consolidation chemotherapy due to chemotherapy-related toxicities; CR1 high-risk ALL.
High risk ALL is defined as having one of the following:
Evidence of high risk cytogenetics, e.g. t(9;22), t(1;19), t(4;11), other MLL rearrangements, IKZF1
30 years of age or older at diagnosis
White blood cell counts of greater than 30,000/mcL (B-ALL) or greater than 100,000/mcL (T-ALL) at diagnosis
CNS leukemia involvement during the course of disease
Slow cytologic response (>10% lymphoblasts in bone marrow on Day 14 of induction therapy)
Evidence of persistent immonophenotypic or molecular minimal residual disease (MRD) at the end of induction and consolidation therapy
Very high risk pediatric patients with ALL: patients <21 years are also considered high risk CR1 if they had M2 or M3 marrow at day 42 from the initiation of induction or M3 marrow at the end of induction. They are eligible once they achieve a complete remission.
Chronic Myelogenous Leukemia excluding refractory blast crisis: To be eligible in first chronic phase (CP1) patient must have failed or be intolerant to one or more tyrosine kinase inhibitors.
Plasma Cell Leukemia after initial therapy, in patients who have achieved at least a partial remission
Myeloproliferative Neoplasms/Myelofibrosis, either primary as a result of polycythemia vera or essential thrombocythemia, with disease risk of intermediate or high-risk according to DIPSS criteria. Blasts must be <10% by bone marrow aspirate morphology.
Myelodysplasia (MDS) IPSS INT-2 or High Risk (i.e. RAEB, RAEBt) or Refractory Anemia with severe pancytopenia, transfusion dependence, or high risk cytogenetics or molecular features. Blasts must be < 10% by a representative bone marrow aspirate morphology.
Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL), Marginal Zone B-Cell Lymphoma or Follicular Lymphoma are eligible if there was disease progression/relapse within 12 of achieving a partial or complete remission. Patients who had remissions lasting > 12 months, are eligible after at least two prior therapies. Patients with bulky disease (nodal mass greater than 5 cm) should be considered for debulking chemotherapy before transplant.
Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia are eligible after initial therapy in CR1+ or PR1+.
Diffuse large Cell NHL > CR/> PR: Patients in CR/PR with initial short remission (<6 months) are eligible, or those who have failed/or are not eligible for autologous transplant.
Lymphoblastic Lymphoma, Burkitt's Lymphoma, and other high-grade NHL after initial therapy if stage III/IV in CR1/PR1 or after progression if stage I/II < 1 year.
Multiple Myeloma beyond PR2: Patients with chromosome 13 abnormalities, first response lasting less than 6 months, or β-2 microglobulin > 3 mg/L, may be considered for this protocol after initial therapy.
Juvenile myelomonocytic leukemia
Biphenotypic/Undifferentiated/Prolymphocytic Leukemias in first or subsequent CR.
MRD positive leukemia (AML, ALL or accelerated/blast phase CML). Selected patients in morphologic CR, but with positive immunophenotypic (flow cytometry) or molecular evidence of MRD may be eligible if recent chemotherapy has not resulted in MRD negative status.
Natural Killer Cell Malignancies
Acquired Bone Marrow Failure Syndromes except for Fanconi Anemia or Dyskeratosis Congenita
Other Leukemia Subtypes: A major effort in the field of hematology is to identify patients who are of high risk for treatment failure so that patients can be appropriately stratified to either more (or less) intensive therapy. This effort is continually ongoing and retrospective studies identify new disease features or characteristics that are associated with treatment outcomes. Therefore, if new features are identified after the writing of this protocol, patients can be enrolled with the approval of two members of the study committee.
Exclusion Criteria:
Chemotherapy refractory large cell and high grade NHL (i.e., progressive disease after > 2 salvage regimens)
CML in blast crisis
Large cell lymphoma, mantle cell lymphoma and Hodgkin disease that is progressing on salvage therapy.
Evidence of progressive disease by imaging modalities or biopsy - persistent PET activity, though possibly related to lymphoma, is not an exclusion criterion in the absence of CT changes indicating progression.
Active central nervous system malignancy
if ≤ 18 years old, prior myeloablative transplant within the last 6 months. If >18 years old prior myeloablative allotransplant or autologous transplant
Active HIV infection or known HIV positive serology
active uncontrolled infection
Pregnant or breastfeeding. The agents used in this study include Pregnancy Category D: known to cause harm to a fetus. Females of childbearing potential must have a negative pregnancy test prior to starting therapy. Clinics and Surgery Center (CSC)
Tissue biopsies for the study of FSHD
A single visit study with muscle and/or skin biopsy / blood draw, performed to determine whether a molecular or cellular defect can be attributed to cells of Fascioscapulohumeral Muscular Dystrophy (FSHD) muscle. This study is recruiting both individuals with genetically confirmed FSHD as well as unaffected healthy (control) individuals.
Michael Kyba
Not specified
STUDY00000409
Inclusion Criteria:
• Genetic confirmation of Fascioscapulohumeral Muscular Dystrophy (FSHD)
• at least 4 years old
• Healthy Family Members: parent or sibling of someone who has FSHD
Exclusion Criteria:
• heart failure, respiratory insufficiency that requires respiratory support
• taking anticoagulants or anti platelet medications (aspirin or NSAIDs)
Rare Diseases, Heart & Vascular
Fascioscapulohumeral Muscular Dystrophy (FSHD), Clinics and Surgery Center (CSC)
Electronic Platform for Assessment of Adherence, Quality of Life, Clinical Response and Safety of Daily and Long‐Acting Growth Hormone Therapy (LAuGH TRACK UMN) (LAuGH TRACK)
The purpose of the study is to compare quality of life (QOL), adherence, insulin resistance, body composition and efficacy of LAGH to DGH in children with GHD.
Brad Miller, MD, PhD
Up to 18 years old
STUDY00011784
Inclusion Criteria:
• girls ages 2-11 years
• boys ages 2-13 years
• established diagnosis of pediatric growth hormone deficiency (GHD).
• For this study, GHD is defined as peak growth hormone response to clonidine/arginine stimulation testing of <10 ng/mL Either treatment-naive or currently treated with a daily growth hormone as approved by health insurance.
Exclusion Criteria:
• any medical condition which, in the opinion of the Investigator, can be an independent cause of short stature and/or limit the response to exogenous growth factor treatment
• current treatment with long-acting growth hormone
• currently pregnant or breastfeeding
Children's Health, Diabetes & Endocrine
Growth Hormone Deficiency
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
Not specified
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
• there are different requirements for each diagnosis category & study staff will review these
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
• history of heart failure or a previous hospital admission for fluid overload.
• history of liver disease
• hemoglobin below 9 g/dL or blood transfusion for anemia within the past 3 months.
• cancer in the past 5 years (except nonmelanoma skin cancer and curatively treated cervical carcinoma in situ)
• women who are pregnant, breastfeeding, or intend become pregnant during the study
• recently received an investigational agent -clinically significant unstable or uncontrolled medical condition (study staff will review)
Kidney, Prostate & Urinary
Glomerular Disease, Alport Syndrome, IgAN, FSGS, Proteinuric Glomerular Diseases
National Pediatric Cardiology Quality Improvement Collaborative (NPC-QIC) - A Collaborative Initiative to Improve Care of Children with Complex Congenital Heart Disease (NPC-QIC)
Kavisha Shah
Up to 18 years old
STUDY00004329
Inclusion Criteria:
• up to 15 months old
• newborns diagnosed with HLHS or other univentricular condition
• intended to undergo Norwood procedure
Children's Health, Heart & Vascular
Hypoplastic Left Heart Syndrome (HLHS)
Accelerating Therapies for Refractory SJIA Patients
This research study aims to learn more about the causes of Systemic Juvenile Idiopathic Arthritis (sJIA) including its complications such as Macrophage Activation Syndrome (MAS) and sJIA associated lung disease and identify new therapeutic targets. The study is asking for biological samples from those diagnosed with sJIA as well as their family members.
Mona Riskalla
Up to 18 years old
SITE00001576
Inclusion Criteria:
• at least 6 months old
• new onset SJIA or established SJIA with or without lung and/or liver complications
• parent or sibling of the enrolled child will also be asked to participate
Exclusion Criteria:
• illness sufficient to prohibit study participation
• inability to cooperate with the study
Arthritis & Rheumatic Diseases, Rare Diseases
SJIA, Systemic Juvenile Idiopathic Arthritis
Phase 1/2 Study to Evaluate Palbociclib (IBRANCE) in Combination With Irinotecan and Temozolomide or in Combination with Topotecan and Cyclophosphamide in Pediatric Patients With Recurrent or Refractory Solid Tumors Protocol No.: ADVL1921/A5481092
This is a Phase 1/2 multicenter, open-label study to evaluate palbociclib in combination with either irinotecan (IRN) and temozolomide (TMZ) or topotecan (TOPO) and cyclophosphamide (CTX) chemotherapy in children, adolescents and young adults with recurrent or refractory solid tumors. The study consists of a non- randomized Phase 1 portion for recurrent or refractory solid tumors followed by potential non- randomized tumor specific cohort(s) and a randomized, Phase 2 portion for recurrent or refractory EWS.
Emily Greengard
Not specified
STUDY00007068
Inclusion Criteria:
• 2 years to 20 years of age
• confirmed relapsed or refractory solid tumor (including CNS tumors but not lymphomas)
• recovered to CTCAE Grade 1 or less, or to baseline, from any non-hematological acute toxicities of prior surgery, chemotherapy, immunotherapy, radiotherapy, differentiation therapy or biologic therapy, with the exception of alopecia
• serum/urine pregnancy test (for all girls 8 or older) negative at screening and at the baseline visit
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
Exclusion Criteria:
• prior irradiation to >50% of the bone marrow
• major surgery within 4 weeks prior to study entry. Surgical biopsies or central line placement are not considered major surgeries
• patients with known symptomatic brain tumors or brain metastases and require steroids, unless they have been on a stable or on a decreasing steroid dose for >14 days
• fertile male patients or female patients of childbearing potential who are unwilling or unable to follow contraceptive requirements
• pregnant or breastfeeding women
• additional exclusion criteria apply (study staff will review)
Cancer
Ewing Sarcoma, Medulloblastoma, Neuroblastoma, Rhabdoid Tumor, Rhabdomyosarcoma, Solid Tumors
MT2022-45 Primary Immune Regulatory Disorders (PIRD): Longitudinal Study of Clinical Presentation, Treatment and Outcomes
The primary objective of this study is to estimate the overall survival (OS) of PIRD patients from the time of diagnosis. Patients may be treated with Best Available Therapy (BAT) or Hematopoietic Cell Transplant (HCT).
Christen Ebens
SITE00001766
Clinics and Surgery Center (CSC)
Udall P1A4
Through this research, the study staff hopes to better understand how DBS works and to define the optimal site in the brain for DBS treatment for Parkinson’s Disease. You will be asked to come for one study visit where you will perform some physical and mental tasks while on and temporarily off your medications and DBS treatment. Participation in this research study will take 7-8 hours.
Jerrold Vitek
Not specified
STUDY00019735
Inclusion Criteria:
• at least 10 years old
• diagnosis or suspected diagnosis of Parkinson's disease, Essential Tremor, or Dystonia
• implanted Deep Brain Stimulator (DBS)
• have a 7T MRI
Exclusion Criteria:
• history of dementia
• women who are pregnant or breastfeeding
• other exclusion criteria (study staff will review)
Brain & Nervous System
DBS, Dystonia, Essential Tremor, ET, Movement Disorders, Parkinson's, Parkinson's Disease, PD
AOST2031: A Phase 3 Randomized Controlled Trial Comparing Open vs Thoracoscopic Management of Pulmonary Metastases in Patients With Osteosarcoma
This phase III trial compares the effect of open thoracic surgery (thoracotomy) to thoracoscopic surgery (video-assisted thoracoscopic surgery or VATS) in treating patients with osteosarcoma that has spread to the lung (pulmonary metastases). Open thoracic surgery is a type of surgery done through a single larger incision (like a large cut) that goes between the ribs, opens up the chest, and removes the cancer. Thoracoscopy is a type of chest surgery where the doctor makes several small incisions and uses a small camera to help with removing the cancer. This trial is being done evaluate the two different surgery methods for patients with osteosarcoma that has spread to the lung to find out which is better.
Emily Greengard
Not specified
SITE00001674
Inclusion Criteria:
• 50 years of age or younger
• have 4 or less nodules in the lung due to metastases or suspected metastases
• diagnosis of osteosarcoma
• contact study team for more detailed criteria
Exclusion Criteria:
• pleural or mediastinal based metastatic lesions, or with pleural effusion
• large, or central tumors that require a lobectomy or pneumonectomy
Cancer
lung cancer, osteosarcoma, pulmonary metastases, thoracotomy
ITCC-101/APAL2020D - A randomized phase 3 trial of fludarabine/cytarabine/gemtuzumab ozogamicin with or without venetoclax in children with relapsed AML (A subtrial of the PedAL/EuPAL relapsed acute leukemia master protocol)
A study to evaluate if the randomized addition of venetoclax to a chemotherapy backbone (fludarabine/cytarabine/gemtuzumab ozogamicin [GO]) improves survival of children/adolescents/young adults with acute myeloid leukemia (AML) in 1st relapse who are unable to receive additional anthracyclines, or in 2nd relapse.
Peter Gordon
Not specified
SITE00001628
Inclusion Criteria:
• participants must be at least 29 days of age and less than 21 years of age at enrollment
• participants must have enrolled on APAL2020SC, NCT Number: NCT04726241
• children, adolescents, and young adults with acute myeloid leukemia without FLT3/internal tandem duplication (ITD) mutation
• second relapse who are sufficiently fit to undergo another round of intensive chemotherapy
• first relapse who per investigator discretion cannot tolerate additional anthracycline containing chemotherapy
• see link to clinicaltrials.gov for complete criteria
Exclusion Criteria:
• participants with Down syndrome
• participants with Acute promyelocytic leukemia (APL) or Juvenile myelomonocytic leukemia (JMML)
• study staff will review additional exclusion criteria
Cancer, Cancer
Acute Myeloid Leukemia, AML
Can spectral power and coherence reflect the integrity of the efferent cerebellar cortical pathway in cerebellar mutism syndrome?
This study will be measuring brain activity using EEG and assessing motor skills and speech in children following cancerous brain tumor resection. No direct cancer treatments or objectives are being targeted.
Sharyl Samagia-Grivette
Not specified
STUDY00019602
Inclusion Criteria:
• Cerebellar Mutism Syndrome (CMS) & Comparison (without CMS) Groups: ages 10 years 0 months to 25 years 11 months of age & fluent in English (parents/guardian do not need to be fluent in English)
• For those with Cerebellar Mutism Syndrome (CMS): history of resection of posterior fossa tumor at least 2 years before starting the study and at least 3 months post chemotherapy and radiation treatment
Exclusion Criteria:
• Comparison group without CMS: any developmental conditions including ADD/ADHD, learning disabilities, speech/language delay or disorder, motor delay/disorder, cognitive delay and/or diagnosis of autism spectrum disorder
• any genetic condition
• any neurologic condition including history of stroke, seizure disorder, or brain injury
• history of brain tumor or other cancer diagnosis
• CMS Group: any developmental conditions including ADD/ADHD, learning disabilities, speech/language delay or disorder, motor delay/disorder, cognitive delay and/or diagnosis of autism spectrum disorder prior to brain tumor diagnosis
• any genetic condition prior to brain tumor diagnosis
• any neurologic condition including history of stroke, seizure disorder, or brain injury disorder prior to brain tumor diagnosis
Brain & Nervous System, Cancer, Children's Health
brain tumor, cerebellar mutism syndrome (CMS)
MT2019-09: A randomized trial of low versus moderate exposure busulfan for infants with severe combined immunodeficiency (SCID) receiving TCR alpha beta +/CD19+ depleted transplantation: A Phase II study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediatric Blood and Marrow Transplant Consortium (PBMTC) PIDTC CSIDE Protocol (CSIDE)
To determine the incidence of humoral immune reconstitution by 2 years post-transplant in 2 SCID cohorts (IL2RG/JAK3, RAG1/RAG2) undergoing alternative donor HCT by randomized assignment to a busulfan preparative regimen targeted at cumulative area-under-the-curve (cAUC) exposure of 25-35 mg*h/L vs 55-65 mg*h/ L.
Christen Ebens
0 years to 2 years old
SITE00000541
Inclusion Criteria:
• Infants with SCID, either typical or leaky or Omenn syndrome. Typical SCID is defined as either of the following Absence or very low number of T cells (CD3+ T cells <300/microliter AND no or very low T cell function (<10% of lower limit of normal) as measured by response to phytohemagglutinin OR Presence of maternally derived T cells Leaky SCID is defined as the following • Absence of maternally derived T cells • AND either one or both of the following (i, ii): i) <50% of lower limit of normal T cell function as measured by response to PHA OR <30% of lower limit of normal T cell function as measured by response to CD3 ii) Absent or <10% of lower limit of normal proliferative responses to candida and tetanus toxoid antigens (must document post vaccination or exposure for this criterion to apply) • AND at least two of the following (i through iii): i) CD3 T cells < 1500/microliter ii) >80% of CD3+ or CD4+ T cells are CD45RO+ AND/OR >80% of CD3+ or CD4+ T cells are CD62L negative AND/OR >50% of CD3+ or CD4+ T cells express HLA-DR (at < 4 years of age) AND/OR are oligoclonal T iii) Low TRECs and/or the percentage of CD4+/45RA+/CD31+ or CD4+/45RA+/CD62L+ cells is below the lower level of normal. Omenn syndrome • Generalized skin rash Maternal lymphocytes tested for and not detected. >80% of CD3+ or CD4+ T cells are CD45RO+ AND/OR >80% of CD3+ or CD4+ T cells are CD62L negative AND/OR >50% of CD3+ or CD4+ T cells express HLA-DR (<2 years of age) Absent or low (up to 30% lower limit of normal (LLN)) T cell proliferation to antigens (Candida, tetanus) to which the patient has been exposed IF: Proliferation to antigen was not performed, but at least 4 of the following 8 supportive criteria, at least one of which must be among those marked with an asterisk (*) below are present, the patient is eligible as Omenn Syndrome. Hepatomegaly Splenomegaly Lymphadenopathy Elevated IgE Elevated absolute eosinophil count *Oligoclonal T cells measured by CDR3 length or flow cytometry (upload report) *Proliferation to PHA is reduced to < 50% of lower limit of normal (LLN) or SI < 30 *Low TRECs and/or percentage of CD4+/RA+ CD31+ or CD4+/RA+ CD62L+ cells below the lower level of normal
• Documented mutation in one of the following SCID-related genes a. Cytokine receptor defects (IL2RG, JAK3) b. T cell receptor rearrangement defects (RAG1, RAG2) 3. No available genotypically matched related donor (sibling) 4. Availability of a suitable donor and graft source Haploidentical related mobilized peripheral blood cells 9/10 or 10/10 allele matched (HLA-A, -B, -C, -DRB1, -DQB1) volunteer unrelated donor mobilized peripheral blood cells 5. Age 0 to 2 years at enrollment Note: to ensure appropriate hepatic metabolism, age at time of busulfan start: For IL2RG/JAK3: 8 weeks For RAG1/RAG2: 12 weeks
• Adequate organ function defined as: Cardiac: Left ventricular ejection fraction (LVEF) at rest ≥ 40% or, shortening fraction (SF) ≥ 26% by echocardiogram. Hepatic: Total bilirubin < 3.0 x the upper limit of normal (ULN) for age (patients who have been diagnosed with Gilbert's Disease are allowed to exceed this limit) and AST and ALT < 5.0 x ULN for age. Renal: GFR estimated by the updated Schwartz formula ≥ 90 mL/min/1.73 m2. If the estimated GFR is < 90 mL/min/1.73 m2, then renal function must be measured by 24-hour creatinine clearance or nuclear GFR, and must be > 50 mL/min/1.73 m2. Pulmonary No need for supplemental oxygen and O2 saturation > 92% on room air at sea level (with lower levels allowed at higher elevations per established center standard of care).
Exclusion Criteria:
Presence of any serious life-threatening or opportunistic infection at time of enrollment and prior to the initiation of the preparative regimen. Serious infections as defined below that occur after enrollment must be reported immediately to the Study Coordinating Center, and enrollment will be put on hold until the infection resolves. Ideally enrolled subjects will not have had any infection. If patients have experienced infections, these must have resolved by the following definitions:
a. Bacterial i. Positive culture from a sterile site (e.g. blood, CSF, etc.): Repeat culture(s) from same site must be negative and patient has completed appropriate course of antibacterial therapy (typically at least 10 days).
ii. Tissue-based clinical infection (e.g. cellulitis): Complete resolution of clinical signs (e.g. erythema, tenderness, etc.) and patient has completed appropriate course of antibacterial therapy (typically at least 10 days).
iii. Pneumonia, organism not identified by bronchoalveolar lavage: Complete resolution of clinical signs (e.g. tachypnea, oxygen requirement, etc.) and patient has completed appropriate course of antibacterial therapy (typically at least 10 days). If possible, radiographic resolution should also be demonstrated.
b. Fungal i. Positive culture from a sterile site (e.g. blood, CSF, etc.): Repeat culture(s) from same site is negative and patient has completed appropriate course of antifungal therapy (typically at least 14 days). The patient may be continued on antifungal prophylaxis following completion of the treatment course.
c. Pneumocystis i. Complete resolution of clinical signs (e.g. tachypnea, oxygen requirement, etc.) and patient has completed appropriate course of therapy (typically at least 21 days). If possible, radiographic resolution should also be demonstrated. The patient may be continued on prophylaxis following completion of the treatment course.
d. Viral i. Viral PCRs from previously documented sites (blood, nasopharynx, CSF) must be re-tested and are negative.
ii. If re-sampling a site is not clinically feasible (i.e. BAL fluid): Complete resolution of clinical signs (e.g. tachypnea, oxygen requirement, etc.). If possible, radiographic resolution should also be demonstrated.
Patients with HIV or HTLV I/II infection will be excluded.A Natural History Study of the Gangliosidoses
This is a research study documenting the natural history of disease in patients with GM1 or GM2 gangliosidosis. The information collected will be a way to evaluate disease progression and create a disease stage and severity index. Our goal is to use the data collected to measure the effectiveness of any treatments that are developed in the future.
Jeanine Jarnes
Not specified
1007M85712
Inclusion Criteria:
• documented gangliosidosis disease
• able to complete neuropsychological and neurobehavioral assessments
• Late-onset gangliosidosis subjects must be able to tolerate MRI of the head
Exclusion Criteria:
• none
Rare Diseases
GM1 Gangliosidosis, GM2 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease
A Phase 1b/2 Study of Abemaciclib in Combination with Irinotecan and Temozolomide (Part A) and Abemaciclib in Combination with Temozolomide (Part B) in Pediatric and Young Adult Patients with Relapsed/Refractory Solid Tumors and Abemaciclib in Combination with Dinutuximab, GM-CSF, Irinotecan, and Temozolomide in Pediatric and Young Adult Patients with Relapsed/Refractory Neuroblastoma (Part C). Protocol Number: I3Y-MC-JPCS
The study's purpose is to see if the drug abemaciclib is safe and effective in combination with temozolomide and irinotecan (Part A) and abemaciclib in combination with temozolomide (Part B) in pediatric and young adult participants with relapsed/refractory solid tumor and abemaciclib in Combination with Dinutuximab, GM-CSF, Irinotecan, and Temozolomide in Pediatric and Young Adult Patients with Relapsed/Refractory Neuroblastoma (Part C).
Emily Greengard
Not specified
STUDY00013998
Inclusion Criteria:
• Parts A and B only: participants must be less than or equal to 18 years of age and with any relapsed/refractory malignant solid tumor (excluding lymphoma), including central nervous system tumors, that have progressed on standard therapies
• Part C only: participants must be less than (<) 21 years of age and with first relapse/refractory neuroblastoma and with any relapsed/refractory malignant solid tumor (excluding lymphoma), including central nervous system tumors, that have progressed on standard therapies Parts A, B & C:
• participants must have discontinued all previous treatments for cancer or investigational agents and must have recovered from the acute effects to Grade = < 1 at the time of enrollment
• able to swallow and/or have a gastric/nasogastric tube
Exclusion Criteria:
• received allogenic bone marrow or solid organ transplant
• diagnosed and/or treated additional malignancy within 3 years prior to enrollment that may affect the interpretation of results, with the exception of curatively treated basal cell carcinoma of the skin, squamous cell carcinoma of the skin, and/or curatively resected in situ cervical and/or breast cancers
• pregnant or breastfeeding
Cancer
Refractory Solid Tumor, Relapsed Solid Tumor
A Phase II, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients with Primary Steroid-Resistant Focal Segmental Glomerulosclerosis
The main purpose of this study is to check how safe the study drug is and how well your body handles taking it. We will also check if the study drug works to improve your kidney function, if has an impact on your daily life and the amount of the study drug in your blood over a period of time (called pharmacokinetics)
Michelle Rheault
Not specified
STUDY00015869
Inclusion Criteria:
• at least 12 years of age
• for people with Alport Syndrome: confirmed diagnosis by genetic testing and /or kidney biopsy
• for primary Focal Segmental Glomerulosclerosis (FSGS), (without any identifiable cause, and where the FSGS is confirmed by renal biopsy) or FSGS where there is documentation of a genetic mutation in a podocyte protein
• female patients, as well as, female partners of male patients who are of child-bearing potential must be willing to not become pregnant for the complete duration of the study (90 days after the last dose of study medication)
• males (including sterilized subjects) whose female partners have child-bearing potential, must agree to use male contraception (condoms) during the period from the time of signing the informed consent form (ICF) through 90 days after the last dose of study drug
• contact study staff for additional criteria
Exclusion Criteria:
• uncontrolled diabetes mellitus as evidenced by an HbA1c greater or equal to 11%
• uncontrolled high blood pressure
• moderate or severe liver impairment
• BMI greater than 40
• women who are pregnant or breast feeding
• additional exclusion criteria apply (study staff will review)
Kidney, Prostate & Urinary
Clinics and Surgery Center (CSC), Alport Syndrome, Focal Segmental Glomerulosclerosis
PEPN2113: A Phase 1 and pharmacokinetic study of Uproleselan (GMI-1271, IND #139758, NSC #801708) in combination with fludarabine and cytarabine for patients with acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that expresses E-selectin ligand on the cell membrane and is in second or greater relapse or that is refractory to relapse therapy
A Phase 1 and pharmacokinetic study of Uproleselan (GMI-1271, IND #139758, NSC #801708) in combination with fludarabine and cytarabine for patients with acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that expresses E-selectin ligand on the cell membrane and is in second or greater relapse or that is refractory to relapse therapy
Emily Greengard
Up to 18 years old
SITE00001697
Inclusion Criteria:
• patient must be enrolled on APAL2020SC (NCT04726241)
• patients must be between 1 and 17 years of age at the time of study enrollment
• patients, with or without Down syndrome (DS), and with de novo acute myeloid leukemia, therapy-related acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that expresses E-selectin ligand on the cell membrane
• second or greater relapse or refractory AML OR refractory myelodysplastic syndrome (MDS) OR mixed phenotype acute leukemia (MPAL)
• see link to clinicaltrials.gov for complete criteria
Exclusion Criteria:
• patients who are currently receiving another investigational drug are not eligible
• patients who are currently receiving other anti-cancer agents are not eligible except patients receiving hydroxyurea, which may be continued until 24 hours prior to start of protocol therapy
• study staff will review additional exclusion criteria
Cancer, Cancer
AML, Myelodysplastic Syndrome Post Cytotoxic Therapy, Recurrent Acute Myeloid Leukemia