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The Up-LIFT Study of Non-Invasive ARC Therapy for Spinal Cord Injury (Up-LIFT)
To provide pilot evidence to the FDA for the LIFT System (a transcutaneous spinal cord stimulator) that the system is safe, effective, and evaluate potential benefits. This is a multisite industry sponsored study (GTX Medical, Inc., Lexington, MA).
All
22 Years to 75 Years old
N/A
NCT04697472
Key
• At least 22 years old and no older than 75 years old at the time of enrollment
• Non-progressive cervical spinal cord injury from C2-C8 inclusive
• American Spinal Injury Association (ASIA) Impairment Scale (AIS) classification B, C, or D
• Indicated for upper extremity training procedures by subject's treating physician or a physical therapist
• Minimum 12 months post-injury
• Capable of providing informed consent Key
• Has uncontrolled cardiopulmonary disease or cardiac symptoms as determined by the Investigator
• Has any unstable or significant medical condition that is likely to interfere with study procedures or likely to confound study endpoint evaluations like severe neuropathic pain, depression, mood disorders or other cognitive disorders
• Has been diagnosed with autonomic dysreflexia that is severe, unstable, and uncontrolled
• Requires ventilator support
• Has an autoimmune etiology of spinal cord dysfunction/injury
• Spasms that limit the ability of the subjects to participate in the study training as determined by the Investigator
• Breakdown in skin area that will come into contact with electrodes
• Has any active implanted medical device
• Pregnant, planning to become pregnant or currently breastfeeding
• Concurrent participation in another drug or device trial that may interfere with this study
• In the opinion of the investigators, the study is not safe or appropriate for the participant
Inclusion Criteria:
Subjects must meet all the following criteria:
• At least 22 years old and no older than 75 years old at the time of enrollment
• Non-progressive cervical spinal cord injury from C2-C8 inclusive
• American Spinal Injury Association (ASIA) Impairment Scale (AIS) classification B, C, or D
• Indicated for upper extremity training procedures by subject's treating physician or a physical therapist
• Minimum 12 months post-injury
• Capable of providing informed consent Key
Exclusion Criteria:
Subjects must not meet any of the following criteria:
• Has uncontrolled cardiopulmonary disease or cardiac symptoms as determined by the Investigator
• Has any unstable or significant medical condition that is likely to interfere with study procedures or likely to confound study endpoint evaluations like severe neuropathic pain, depression, mood disorders or other cognitive disorders
• Has been diagnosed with autonomic dysreflexia that is severe, unstable, and uncontrolled
• Requires ventilator support
• Has an autoimmune etiology of spinal cord dysfunction/injury
• Spasms that limit the ability of the subjects to participate in the study training as determined by the Investigator
• Breakdown in skin area that will come into contact with electrodes
• Has any active implanted medical device
• Pregnant, planning to become pregnant or currently breastfeeding
• Concurrent participation in another drug or device trial that may interfere with this study
• In the opinion of the investigators, the study is not safe or appropriate for the participant
Device: LIFT System
Chronic Spinal Cord Injury
SCI, Tetraplegia, ARC Therapy
A Multicenter Observational Study of GammaTile Surgically Targeted Radiation Therapy (STaRT) in Intracranial Brain Neoplasms
The primary objectives of this prospective non-interventional (NI) study are to evaluate real-world clinical outcomes and patient reported outcomes (PRO) that measure the effectiveness and safety of GammaTilesTM (GT). Data will be used to 1) benchmark clinical outcomes of GT therapy, 2) allow for comparisons of these outcomes to published clinical outcomes in the target population treated with standard of care (SOC) treatment(s), and (3) test for non-inferiority of surgical bed recurrence-free survival to current identified SOC for recurrent metastatic tumors and recurrent meningiomas as well as test for non-inferiority of mean overall survival for recurrent high grade gliomas.
Clark Chen
All
Not specified
NA
NCT04427384
STUDY00010689
Inclusion Criteria:
• Patients who undergo maximum safe resection of intracranial neoplasm(s) AND implantation of GammaTiles.
• Willing and able to provide informed consent and to participate in all evaluations.
Exclusion Criteria:
• Inability to undergo pre-operative and post-operative imaging for disease and implant assessment.
• Major medical or psychiatric illness, which, in the investigator's opinion would prevent completion of treatment, ability to complete assessments at the time of enrollment, and/or interfere with follow ups.
• Lack of English language fluency sufficient to allow for completion of neurocognitive and QOL tests (which are in English).
Device: GammaTile
Brain Tumor, Recurrent, Brain Tumor, Brain Tumor, Primary, Brain Tumor - Metastatic, Brain Tumor, Adult: Glioblastoma, Brain Tumor, Adult Meningioma
PRescribing INterventions for Chronic Pain Via the Electronic Health Record Study - Primary Care Providers (PRINCE)
All
Not specified
N/A
NCT04601506
Inclusion Criteria:
• All primary care providers from all of the Fairview and University of Minnesota Physicians study clinics
Exclusion Criteria:
• Primary care providers who work less than 20% full time equivalent (FTE)
Behavioral: Choice Architecture Nudge, Behavioral: PMP Integration & Nudge
Opioid-use Disorder, Opioid Use, Opioid Abuse
PRescribing INterventions for Chronic Pain Via the Electronic Health Record Study - Opioid-Naive Population (PRINCE)
All
Not specified
N/A
NCT04601493
Inclusion Criteria:
• All primary care providers from all of the Fairview and University of Minnesota Physicians study clinics
Exclusion Criteria:
• Primary care providers who work less than 20% full time equivalent (FTE)
Behavioral: Choice Architecture Nudge, Behavioral: PMP Integration & Nudge
Opioid-use Disorder, Opioid Use, Opioid Abuse
Extension Study to Evaluate Safety and Efficacy of CTP-543 in Adults With Alopecia Areata
All
18 Years to 65 Years old
Phase 2/Phase 3
NCT03898479
Inclusion Criteria:
• Have completed 24 weeks of treatment in a previous qualifying CTP-543 clinical trial
Exclusion Criteria:
• Active scalp inflammation, psoriasis, or seborrheic dermatitis requiring topical treatment to the scalp, significant trauma to the scalp, or untreated actinic keratosis
• Females who are nursing, pregnant, or planning to become pregnant while in the study, and for 30 days after last dose of study medication
• Donation of blood at any point throughout the study and for 30 days after last dose of study medication
Drug: CTP-543
Alopecia Areata
Phase II Study of Low-Dose Ibuprofen for Cognitive Impairment in Cancer Patients Receiving Chemotherapy
Phase II
NCT03186638
0123456789
Cognitive Impairment, Malignant Neoplasm
An Open-label, Phase 2 Basket Study of SEA-CD40 Combination Therapies in Advanced Malignancies
This is a phase 2, global, open-label, multicenter trial designed to assess the activity, safety, and tolerability of SEA-CD40 in combination with standard-of-care therapies in adults with selected solid tumors. The study will include multiple indication-specific cohorts. Up to approximately 200 subjects may be enrolled in this study.
Evidio Domingo Musibay
All
18 Years and over
Phase 2
NCT04993677
STUDY00014590
Inclusion Criteria:
• Histologically or cytologically confirmed unresectable malignancy defined as one of the following:
• Cohort 1: Relapsed and/or refractory metastatic melanoma
• Uveal/ocular melanoma is excluded
• Must have progressed on treatment with an anti-PD-(L)1 mAb. PD-(L)1 treatment progression is defined as meeting all of the following criteria:
• Has received at least 2 doses of an approved anti-PD-(L)1 mAb
• Has demonstrated disease progression after PD-(L)1 as defined by RECIST v1.1.
• Progressive disease has been documented within 12 weeks from the last dose of anti- PD-(L)1 mAb
• Last dose of anti-PD-(L)1 must have been within 90 days prior to enrollment
• Participants with a targetable BRAF mutation must have been treated with, been intolerant of, or declined treatment with BRAF/MEK targeted therapy prior to study entry
• Cohort 2: Metastatic uveal melanoma
• Must not have received prior treatment for advanced or metastatic disease except for prior adjuvant/neoadjuvant immunotherapy
• No prior liver-directed therapy
• Cohort 3: Metastatic PD-(L)1-naive melanoma
• Uveal/ocular melanoma is excluded
• Must not have received prior treatment for advanced or metastatic disease except for prior adjuvant/neoadjuvant immunotherapy.
• For participants with a targetable BRAF mutation, prior BRAF/MEK targeted therapy is allowed if completed 4 weeks prior to first dose of study treatment.
• Cohorts 4 and 5: Non-squamous NSCLC
• Participants must have stage IV disease per AJCC 8th edition
• No known driver mutations/alterations mutation for which targeted therapy is available
• Must have non-squamous histology.
• No prior therapy for metastatic disease
• No prior treatment with anti-PD-(L)1 or PD-L2 agent or an antibody targeting other immuno-regulatory receptors or mechanisms
• Able to provide archival tumor tissue from locations not radiated prior to biopsy. If archival tumor sample is not available a fresh baseline biopsy is required.
• Eastern Cooperative Oncology Group (ECOG) Performance Status score of 0 or 1
• Measurable disease per RECIST v1.1 at baseline
Exclusion Criteria:
• History of another malignancy within 3 years of first dose of study drug
• Active central nervous system (CNS) metastases and/or carcinomatous meningitis.
• Previous exposure to CD40-targeted therapy
• Currently on chronic systemic steroids in excess of physiologic replacement
• Has had an allogeneic tissue/solid organ transplant.
• History of autoimmune disease that has required systemic treatment in the past 2 years
Drug: SEA-CD40, Drug: pembrolizumab, Drug: pemetrexed, Drug: carboplatin
Melanoma, Carcinoma, Non-Small- Cell Lung
Relapsed melanoma, Refractory melanoma, Metastatic uveal melanoma, Metastatic PD-(L)1-naïve melanoma, Non-squamous NSCLC, NSCLC, Non-small cell lung cancer, Seattle Genetics, Clinics and Surgery Center (CSC), Phase I Clinic
Post Approval Study Investigating Lutonix Drug Coated Balloon for Treatment of Dysfunctional Arteriovenous Fistulae (AVPAS)
This post-market approval study (PAS) is a prospective, multi-center, single-arm study intended to demonstrate safety and assess the clinical use and outcomes of the LUTONIX® Catheter in dysfunctional arteriovenous fistulae (AVF) in a heterogeneous patient population in real world clinical practice. The study will include approximately 213 subjects across 30 U.S. and international sites. The LUTONIX® Catheter was approved by FDA on August 25, 2017 (PMA P170003) for the indication of percutaneous transluminal angioplasty (PTA), after pre-dilatation, for the treatment of stenotic lesions in dysfunctional arteriovenous dialysis fistulae that are 4 mm to 12 mm in diameter and up to 80 mm in length. The study involves no intervention and all medical procedures are standard of care and not research. Follow-up information will be obtained at 6, 12, 18, and 24 months after the index procedure, and vital status will be assessed at 36, 48, and 60 months.
All
18 Years and over
N/A
NCT03506308
Inclusion Criteria:
• Male or non-pregnant, non-breastfeeding female ≥18 years of age
• Subject is willing to provide informed consent, and is willing to comply with the protocol-required follow up visits
• Target lesion must be a mature arteriovenous fistula located in the arm presenting with any clinical, physiological or hemodynamic abnormalities warranting angiographic imaging as defined in the K/DOQI guidelines
• Subject has a target lesion that can be treated with available LUTONIX DCB according to the Instructions For Use (IFU)
• Venous stenosis of an AV fistula in which the target lesion is located from the anastomosis to the axillosubclavian junction, as defined by insertion of the cephalic vein
• Successful pre-dilation of the target lesion with an uncoated percutaneous transluminal angioplasty (PTA) balloon defined as:
• No clinically significant dissection;
• No extravasation requiring treatment;
• Residual stenosis ≤30% by angiographic measurement;
• Ability to completely efface the waist using the pre-dilation balloon.
Exclusion Criteria:
• Subject is currently participating in an investigational drug, biologic, or device study, or previous enrollment in this study
• Subject has a non-controllable allergy to contrast
• Subject has another medical condition that, in the opinion of the Investigator, may confound the data interpretation or is associated with a life expectancy insufficient to allow for completion of subject study procedure and follow up
• Target lesion is located central to the axillosubclavian junction
• A thrombosed access or an access with a thrombosis treated ≤7 days before to the index procedure
• Prior surgical interventions of the access site ≤30 days before the index procedure
• Target lesion is located within a bare metal or covered stent
Device: LUTONIX 035 Drug Coated Balloon PTA Catheter
Arteriovenous Fistula
A PROSPECTIVE OBSERVATIONAL STUDY OF SOLID ORGAN TRANSPLANTATION UTILIZING HIV-POSITIVE DONORS IN HIV-POSITIVE RECIPIENTS
This is a prospective, observational study designed to evaluate the safety of solid organ transplantation using HIV-positive deceased donors (liver, kidney) and HIV-positive living donors (liver) in HIV-positive recipients.
Timothy Pruett
All
18 Years and over
N/A
NCT03170414
1608M93841
RECIPIENT ELIGIBILITY CRITERIA HIV-Positive Recipient Inclusion Criteria (liver, kidney)
• Participant is able to understand and provide informed consent.
• Participant meets standard listing criteria for transplant.
• Documented HIV infection (by any licensed ELISA and confirmation by Western Blot, positive HIV ab IFA, or documented history of detectable HIV-1 RNA).
• Participant is ≥ 18 years old.
• No evidence of active opportunistic complications of HIV infection.
• Participant CD4+ T-cell count is >/= 200/µL within 16 weeks prior to transplant for kidney transplant recipients. For liver transplant recipient, CD4+ T-cell counts need to be >/= 100/ul (or >/= 200/µL if history of opportunistic infection) within 16 weeks prior to transplant.
• Participant most recent HIV-1 RNA < 50 copies/mL (by any FDA-approved assay performed in CLIA-approved laboratory), in the 26 weeks prior to transplant. Participants unable to tolerate ART due to organ failure or who have only recently started ART may have detectable viral load and still be considered eligible if the study team is confident there will be a safe, tolerable, and effective antiretroviral regimen once organ function is restored after transplantation.
• Concurrence by the study team that based on medical history and ART, viral suppression can be achieved in the recipient post-transplant.
• No history of primary CNS lymphoma or progressive PML.
• On a stable antiretroviral regimen. Participants unable to tolerate ART due to organ failure may still be considered eligible if the study team is confident there will be a safe, tolerable, and effective antiretroviral regimen once organ function is restored after transplantation. HIV-Positive Recipient Exclusion Criteria (liver, kidney)
• Participant has concomitant conditions that, in the judgment of the investigators, would preclude transplantation or immunosuppression. DONOR ELIGIBILITY CRITERIA HIV-Positive Deceased Donor (liver, kidney)
• Must meet all clinical criteria for HIV-uninfected organ donors.
• No evidence of invasive opportunistic complications of HIV infection.
• Pre-implant donor organ biopsy showing no disease process that would put the recipient at increased risk of rapid progression to end-stage organ failure, to be stored for the duration of the study.
• Donor has documented HIV infection (by any licensed ELISA and confirmation by Western Blot, positive HIV ab IFA, or history of detectable HIV-1 RNA) from a CLIA-approved laboratory.
• If known history of HIV infection and prior antiretroviral therapy, the study team must describe the anticipated post-transplant antiretroviral regimen to be prescribed for the recipient and justify its conclusion that the regimen will be safe, tolerable and effective. HIV-Positive Living Donor (liver)
• Donor meets all clinical criteria to be a living liver donor other than being HIV positive.
• Donor has documented HIV infection (by any licensed ELISA and confirmation by Western Blot, positive HIV ab IFA, or history of detectable HIV-1 RNA) from a CLIA-approved laboratory.
• No evidence of invasive opportunistic complications of HIV infection
• Donor CD4+ T-cell count is >/= 500/µL in the 26 weeks prior to donation.
• The most recent HIV-1 RNA has been below 50 copies RNA/ml in the 26 weeks prior to donation.
• On a stable antiretroviral regimen.
• Must be evaluated by the HIV/Transplant Infectious Diseases team to verify resistance history and current ART regimens. The potential for transmission of resistant strain of HIV will be assessed.
• Pre-implant donor liver biopsy to be stored for the duration of the study showing no evidence of a disease process that would put the donor at increased risk of progressing to end-stage organ failure after donation, or that would present a risk of poor graft function to the recipient.
• Must be evaluated by an independent HIV study living donor advocate separate from the transplant service in addition to the living donor advocate seen by all living donors.
• Participant is able to understand and provide informed consent.
• Participant meets standard listing criteria for transplant.
• Documented HIV infection (by any licensed ELISA and confirmation by Western Blot, positive HIV ab IFA, or documented history of detectable HIV-1 RNA).
• Participant is ≥ 18 years old.
• No evidence of active opportunistic complications of HIV infection.
• Participant CD4+ T-cell count is >/= 200/µL within 16 weeks prior to transplant for kidney transplant recipients. For liver transplant recipient, CD4+ T-cell counts need to be >/= 100/ul (or >/= 200/µL if history of opportunistic infection) within 16 weeks prior to transplant.
• Participant most recent HIV-1 RNA < 50 copies/mL (by any FDA-approved assay performed in CLIA-approved laboratory), in the 26 weeks prior to transplant. Participants unable to tolerate ART due to organ failure or who have only recently started ART may have detectable viral load and still be considered eligible if the study team is confident there will be a safe, tolerable, and effective antiretroviral regimen once organ function is restored after transplantation.
• Concurrence by the study team that based on medical history and ART, viral suppression can be achieved in the recipient post-transplant.
• No history of primary CNS lymphoma or progressive PML.
• On a stable antiretroviral regimen. Participants unable to tolerate ART due to organ failure may still be considered eligible if the study team is confident there will be a safe, tolerable, and effective antiretroviral regimen once organ function is restored after transplantation. HIV-Positive Recipient Exclusion Criteria (liver, kidney)
• Participant has concomitant conditions that, in the judgment of the investigators, would preclude transplantation or immunosuppression. DONOR ELIGIBILITY CRITERIA HIV-Positive Deceased Donor (liver, kidney)
• Must meet all clinical criteria for HIV-uninfected organ donors.
• No evidence of invasive opportunistic complications of HIV infection.
• Pre-implant donor organ biopsy showing no disease process that would put the recipient at increased risk of rapid progression to end-stage organ failure, to be stored for the duration of the study.
• Donor has documented HIV infection (by any licensed ELISA and confirmation by Western Blot, positive HIV ab IFA, or history of detectable HIV-1 RNA) from a CLIA-approved laboratory.
• If known history of HIV infection and prior antiretroviral therapy, the study team must describe the anticipated post-transplant antiretroviral regimen to be prescribed for the recipient and justify its conclusion that the regimen will be safe, tolerable and effective. HIV-Positive Living Donor (liver)
• Donor meets all clinical criteria to be a living liver donor other than being HIV positive.
• Donor has documented HIV infection (by any licensed ELISA and confirmation by Western Blot, positive HIV ab IFA, or history of detectable HIV-1 RNA) from a CLIA-approved laboratory.
• No evidence of invasive opportunistic complications of HIV infection
• Donor CD4+ T-cell count is >/= 500/µL in the 26 weeks prior to donation.
• The most recent HIV-1 RNA has been below 50 copies RNA/ml in the 26 weeks prior to donation.
• On a stable antiretroviral regimen.
• Must be evaluated by the HIV/Transplant Infectious Diseases team to verify resistance history and current ART regimens. The potential for transmission of resistant strain of HIV will be assessed.
• Pre-implant donor liver biopsy to be stored for the duration of the study showing no evidence of a disease process that would put the donor at increased risk of progressing to end-stage organ failure after donation, or that would present a risk of poor graft function to the recipient.
• Must be evaluated by an independent HIV study living donor advocate separate from the transplant service in addition to the living donor advocate seen by all living donors.
HIV, Awaiting Organ Transplant
HIV, Kidney Transplant, Liver Transplant, Transplant, HOPE Act, Clinics and Surgery Center (CSC)
A 5-year Longitudinal Observational Study of Patients Undergoing Therapy for Inflammatory Bowel Disease (TARGET-IBD)
This is a 5-year, longitudinal, observational study of adult and pediatric patients (age 2 and above) undergoing IBD therapy designed to specifically address important clinical questions that remain incompletely answered from registration trials. Patients being prescribed medical therapy for IBD outside of a clinical trial will be eligible for enrollment. Treatment algorithms will follow each site’s local standard of care and no specific treatments, assessments, and or laboratory tests will be dictated by enrollment in TARGET-IBD. Enrolled patients will consent to the possibility of up to 3 years of retrospective, redacted medical record collection as well as prospective collection for up to 5 years. Medical records will include but will not be limited to: records of hospitalizations, laboratory reports, clinic notes, telephone contact reports, medication lists, reasons for medication initiation and/or discontinuation, endoscopy reports, biopsy results, and imaging results. Patients will also be asked to provide a blood sample for biomarker, anti-drug antibody, and DNA assays and complete patient reported outcome (PRO) surveys, although participation in these two portions of the study is not mandatory for study participation. Consent for linkage of patient health information (PHI) to external healthcare databases (such as patient support programs) will also be requested as an optional portion of the study.
Boris Sudel
All
2 Years and over
NA
NCT03251118
STUDY00005662
Inclusion Criteria:
• Adults and children (age 2 or older) with a diagnosis of Crohn's disease (CD), Ulcerative colitis (UC), or Indeterminate colitis (IBDU) having been prescribed any IBD treatment (initial or subsequent) outside of a clinical trial.
• Have plans for future visits at the site for continued management of IBD.
Exclusion Criteria:
• Inability to provide written informed consent/assent.
• Being enrolled in any interventional study or trial for IBD treatment. Note: Patient may be enrolled in other registries or studies where IBD treatment outcomes are observed and/or reported (such as center-based registries).
• Prior total abdominal colectomy for UC or IBDU.
Inflammatory Bowel Diseases, Crohn's Disease, Ulcerative Colitis, Indeterminate Colitis
Crohn's Disease, Ulcerative colitis, Indeterminate colitis, Digestive System Diseases
A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma
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
All
2 Years to 21 Years old
Phase 3
NCT04166409
STUDY00009277
Inclusion Criteria:
• 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 7 days prior to enrollment)
• Corrected QT (QTc) interval =< 450 msec by electrocardiography (EKG) (performed within 7 days 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)
• All patients must have ophthalmology toxicity assessments performed within 4 weeks prior to enrollment
• 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
• 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 eligible
Drug: Carboplatin, Other: Quality-of-Life Assessment, Other: Questionnaire Administration, Drug: Selumetinib Sulfate, Drug: Vincristine Sulfate
Low Grade Astrocytoma, Low Grade Glioma, Metastatic Low Grade Astrocytoma, Metastatic Low Grade Glioma
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Subjects With GM1 Gangliosidosis (Imagine-1)
This study is a prospective multi-cohort, open-label, dose-escalation assessment of the safety and efficayc of PBGM01, an AAVHu68, intra-cisternal magna delivered gene therapy for the treatment of GM1 gangliosidosis in infants 1-24 months of age.
All
4 Months to 36 Months old
Phase 1/Phase 2
NCT04713475
Inclusion Criteria:
• All Patients: Documented GM1 gangliosidosis diagnosis based on genotyping confirming 2 mutations in the GLB1 gene and documented deficiency of beta-galactosidase enzyme by laboratory testing
• Age: 4 to 36 months (first cohort will be 12-36 months) Subjects:
• Early onset infantile (Type 1): Subjects who have signs and/or symptoms of GM1 gangliosidosis that started at or before 6 months of age and have specific developmental milestones remaining
• Late onset infantile (Type 2a): Subjects who have signs and/or symptoms of GM1 gangliosidosis that started between 6 and 18 months of age and have specific developmental milestones remaining
Exclusion Criteria:
• Any clinically significant neurocognitive deficit not attributable to GM1 gangliosidosis or any other condition that may, in the opinion of the investigator, confound interpretation of study results.
• If a subject had an acute illness requiring hospitalization within 30 days of enrollment, the history must be discussed with the sponsor's medical monitor before allowing the subject to be enrolled.
• History of ventilation assisted respiratory support or a need for tracheostomy as a result of their disease.
• Intractable seizure or uncontrolled epilepsy defined as having had an episode of status epilepticus, or seizures requiring hospitalization within 30 days prior to dosing of PBGM01.
• Any contraindication to the ICM administration procedure, including contraindications to fluoroscopic imaging and anesthesia or any condition that would increase the risk of adverse outcomes from the ICM procedure including, but not limited to, the presence of a space occupying lesion causing mass effects or signs of increased intracranial pressure, space occupying lesion in the posterior fossa or foramen magnum, aberrant vascular anatomy such as a large midline posterior inferior cerebellar artery, aberrant venous anatomy such as a large cerebellar vein or occipital sinus, or congenital anatomical abnormalities such as a Chiari malformation.
• Any contraindication to MRI or lumbar puncture (LP).
• Prior gene therapy.
• Use of miglustat within 48 hours prior to dosing of PBGM01. The use of miglustat is prohibited throughout the study.
• Use of enzyme replacement therapy or other investigational therapy within 5 half-lives prior to dosing of PBGM01. The use of enzyme replacement is prohibited throughout the study.
• Receipt of a vaccine within 14 days of dosing.
• Estimate glomerular filtration rate (eGFR) <30 mL/minute based on creatinine
• Coagulopathy (INR > 1.5) or activated partial thromboplastin time [aPTT] > 40 seconds
• Thrombocytopenia (platelet count < 100,000 per μL.
• AST or ALT > 3 times the upper limit of normal (ULN) or total bilirubin > 1.5x ULN
• Cardiomyopathy (screening troponin level above the ULN).
• Peripheral neuropathy
• Medical conditions or laboratory or vital sign abnormalities that would increase risk of complications from intra-cisterna magna injection, anesthesia, fluoroscopy, LP, and/or MRI including temperature over 38°C, oxygen saturation below 95% on room air or baseline oxygen requirement, heart rate or respiratory rate abnormal for age of the subject, abnormal blood pressure for age, or evidence of infection.
• Any condition (e.g., history of any disease, evidence of any current disease, any finding upon physical examination, or any laboratory abnormality) that, in the opinion of the investigator, would put the subject at undue risk during the administration procedure or would interfere with evaluation of PBGM01 or interpretation of subject safety or study results.
Biological: PBGM01
GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
Infantile, Late Infantile, Rare disease, Lysosomal storage disease
PRescribing INterventions for Chronic Pain Via the Electronic Health Record Study - Current Opioid-User Population (PRINCE)
A clinic-randomized study of behavioral economics-inspired "nudges" build into the the electronic health record system, with the goal of improving opioid prescribing decisions.
All
Not specified
N/A
NCT04601480
Inclusion Criteria:
• All primary care providers from all of the Fairview and University of Minnesota Physicians study clinics
Exclusion Criteria:
• Primary care providers who work less than 20% full time equivalent (FTE)
Behavioral: Choice Architecture Nudge, Behavioral: PMP Integration & Nudge
Opioid-use Disorder, Opioid Use, Opioid Abuse
Bone as Regulator of Energy Balance and Male Fertility after SCI: A Pilot Study (Osteocalcin Protocol)
This study proposes a cross-sectional case-control pilot study. Spinal Cord Injury (SCI) is associated with altered bone metabolism, male infertility, and increased rates of insulin resistance. The researchers will perform testing for 30 men with SCI and 10 without SCI. Data will be used to power subsequent clinical trials. A Fairview letter of support has also been uploaded.
Leslie Morse
18 Years and over
Pilot
PMR-2020-29213
STUDY00011054
Inclusion Criteria:
SCI Inclusion Criteria:
-Stated willingness to comply with all study procedures and availability for the duration of the study
-Male age 18-50
-Have completed inpatient rehabilitation and are living in the community
-Have motor complete SCI
Use a wheelchair as primary mobility mode
-English and non-English speakers
No SCI Inclusion Criteria:
-Stated willingness to comply with all study procedures and availability for the duration of the study
-Male age 18-50
-Able to ambulate independently
-English and non-English speakers
Exclusion Criteria:
SCI Exclusion Criteria:
-presence of other neurological condition
-use of chronic ventilator support
-metabolic bone disease
-thyroid disorder
-current use of medications potentially affecting bone health (including bisphosphonates (etidronate or didronel, clodronate or bonefos, tiludronate or skelid, pamidronate, or aredia, alendronate or fosamax, ibandronate or boniva, risedronate or actonel, zoledronate or reclast) parathyroid hormone (forteo, teriparatide, abaloparatide), denosumab (prolia), testosterone, estrogen, anti-epileptics (phenytoin or dilantin, phenobarbital, valproic acid or depakene) lithium, glucocorticoid use for more than 3 months, and those who have received inhaled glucocorticoids in the past year).
-Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.
No SCI Exclusion Criteria:
-presence of neurological condition
-metabolic bone disease
-thyroid disorder
-current use of medications potentially affecting bone health (including bisphosphonates (etidronate or didronel, clodronate or bonefos, tiludronate or skelid, pamidronate, or aredia, alendronate or fosamax, ibandronate or boniva, risedronate or actonel, zoledronate or reclast) parathyroid hormone (forteo, teriparatide, abaloparatide), denosumab (prolia), testosterone, estrogen, anti-epileptics (phenytoin or dilantin, phenobarbital, valproic acid or depakene) lithium, glucocorticoid use for more than 3 months, and those who have received inhaled glucocorticoids in the past year).
-known history of or found on baseline testing to have osteoporosis.
-known history of or found on baseline testing to have diabetes.
-known history of or found on baseline testing to have infertility.
-Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data. Spinal Cord Injury
A Multi-Center, Low lnterventional Study with a Retrospective Component in Participants with Late Onset Pompe Disease
This is a single visit study with retrospective targeted data collection for subjects with late onset Pompe disease. The Sponsor is developing a therapy for which this study will serve as the comparative natural history.
Chester Whitley, MD, PhD
All
18 Years and over
N/A
NCT03893240
STUDY00006997
Inclusion Criteria:
• Provide written informed consent and authorization to use protected health information in accordance with national and local privacy regulations
• Male or females ≥18 years of age
• Currently on ERT using regular recombinant human GAA infusions for at least 18 months prior to screening
• Documented history of clinically moderate late-onset Pompe disease.
Exclusion Criteria:
• History of HIV infection
• Requires any invasive ventilation (other than BiPAP at night) or noninvasive ventilation while awake and upright
• Previously received SPK-3006
• Previously dosed with any investigational or approved gene therapy product at any time or treated with an investigational drug within the last 12 weeks (vaccination studies are accepted)
• Any concurrent clinically significant condition that would not allow the potential participant to complete the Day 1 examinations, or other condition that, in the opinion of the Investigator and/or Sponsor, makes the subject unsuitable for participation in the study
• Unable or unwilling to comply with the schedule of visits and/or study assessments described in the clinical protocol.
Diagnostic Test: Neutralizing Antibody to SPK-3006 capsid
Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma
This study is a randomized phase 3 study comparing selumetinib to Carboplatin and Vincristine (CV) in previously untreated NF1-associated LGG. This study will compare both the event-free survival (EFS) and visual functional outcomes between the 2 randomized arms.
Christopher Moertel, MD
All
2 Years to 21 Years old
Phase 3
NCT03871257
STUDY00008583
Inclusion Criteria:
• Patients must have a body surface area (BSA) of >= 0.5 m^2 at enrollment
• Patients must have neurofibromatosis type 1 (NF1) based on clinical criteria and/or germline genetic testing
• Patients must be newly diagnosed or have previously diagnosed NF-1 associated LGG that has not been treated with any modality other than surgery
• For patients with optic pathway gliomas (OPGs):
• Newly-diagnosed patients with OPG are eligible if there are neurologic symptoms (including visual dysfunction, as defined below) or other exam findings associated with the tumor
• Previously-diagnosed patients with OPG are eligible if they have new or worsening neurologic symptoms (including visual dysfunction, as defined below) or have tumor growth
• For both newly-diagnosed and previously-diagnosed OPG, the patient may be eligible, irrespective of whether there has been tumor growth or other neurological symptoms or worsening, if they meet at least one of the following visual criteria:
• Visual worsening, defined as worsening of visual acuity (VA) or visual fields (VF) documented within the past year (by examination or history); OR
• Significant visual dysfunction (defined as VA worse than normal for age by
• 6 logMAR [20/80, 6/24, or 2.5/10] or more in one or both eyes)
• For patients with LGG in other locations (i.e., not OPGs):
• Newly-diagnosed patients with LGG are eligible if there are neurologic symptoms or other exam findings associated with the tumor
• NOTE: Newly-diagnosed patients with LGG without associated neurologic symptoms or exam findings are not eligible
• Previously-diagnosed patients with LGG are eligible if they have new or worsening neurologic symptoms or have tumor growth
• Although not required, if a biopsy/tumor resection is performed, eligible histologies will include all tumors considered LGG 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 must have two-dimensional measurable tumor >= 1 cm^2
• Patients with metastatic disease or multiple independent primary LGGs are allowed on study
• Creatinine clearance or radioisotope glomerular filtration Rate (GFR) >= 70 mL/min/1.73 m^2 OR a serum creatinine based on age/gender within 7 days prior to enrollment as follows:
• Age; maximum serum creatinine (mg/dL)
• 2 to < 6 years; 0.8 (male) and 0.8 (female)
• 6 to < 10 years; 1 (male) and 1 (female)
• 10 to < 13 years; 1.2 (male) and 1.2 (female)
• 13 to < 16 years; 1.5 (male) and 1.4 (female)
• >= 16 years; 1.7 (male) and 1.4 (female)
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age 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 glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 3 x upper limit of normal (ULN) = 135 U/L within 7 days prior to enrollment. For the purpose of this study, the ULN for SGPT is 45 U/L
• Albumin >= 2 g/dL 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 within 7 days prior to enrollment
• Corrected QT (QTc) interval =< 450 msec by electrocardiography (EKG) within 7 days prior to enrollment
• Absolute neutrophil count >= 1,000/uL (unsupported) within 7 days prior to enrollment
• Platelets >= 100,000/uL (unsupported) within 7 days prior to enrollment
• Hemoglobin >= 8 g/dL (may be supported) within 7 days prior to enrollment
• Patients with a known seizure disorder should be stable and should have not 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. Patients >= 18 years of age must have a blood pressure =< 130/80 mmHg at the time of enrollment (with or without the use of antihypertensive medications).
• Note: 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, an 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
• For patients who undergo a surgery on the target tumor (not required), a pre- and post-operative* MRI of the brain (with orbital cuts for optic pathway tumors) or spine (depending on the site(s) of primary disease) with and without contrast must also be performed within 4 weeks prior to enrollment
• The post-operative MRIs should be performed ideally within 48 hours after surgery if possible
• 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
• Patients must have receptive and expressive language skills in English or Spanish to complete the quality of life (QOL) and neurocognitive assessments
• All patients and/or their parents or legal guardians must sign a written informed consent.
• 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 may not be receiving any other investigational agents
• Patients with any serious medical or psychiatric illness/ condition, including substance use disorders likely in the judgement of the investigator to interfere or limit compliance with study requirements/treatment are not eligible
• 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
• Cardiac conditions:
• 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 Heart Association (NYHA) class II-IV prior or current cardiomyopathy
• Severe valvular heart disease
• History of atrial fibrillation
• Ophthalmologic conditions:
• 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
• Ophthalmological findings secondary to long-standing optic pathway glioma (such as visual loss, optic nerve pallor, or strabismus) or longstanding orbito-temporal plexiform neurofibroma (PN, such as visual loss, strabismus) will NOT be considered a significant abnormality for the purposes of the study
• Treatments and/or medications patient is receiving that would make her/him ineligible, such as:
• 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 placement for vascular access or cerebrospinal fluid (CSF) diverting procedures such as endoscopic third ventriculostomy (ETV) and ventriculo-peritoneal (VP) shunt.
• Note: Patients must have healed from any prior surgery prior to enrollment
• Patients who have an uncontrolled infection are not eligible
Drug: Carboplatin, Other: Quality-of-Life Assessment, Other: Questionnaire Administration, Drug: Selumetinib Sulfate, Drug: Vincristine Sulfate
Low Grade Glioma, Neurofibromatosis Type 1, Visual Pathway Glioma
CAMPFIRE: A Study of Ramucirumab (LY3009806) in Children and Young Adults With Desmoplastic Small Round Cell Tumor
All
12 Months to 29 Years old
Phase 1/Phase 2
NCT04145349
Inclusion Criteria:
• Participants must have discontinued all previous treatments for cancer or investigational agents ≥7 days after the last dose or per the type of previous treatment as stated in the protocol and must have recovered from the acute effects to ≤Grade 2 for alopecia and decreased tendon reflex and to ≤Grade 1 for all other effects at the time of enrollment, unless otherwise noted. Consult with the Lilly clinical research physician or scientist for the appropriate length of time prior to the first dose of study treatment.
• Participants with relapsed, recurrent, or refractory DSRCT.
• Participants must:
• Have measurable disease by Response Evaluation Criteria in Solid Tumors, Version (RECIST) 1.1.
• Have received at least one prior line of systemic treatment (including neoadjuvant and adjuvant chemotherapy). This prior treatment must include approved therapies for which they are eligible, unless the participant is not a suitable candidate for the approved therapy.
• Not be eligible for surgical resection at time of enrollment.
• Adequate cardiac function, defined as: Shortening fraction of ≥27% by echocardiogram, or ejection fraction of ≥50% by gated radionuclide study.
• Adequate blood pressure (BP) control, defined as:
• Participants ≥18 years: Controlled hypertension defined as systolic BP ≤150 millimeters of mercury (mmHg) or diastolic BP ≤90 mmHg where standard medical management is permitted. Please note that ≥2 serial BP readings should be obtained and averaged to determine baseline BP.
• Participants <18 years: A BP ≤95th percentile for age, height, and gender measured as described in National High Blood Pressure Education Program Working Group (NHBPEPWG) on High Blood Pressure in Children and Adolescents (2004), where standard medical management is permitted. Please note that ≥2 serial BP readings should be obtained and averaged to determine baseline BP.
• Adequate hematologic function, as defined as:
• Absolute neutrophil count (ANC): ≥750/microliters (µL) granulocyte-colony stimulating factor (G-CSF) permitted up to 48 hours prior. Participants with documented history of benign ethnic neutropenia or other conditions could be considered with a lower ANC after discussion with and approval from the Lilly clinical research physician or scientist.
• Platelets: ≥75,000/cubic millimeters. Platelet transfusion permitted up to 72 hours prior.
• Hemoglobin: ≥8 grams per deciliter (g/dL) (≥80 g/liter). Transfusions to increase the participant's hemoglobin level to at least 8 g/dL are permitted; however, study treatment must not begin until 7 days after the transfusion, and complete blood count criteria for eligibility are confirmed within 24 hr of first study dose.
• Adequate renal function, as defined as:
• Creatinine clearance or radioscope glomerular filtration rate (GFR) ≥60 milliliters/minute/meters squared OR serum creatinine meeting the following parameters:
• for participants ≥18 years of age serum creatinine ≤1.5×upper limit of normal (ULN);
• for participants <18 years of age, serum creatinine based on age/gender as follows: Age 1 to <2 years maximum serum creatinine 0.6, Age 2 to <6 years maximum serum creatinine 0.8, Age 6 to <10 years maximum serum creatinine
• 0, Age 10 to <13 years maximum serum creatinine 1.2, Age 13 to <16 years maximum serum creatinine 1.5 for males and 1.4 for females, Age 16 to <18 years maximum serum creatinine 1.7 for males and 1.4 for females.
• Urine protein meeting the following parameters:
• for participants ≥18 years of age: <2+ on dipstick or routine urinalysis. If urine dipstick or routine analysis indicates proteinuria ≥2+, then a 24-hour urine must be collected and must demonstrate <2 grams of protein in 24 hours to allow participation in the study.
• for participants <18 years of age: ≤30 milligrams per deciliter urine analysis or <2+ on dipstick. If urine dipstick or routine analysis indicates proteinuria ≥2+, then a 24-hour urine must be collected and must demonstrate <1 g of protein in 24 hours to allow participation in the study.
• Adequate liver function:
• Total bilirubin: ≤1.5×ULN. Except participants with document history of Gilbert Syndrome who must have a total bilirubin level of <3.0×ULN.
• Alanine aminotransferase (ALT) and aspartate aminotransferase (AST): ≤2.5×ULN OR ≤5.0×ULN if the liver has tumor involvement.
• The participant has an adequate coagulation function as defined by International Normalized Ratio ≤1.5 or prothrombin time ≤1.5×ULN, and partial thromboplastin time ≤1.5×ULN if not receiving anticoagulation therapy. For participants receiving anticoagulants, exceptions to these coagulation parameters are allowed if they are within the intended or expected range for their therapeutic use. Participants must have no history of clinically significant active bleeding (defined as within 14 days of first dose of study drug) or pathological condition that carries a high risk of bleeding (for example, tumor involving major vessels or known esophageal varices).
• The participant has adequate hematologic and organ function ≤1 week (7 days) prior to first dose of study drug.
• Female participants of childbearing potential must have a negative urine or serum pregnancy test within 7 days prior to randomization. Male and female participants must agree to use highly effective contraception for the duration of the study and up to 3 months following the last dose of ramucirumab and vinorelbine, and 12 months following the last dose of cyclophosphamide in order to prevent pregnancy.
Exclusion Criteria:
• Participants with severe and/or uncontrolled concurrent medical disease or psychiatric illness/social situation that in the opinion of the investigator could cause unacceptable safety risks or compromise compliance with the protocol.
• Participants who have active infections requiring therapy.
• Participants with an active fungal, bacterial, and/or known severe viral infection including, but not limited to, human immunodeficiency virus (HIV) or viral (A, B, or C) hepatitis (screening is not required).
• Participants who have had allogeneic bone marrow or solid organ transplant are excluded.
• Surgery: Participants who have had, or are planning to have, the following invasive procedures are not eligible:
• Major surgical procedure, laparoscopic procedure, or significant traumatic injury within 28 days prior to enrollment.
• Central line placement or subcutaneous port placement is not considered major surgery.
• Core biopsy, fine needle aspirate, and bone marrow biopsy/aspirate are not considered major surgeries.
• Surgical or other wounds must be adequately healed prior to enrollment.
• Bleeding and thrombosis:
• Participants with evidence of active bleeding or a history of significant (≥Grade 3) bleeding event within 3 months prior to enrollment are not eligible.
• Participants with a bleeding diathesis or vasculitis are not eligible.
• Participants with known or prior history in the prior 3 months of esophageal varices are not eligible.
• Participants with a history of deep vein thrombosis requiring medical intervention (including pulmonary embolism) within 3 months prior to study enrollment are not eligible.
• Participants with a history of hemoptysis or other signs of pulmonary hemorrhage within 3 months prior to study enrollment are not eligible.
• Cardiac:
• Participants with a history of central nervous system (CNS) arterial/venous thromboembolic events (VTEs) including transient ischemic attack (TIA) or cerebrovascular accident (CVA) within 6 months prior to study enrollment are not eligible.
• Participants with myocardial infarction or unstable angina within the prior 6 months.
• Participants with New York Heart Association Grade 2 or greater congestive heart failure (CHF).
• Participants with serious and inadequately controlled cardiac arrhythmia.
• Participants with significant vascular disease (eg, aortic aneurysm, history of aortic dissection).
• Participants with clinically significant peripheral vascular disease.
• Participants who have a history of fistula, gastrointestinal (GI) ulcer or perforation, or intra-abdominal abscess within 3 months of study enrollment are not eligible.
• Participants with a history of hypertensive crisis or hypertensive encephalopathy within 6 months of study enrollment are not eligible.
• Participants who have non-healing wound, unhealed or incompletely healed fracture, or a compound (open) bone fracture at the time of enrollment are not eligible.
• Participants previously treated and progressed on combination cyclophosphamide and vinorelbine regimen. Participants who received combination as maintenance therapy, without progression, would be eligible.
• Participants with a known hypersensitivity to ramucirumab, cyclophosphamide, vinorelbine or any of the excipients of the medicinal products.
• Hepatic impairment:
• Severe liver cirrhosis Child-Pugh Class B (or worse).
• Cirrhosis with a history of hepatic encephalopathy.
• Clinically meaningful ascites resulting from cirrhosis and requiring ongoing treatment with diuretics and/or paracentesis.
• History of hepatorenal syndrome.
• The participant has a bowel obstruction, history or presence of inflammatory enteropathy or extensive intestinal resection (eg, hemicolectomy or extensive small intestine resection with chronic diarrhea), Crohn's disease, ulcerative colitis, or chronic diarrhea.
• The participant has a urinary outflow obstruction.
• The participant has Grade 2 hematuria or non-infectious cystitis at the time of screening.
• Participants with central nervous system (CNS) involvement are ineligible.
Drug: Ramucirumab, Drug: Cyclophosphamide, Drug: Vinorelbine
Desmoplastic Small Round Cell Tumor
soft tissue sarcoma, adolescents and young adults (AYAs), adolescent
MAYFLOWERS-0B-20: A Prospective Study Evaluating Maternal and FetaL Outcomes in the ERa of ModulatorS (MAYFLOWERS)
This is a prospective, multi-center, observational study in pregnant women with cystic fibrosis (CF) to characterize forced expiratory volume in 1 second (FEV1) changes based on exposure to highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators. The key factors contributing to the change in lung function during pregnancy and for 2 years post-delivery will be evaluated along with assessment of fetal and maternal outcomes.
Joanne Billings
NA
NCT04828382
STUDY00012925
Cystic Fibrosis, Pregnancy Related
CFTR modulators, Clinics and Surgery Center (CSC), Cystic Fibrosis, Pregnancy, Pregnancy/obstetric health in women with cystic fibrosis
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
NCT02724579
STUDY00002501
Inclusion Criteria:
• Patients must be newly diagnosed and have:
• Eligibility confirmed by rapid central pathology and molecular screening review on APEC14B1:
• 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 >= 10.0 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
Drug: Cisplatin, Drug: Cyclophosphamide, Other: Laboratory Biomarker Analysis, Drug: Lomustine, Radiation: Radiation Therapy, Drug: Vincristine, Drug: Vincristine Sulfate
Medulloblastoma
A Phase II Study of Carboplatin, Cabazitaxel and Abiraterone in High Volume Metastatic Castration Sensitive Prostate Cancer (CASCARA)
This is a phase II clinical trial in participants with metastatic castration sensitive prostate cancer who are still responding to hormone therapy (androgen deprivation therapy or ADT). This study is done to see if giving a course of anti-cancer drugs, carboplatin and cabazitaxel, followed by an oral drug, abiraterone, improves cancer control as measured by prostate-specific antigen (PSA) level (may indicate the presence of prostate cancer) and imaging studies (e.g. CT scan, bone scan).
Charles Ryan
Male
18 Years and over
Phase 2
NCT03934840
STUDY00006089
Inclusion Criteria:
• Willing and able to provide, or have a legally authorized representative provide, written informed consent and HIPAA authorization for the release of personal health information. A signed informed consent must be obtained before screening procedures are performed.
• Histologically confirmed prostate cancer.
• High volume metastatic disease (defined as the presence of visceral metastases or ≥3 bone lesions).
• ADT for ≤3 months by day 1 of study chemotherapy; Prior episodes of ADT are allowed (i.e. ADT used previously in courses of radiation).
• Testosterone <50 ng/dL. Patients must continue primary ADT with an LHRH analogue if they have not undergone orchiectomy.
• ECOG Performance Status 0 or 1 (see Appendix A)
• Patient has adequate bone marrow and organ function as defined by the following laboratory values:
• Absolute neutrophil count ≥ 1.5 × 10^9/L
• Platelets ≥ 100 × 10^9/L
• Hemoglobin ≥ 9 g/dl
• Serum creatinine ≤ 1.5mg/dL or estimated creatinine clearance ≥ 50 ml/min
• In the absence of liver metastases, alanine aminotransferase (ALT) and aspartate aminotransferase (AST) <2.5 x ULN. If the patient has liver metastases, ALT and AST <5 x ULN
• Total bilirubin < ULN; or total bilirubin ≤3.0 x ULN or direct bilirubin ≤1.5 x ULN in patients with well-documented Gilbert's Syndrome.
• Sexually active males must use a condom during intercourse while taking study drugs and for 30 days after stopping treatment and should not father a child in this period. A condom is required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid. Fertile males must use a condom with spermicide (double barrier method).
• Age ≥ 18 years
Exclusion Criteria:
• Prior exposure to any chemotherapy, PARPi, or immunotherapy for prostate cancer.
• Prior abiraterone or enzalutamide, unless therapy was for < 2 weeks
• Radiation therapy (including palliative radiotherapy to a metastatic lesion) within 14 days or major surgery (e.g., open abdominal, pelvic, thoracic, orthopedic or neurosurgery) within 28 days of the date of the first dose.
• Other systemic therapies for prostate cancer within 28 days or 5 half-lives, whichever is shorter, prior to day 1 of chemotherapy (with the exception of anti-androgens like bicalutamide).
• PSA <2.0 ng/mL at diagnosis.
• If present, peripheral neuropathy must be ≤ Grade 1
• Patients with an active second malignancy that could, in the investigator's opinion, potentially interfere with the patient's ability to participate and/or complete this trial.
• Patients with central nervous system (CNS) involvement unless they meet ALL of the following criteria:
• At least 4 weeks from prior therapy completion (including radiation and/or surgery) prior to starting the study treatment
• Clinically stable CNS tumor at the time of screening.
• Baseline screening for CNS metastases is not required unless presence of signs and/or symptoms of involvement
• Patients with severe psychiatric illness/social situations that would limit compliance with study requirements in the judgment of treating investigator.
• Patient has a history of non-compliance to medical regimen or inability to grant consent.
Drug: Cabazitaxel, Drug: Carboplatin, Drug: Abiraterone, Drug: Prednisone
Prostate Cancer
Clinics and Surgery Center (CSC)
MT2020-11 An Open Label Expanded Access Study of Omidubicel, for Allogeneic Transplantation in Patients with Hematological Malignancies
The overall study objectives are to provide access to omidubicel for transplantation in patients with hematological malignancies and to collect additional safety and efficacy data.
John Wagner, MD
All
12 Years and over
Phase 3
NCT04260698
STUDY00010454
Inclusion Criteria:
• Patients must be at least 12 years of age
• Applicable disease criteria
• Patients must have one or two partially HLA-matched CBUs
• Back-up stem cell source
• Sufficient physiological reserves
• Females of childbearing potential agree to use appropriate method of contraception
• Signed written informed consent
Exclusion Criteria:
• Extensive bone marrow fibrosis
• Donor specific anti-HLA antibodies
• Pregnancy
• Medically unsuitable for transplant
Biological: omidubicel
Hematological Malignancies
Clinics and Surgery Center (CSC)
A natural history study to TRACK brain and spinal changes in individuals with Friedreich Ataxia (TRACK-FA) ((TRACK-FA))
TRACK-FA is a multi-site, international, prospective, observational neuroimaging study in Friedreich’s Ataxia. The objective is to obtain natural history MRI and MRS data over time in the brain and spinal cord in a large cohort of FRDA patients and healthy control. This will permit validation of neuroimaging biomarkers in FRDA for future clinical trials. In addition to neuroimaging data, the study will collect data from clinical, cognitive and mood assessments as well as blood samples to measure blood markers (frataxin and neurofilament light chain). The present IRB protocol covers only subjects enrolled at the University of Minnesota.
Pierre-Gilles Henry
All
5 Years and over
NA
NCT04349514
STUDY00009047
Inclusion Criteria:
• Age ≥ 5 years
• Written informed consent provided
• Individuals with FA must have a genetic confirmation of diagnosis and be biallelic for a GAA repeat length > 55 in intron 1 of FXN and/or have a GAA repeat length > 55 in intron 1 of FXN in one allele and another type of mutation that is inferred to cause loss of function in the second FXN allele
• Individuals with FA must have an age of disease onset ≤ 25 years
• Individuals with FA must have a disease duration ≤ 25 years
• Individuals with FA must have a Friedreich Ataxia Rating Scale (FARS) Functional staging score of ≤ 5 and total modified FARS (mFARS) score of ≤ 65 on enrolment
Exclusion Criteria:
• Age < 5 years
• Unable to provide written informed consent
• Magnetic resonance contraindications (e.g. pacemaker or other metallic surgical implants)
• Presence of metallic dental braces
• Pregnancy (ascertained via a question or test as mandated at particular sites)
• Individuals with FA must not have acute or ongoing medical or other conditions that, after discussion between the Site Investigator and steering committee, is deemed to interfere with the conduct and assessments of the study
• Individuals with FA must not have another neurological condition apart from FA
• Individuals with FA must not have other neurologic conditions that, in the opinion of the Site Investigator, would interfere with the conduct and assessments of the study
• Controls must not have a diagnosed psychiatric or neurological condition
• Controls must not have acute or ongoing medical or other conditions that would interfere with the conduct and assessments of the study
Other: Natural history
Friedreich Ataxia
Neuroimaging, Longitudinal, Biomarkers, Natural history
A One-Year, Multicenter, Non-Interventional, Follow-Up, Safety Study in Subjects Previously Implanted with VC-02TM Combination Product
This is a one-year follow-up study from the parent study; An Open-Label, First-in-Human, Study Evaluating the Safety, Tolerability, and Efficacy of VC-02 Combination Product in Subjects with Type 1 Diabetes Mellitus and Hypoglycemia Unawareness Once a subject completes participation in the parent study with the VC-02 implants and then has all units removed, they will be consented and enrolled into this follow-up study. Clinic visits will occur at the following time points: Day 1, Month 3, Month 6, and Month 12. Visit assessments will include an abbreviated physical exam, vital signs, ultrasound of the areas that were previously implanted, adverse event collection, and limited laboratory assessments. The primary objective is to establish the long-term safety profile of VC-02 through 12 months post-explant. The secondary objective is to evaluate glycemic control following explant.
Melena Bellin
Phase II
NCT02939118
STUDY00003133
Type 1 Diabetes Mellitus
A Phase 1/2 Study of [225Ac]-FPI-1434 Injection in Patients with Locally Advanced or Metastatic Solid Tumours
The study consists of a Phase 1 dose-escalation portion (including both single and multi-dosing cohorts), a Cold Antibody Sub-Study, and a Phase 2. This is a Phase 1/2 clinical trial of a therapeutic investigational drug called [225Ac]-FPI-1434 to investigate safety, tolerability, pharmacokinetics, and anti-tumor activity. The study uses two additional investigational products: • [111In]-FPI-1547 Injection (imaging agent) • FPI-1175 Infusion (cold antibody)
Douglas Yee, MD
All
18 Years and over
Phase 1/Phase 2
NCT03746431
STUDY00013618
Inclusion Criteria:
• Age ≥18 years old.
• Pathologically documented, definitively diagnosed, advanced solid tumour that is refractory to all standard treatment, for which no standard treatment is available, or it is contraindicated, or the patient refuses standard therapy.
• At least 1 measurable lesion which is ≥ 20 mm in largest diameter.
• Eastern Cooperative Oncology Group (ECOG) Performance status of 0 or 1.
• Life expectancy of greater than 3 months as judged by the treating physician.
• Available tumour tissue (either archival or fresh biopsy) for IGF-1R immunohistochemistry. Submission of the tissue is not required prior to enrollment.
• Adequate organ function as indicated by the following laboratory values (all laboratory tests must occur within 14 days before the first dose of study treatment):
• Adequate bone marrow reserves without the use of hematopoietic growth factors, red cell or platelet transfusion as evidenced by:
• Absolute neutrophil count ≥ 1,500/ mm^3 (≥ 1.5 x 10^9/L)
• Platelet count ≥ 100,000/ mm^3 (≥ 100 x 10^9/L)
• Hemoglobin ≥ 9 g/dL (≥ 90 g/L)
• Adequate renal function as evidenced by a creatinine clearance ≥ 60 mL/min using the Cockcroft-Gault Equation. Actual body weight should be used for calculating creatinine clearance using the Cockcroft-Gault Equation (except for patients with body mass index (BMI) > 30 kg/m2 when lean body weight should be used instead).
• Adequate hepatic function as evidenced by:
• Serum total bilirubin ≤ 1.5x upper limit of normal (ULN), unless patient has Gilbert's disease
• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 x ULN (≤ 5 x ULN in patients with known liver metastasis)
• Other laboratory results, vital signs and ECGs are acceptable for enrollment (in the opinion of the Investigator).
• Females of childbearing potential must have a negative pregnancy test results during the General and Imaging Screening Periods.
• Females of childbearing potential and all men must agree to use at least two highly effective forms of contraception one of which must be a barrier method, or agree to remain abstinent, for the duration of study participation and 28 days following the [111In]-FPI-1547 injection, and 5 months (for males) or 8 months (for females)following the final [225Ac]-FPI-1434 Injection.
• Ability to understand and the willingness to sign a written informed consent document.
• In the opinion of the Investigator, the patient is expected to be compliant and have a high probability of completing the study.
• Have measurable disease per RECIST 1.1 as assessed by the local site investigator/radiology. Lesions situated in a previously irradiated area are considered measurable if progression has been demonstrated in such lesions.
Exclusion Criteria:
• Received a systemic therapeutic radiopharmaceutical within 6 months prior to enrollment into this study.
• Contraindications to or inability to perform the required imaging procedures in this study (e.g. inability to lay flat for the image acquisitions, etc.)
• Uncontrolled brain metastasis, including but not limited to the need for treatment with steroids, surgery or radiation therapy. a. To be eligible for the study treatment, patients must have stable disease ≥ 1 month, confirmed by magnetic resonance imaging (MRI) or computed tomography (CT) scan, and have CNS metastases well controlled by low-stable or decreasing dose of steriods, anti-epileptics, or other symptom-relieving medications.
• Anticancer therapy (including investigations agents) or external beam radiation therapy within 14 days of the dosing of [111In]-FPI-1547 (6 weeks for mitomycin-C). Ongoing androgen deprivation therapy for prostate cancer, thyroid stimulating hormone suppression for differentiated thyroid cancer, somatostatin analogues for neuroendocrine tumors are permitted.
• Has known additional malignancy that is progressing or has required active treatment within the past 3 years. Patients with basal cell carcinoma of the skin, squamous cell carcinoma of the skin or carcinoma in situ (e.g., breast cancer, cervical cancer, prostate) that have undergone potentially curative therapy are not excluded.
• Residual CTCAE ≥ Grade 2 side effects of prior therapy, with the exception of residual grade 2 alopecia.
• Prior organ transplantation, including stem cell transplantation.
• Any prior treatment with nitrosoureas or actinomycin-D.
• Clinically relevant proteinuria (e.g. urinary dipstick analysis for proteins is 3+ [300 mg/dL or 4+ [1,000 mg/dL], or daily urinary protein excretion > 500 mg/dL).
• Known or suspect allergies or contraindications to the Investigational Products or any component of the investigational drug formulation.
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, diabetes (random blood sugar during screening ≥250 mg/dL [≥ 13.9 mmol/L]), or psychiatric illness/social situations that would limit compliance with study requirements.
• Received > 20 Gy prior radiation to large areas of the bone marrow (e.g. external radiation therapy to whole pelvis)
Drug: [111In]-FPI-1547 Injection, Drug: [225Ac]-FPI-1434 Injection multi-dose, Biological: FPI-1175 Infusion, Drug: [225Ac]-FPI-1434 Injection single-dose
Advanced Solid Tumours, Actinium-225, Antineoplastic Agents, Targeted Alpha Therapy, Radioimmunoconjugate
225-Ac Labelled Humanized Monoclonal Antibody Against IGF-1R, [225Ac]-FPI-1434, IGF-IR Targeted Alpha Therapeutic, IGF-IR Radioligand Therapy, Clinics and Surgery Center (CSC), Phase I Clinic
Study of Posoleucel (ALVR105, Formerly Viralym-M) for Multi-Virus Prevention in Patients Post-Allogeneic Hematopoietic Cell Transplant (Prevent)
All
1 Year and over
Phase 2/Phase 3
NCT05305040
Key
• ≥1 year of age at the day of screening visit.
• No known or suspected clinically significant disease from AdV, BKV, CMV, EBV, HHV-6, and/or JCV
• Within 15 and 42 days of receiving a first allogeneic HCT and have demonstrated clinical engraftment
• Meet one or more of the following criteria at the time of randomization:
• Related (sibling) donor with at least one mismatch at one of these HLA-gene loci: HLA-A, -B or -DR
• Haploidentical donor
• Matched or Mismatched unrelated donor
• Use of umbilical cord blood as stem cell source
• Ex vivo graft manipulation resulting in T cell depletion
• Received anti-thymocyte globulin or alemtuzumab (Campath-1H) Key
• History of AdV, BKV, CMV, EBV, HHV-6, and/or JCV end-organ disease within 6 months prior to randomization
• Evidence of active Grade >2 acute GVHD
• Presence of non-minor uncontrolled or progressive bacterial, viral or fungal infections
• Known history or current (suspected) diagnosis of CRS requiring treatment associated with the administration of peptides, proteins, and/or antibodies
• Ongoing therapy with high-dose systemic corticosteroids (ie, prednisone equivalent dose >0.5 mg/kg/day) within 24 hours prior to dosing
• Relapse of primary malignancy other than minimal residual disease Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Inclusion Criteria:
• ≥1 year of age at the day of screening visit.
• No known or suspected clinically significant disease from AdV, BKV, CMV, EBV, HHV-6, and/or JCV
• Within 15 and 42 days of receiving a first allogeneic HCT and have demonstrated clinical engraftment
• Meet one or more of the following criteria at the time of randomization:
• Related (sibling) donor with at least one mismatch at one of these HLA-gene loci: HLA-A, -B or -DR
• Haploidentical donor
• Matched or Mismatched unrelated donor
• Use of umbilical cord blood as stem cell source
• Ex vivo graft manipulation resulting in T cell depletion
• Received anti-thymocyte globulin or alemtuzumab (Campath-1H) Key
Exclusion Criteria:
• History of AdV, BKV, CMV, EBV, HHV-6, and/or JCV end-organ disease within 6 months prior to randomization
• Evidence of active Grade >2 acute GVHD
• Presence of non-minor uncontrolled or progressive bacterial, viral or fungal infections
• Known history or current (suspected) diagnosis of CRS requiring treatment associated with the administration of peptides, proteins, and/or antibodies
• Ongoing therapy with high-dose systemic corticosteroids (ie, prednisone equivalent dose >0.5 mg/kg/day) within 24 hours prior to dosing
• Relapse of primary malignancy other than minimal residual disease Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Biological: Posoleucel (ALVR105), Biological: Placebo
Adenovirus Infection, BK Virus Infection, Cytomegalovirus Infections, Epstein-Barr Virus Infections, Human Herpes Virus-6 Infection, JC Virus Infection
Allogeneic Hematopoietic Cell Transplant, ALVR105, Posoleucel, Viralym-M
Post Approval Study of Liposorber LA-15 System for the Treatment of Focal Segmental Glomerulosclerosis in Children (FSGS pediatric)
This multicenter, prospective, single-arm clinical study will evaluate the probable benefit and safety of the LIPOSORBER® LA-15 System for the treatment of adult and pediatric patients with nephrotic syndrome associated with primary focal segmental glomerulosclerosis, when the standard treatment options, including corticosteroid and/or calcineurin inhibitors treatments, have been unsuccessful or not well tolerated, and the patient has a GFR ≥ 60 ml/min/1.73m2, or the patient is post renal transplantation.
All
up to 21 Years old
N/A
NCT02235857
Inclusion Criteria:
• A pediatric patient is deemed suitable for inclusion in the study if the patient has FSGS with a GFR ≥ 45 ml/min/1.73 m 2 and any of the following:
• Refractory nephrotic syndrome in which standard treatment options are unsuccessful (i.e., patient is unresponsive to standard corticosteroid and/or calcineurin inhibitor therapy for at least 8 weeks resulting in failure to achieve complete or partial remission);
• Refractory nephrotic syndrome in which standard treatment options are not well tolerated (i.e., patients intolerant to standard therapies due to severe side effects that negatively affect quality of life without providing an acceptable level of clinical benefit);
• Refractory or recurrent nephrotic syndrome in which standard therapy is contraindicated. or
• Pediatric post renal transplant patients with nephrotic syndrome associated with primary FSGS.
Exclusion Criteria:
• General Exclusion Criteria
• Patient is greater than 21 years of age
• Parent or patient is unwilling or unable to sign and date the informed consent (Note: Only patients 18-21 years of age may sign the informed consent on their own behalf)
• Pregnant, lactating, or planning to become pregnant prior to completing the study (Note: The safety of the use of Liposorber® in pregnant women has not been studied. There may be unknown risks to an embryo/fetus. Sexually active women of child bearing potential should avoid pregnancy during the use of the Liposorber device and throughout the study duration.)
• Unable or unwilling to comply with the follow-up schedule
• Simultaneously participating in another investigational drug or device study
• Body weight < 15 kg (33.1 lbs)
• Medical Exclusion Criteria
• Currently being administered ACE inhibitors that cannot be withheld for at least 24 hours prior to each apheresis treatment (Note: The time period to withhold ACE inhibitors should be prolonged, if determined by the treating physician, considering each individual's renal function and the biological half-life of the ACE-inhibitor currently in use.)
• Currently being administered antihypertensive drugs other than ACE inhibitors (e.g., Angiotensin II receptor blockers (ARBs) that cannot be withheld on the day of apheresis until after the procedure
• Medical condition or disorder that would limit life expectancy to less than the primary clinical study endpoint or that may cause noncompliance with the study plan or confound the data analysis
• Hypersensitivity to dextran sulfate, heparin, or ethylene oxide
• Adequate anticoagulation cannot be achieved due to severe hemophilia, severe hemorrhage diathesis, severe gastrointestinal ulcers, or are recipients of vitamin K antagonist medications
• Extracorporeal circulation therapy with Liposorber® LA-15 System cannot be tolerated due to severe cardiac insufficiency, acute myocardial infarction, severe cardiac arrhythmia, acute apoplexy, severe uncontrollable hypertension, or severe uncontrollable hypotension
• Cardiac impairments such as uncontrolled arrhyth¬mia, unstable angina, decompensated congestive heart failure, or valvular disease
• Functional thyroid disease or liver abnormalities
• Unresolved systemic or local infection that could affect the clinical study outcomes
Device: LIPOSORBER® LA-15 System
Focal Segmental Glomerulosclerosis
pediatric, renal transplantation, recurrence, drug-resistant
Targeting emotional eating and weight loss in adolescents (a.k.a Families, Responsibility, Education, Support, Health for Teens (FRESH-TEEN Study) (FRESH-TEEN)
The two primary aims of the study are: -To evaluate the efficacy of PEER and BWL on the target adolescent’s weight over the 18 months of the study, and -To evaluate the efficacy of PEER and BWL on the target adolescent’s emotion regulation skills and emotional eating over the 18-months of the study.
Carol Peterson
All
13 Years to 16 Years old
N/A
NCT03674944
STUDY00003419
Inclusion Criteria:
• Adolescent age 13-16 years
• BMI percentile 85%85%-99.9% (adolescent only)
• Ability to read English at a minimum 6th grade level (both adolescent and parent); and willing to participate in a 6-month treatment and all assessments (both adolescent and parent)
Exclusion Criteria:
• Current enrollment in a weight management program (Child and Parent);
• Medication that is specifically prescribed for weight loss (Child and Parent);
• Medical or psychiatric condition that may interfere with treatment participation (Child and Parent);
• Regular use of compensatory behavior for weight loss (e.g., purging) during the past six months (Child and Parent);
• Current pregnancy or lactating (Child and Parent);
• Change in psychotropic medication during the previous three months (Child and Parent).
Behavioral: Behavioral Weight Loss (BWL) + Emotion Regulation (ER), Behavioral: Behavioral Weight Loss (BWL)
Overweight and Obesity
Overweight, Obesity, Overeating, Treatment, Body Mass Index, Intervention, Behavioral Treatment, Dialectical Behavior Therapy, Emotion Regulation
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 and over
Phase 2
NCT03732950
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
• 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
Biological: Pembrolizumab
Recurrent Fallopian Tube Carcinoma, Recurrent Ovarian Carcinoma, Recurrent Primary Peritoneal Carcinoma
Clinics and Surgery Center (CSC)
RPC01-3204: A PHASE 3, MULTICENTER, OPEN-LABEL EXTENSION STUDY OF ORAL OZANIMOD FOR MODERATELY TO SEVERELY ACTIVE CROHN S DISEASE
This is a Phase 3, open-label, multicenter extension study to evaluate safety and efficacy of ozanimod in subjects with moderately to severely active CD. Approximately 1200 subjects who have previously participated in a study of ozanimod for CD will be eligible to participate in this study if they meet the eligibility criteria as outlined in the prior study (eg, RPC01-3201, RPC01-3202, RPC01-3203, or RPC01-2201).
Eugenia Shmidt
All
18 Years to 75 Years old
Phase 3
NCT03467958
STUDY00003229
For more information regarding Bristol-Myers Squibb Clinical Trial participation, please
visit: www.BMSStudyConnect.com
• Is not in clinical response or clinical remission after completing 12 weeks in the Induction Studies
• Experience relapse or who complete the Maintenance Study
• Complete a study of ozanimod for Crohn's Disease and meet the criteria for participation
• Has any clinically relevant hepatic, neurological, pulmonary, ophthalmological, endocrine, psychiatric, or other major systemic disease making implementation of the protocol or interpretation of the study difficult or that would put the subject at risk by participating in the study
• Has suspected or diagnosed intra-abdominal or perianal abscess that has not been appropriately treated
• Is receiving treatment with any of the following drugs or interventions within the CYP2C8 inhibitors, inducers and Monoamine oxidase inhibitors Other protocol-defined inclusion/exclusion criteria apply
Inclusion Criteria:
• Is not in clinical response or clinical remission after completing 12 weeks in the Induction Studies
• Experience relapse or who complete the Maintenance Study
• Complete a study of ozanimod for Crohn's Disease and meet the criteria for participation
Exclusion Criteria:
• Has any clinically relevant hepatic, neurological, pulmonary, ophthalmological, endocrine, psychiatric, or other major systemic disease making implementation of the protocol or interpretation of the study difficult or that would put the subject at risk by participating in the study
• Has suspected or diagnosed intra-abdominal or perianal abscess that has not been appropriately treated
• Is receiving treatment with any of the following drugs or interventions within the CYP2C8 inhibitors, inducers and Monoamine oxidase inhibitors Other protocol-defined inclusion/exclusion criteria apply
Drug: Ozanimod
Digestive & Liver Health, Crohn Disease
inflammatory bowel disease, Crohn's Disease, Crohn Disease, Oral, Ozanimod, Moderately active, Severely active, RPC01, RPC01-3204, Clinics and Surgery Center (CSC)
Integrating Fall Prevention Balance Exercises Into a Program for Older Adults With Peripheral Artery Disease (PAD): A Mixed Methods Feasibility Study
All
60 Years and over
N/A
NCT05090774
Inclusion Criteria:
• Enrolled in supervised exercise therapy (SET)
• One or more fall risk factors as determined by CDC STEADI fall risk screener
• One or more falls in the last year
• Unsteadiness when standing or walking
• Worries about falling
• Have symptomatic peripheral artery disease (PAD) with objective diagnosis (ABI less than or equal to 0.90 or previous revascularization)
Exclusion Criteria:
• Formal diagnosis of neurocognitive impairment or <3 on Callahan 6-Item Screener
• Current uncontrolled vestibular dysfunction (e.g. Meniere's Disease, benign paroxysmal positional vertigo)
• Participants who self-report the following symptoms will require clearance from a primary provider (as guided by the Exercise and Screening for You Questionnaire):
• Pain, tightness or pressure in chest during physical activity (walking, climbing stairs, household chores, similar activities) that have not been checked and/ or treated by a healthcare provider
• Current dizziness that have not been checked and/ or treated by a healthcare provider
• Current, frequent falls that have not been checked and/ or treated by a healthcare provider
• Other clinically significant disease that is, in the opinion of the study team, not stabilized or may otherwise confound the results of the study
Behavioral: Adapted Otago Exercise Program (OEP)
Fall, Fall Injury, Peripheral Artery Disease, Peripheral Arterial Disease