Education and Training

  • A Phase 2, 2-Stage, 2-Cohort Study of Talazoparib (BMN 673), in Locally Advanced and/or Metastatic Breast Cancer Patients With BRCA Mutation (ABRAZO Study)

    The purpose of this 2-stage, 2-cohort Phase 2 trial is to evaluate the safety and efficacy of talazoparib (also known as BMN 673) in subjects with locally advanced or metastatic breast cancer with a deleterious germline BRCA 1 or BRCA 2 mutation. Subjects will be assigned to either Cohort 1 or 2 based on prior chemotherapy for metastatic disease:

    - Cohort 1) Subjects with a documented PR or CR to a prior platinum-containing regimen for metastatic disease with disease progression > 8 weeks following the last dose of platinum; or

    - Cohort 2) Subjects who have received > 2 prior chemotherapy regimens for metastatic disease and who have had no prior platinum therapy for metastatic disease

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  • A Therapy Treatment Response Trial in Patients With Leptomeningeal Metastases ((LM) Using CNSide

    The FORESEE Study is a multi-center, prospective clinical trial enrolling patients with Breast or Non-Small Cell Lung Cancer (NSCLC) who have suspicious or confirmed Leptomeningeal Metastases (LM). Standard of Care methods to diagnose, or assess the treatment response of LM (Clinical Evaluation, MRI and Cytology) have limited sensitivity and specificity. This creates challenges for physicians to manage LM or determine the best course of treatment. CNSide, is a Laboratory Developed Test (LDT ) that is used commercially at the Physician's discretion in Biocept's CLIA certified, CAP accredited laboratory. CNSide can detect and quantify tumor cells in the CSF from patients with Breast Cancer or NSCLC having a suspicious or confirmed LM. The goal of the FORESEE Study is to evaluate the performance of CNSide in monitoring the LM's response to treatment and to assess the impact of CNSide on treatment decisions made by Physicians.

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  • A Study of Pregnenolone in the Treatment of Individuals With Autism

    This study will assess the tolerability and effectiveness of pregnenolone in the treatment of behavioral deficits in adults with autism. Pregnenolone is a naturally occurring hormone found in the body which has been shown to help with the function of nerve cells. It is also shown to modulate the activity of certain brain receptors implicated in autism. We hope to examine the tolerability of pregnenolone in adults with autism.

    Investigator

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  • Antibiotic Therapy With or Without G-CSF in Treating Children With Neutropenia and Fever Caused by Chemotherapy

    RATIONALE: Antibiotics may decrease the side effects of neutropenia and fever caused by chemotherapy. Colony-stimulating factors such as G-CSF may increase the number of immune cells found in bone marrow or peripheral blood and may help a person's immune system recover from the side effects of chemotherapy. It is not yet known whether antibiotic therapy plus G-CSF is more effective than antibiotic therapy alone for treating side effects caused by chemotherapy.

    PURPOSE: Randomized phase III trial to compare the effectiveness of antibiotic therapy with or without G-CSF in treating children who have neutropenia and fever that are caused by chemotherapy.

    Investigator

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  • A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel

    The main aim of this study is to assess and describe the safety outcomes, including newly diagnosed malignancies, of patients with CALD treated with eli-cel in the post-marketing setting (tradename Skysona) and to describe major functional disability (MFD)-free survival over time in participants with more advanced early active CALD. All enrolled participants with CALD treated with eli-cel in the post-marketing setting will be followed in this study for 15 years. No investigational drug product will be administered in this study. This study will enroll 120 participants with CALD treated with eli-cel in the post-marketing setting. A subpopulation of 24 participants with more advanced early active CALD will be specifically enrolled as required by the US FDA as a condition of accelerated approval and will be considered as a separate cohort for effectiveness outcomes.

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  • Adjuvant ctDNA-Adapted Personalized Treatment in Early Stage NSCLC (ADAPT-E)

    In this study circulating tumor DNA (ctDNA) blood testing is used to detect the residual blood cancer. If residual cancer using this blood test is detected there may be at higher risk of having the cancer return. The study is going to test whether or not the number of circulating cancer cells detected in the blood can be reduced by administration durvalumab after the standard treatment if you are tested positive for the residual cancer.

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  • A Study of JNJ-73763989 + Nucleos(t)Ide Analog in Participants Co-Infected With Hepatitis B and Hepatitis D Virus

    The purpose of the study is to evaluate on-treatment efficacy against hepatitis D virus (HDV) of JNJ-73763989 + nucleos(t)ide analog (NA) regimen compared to NA alone.

    Investigator

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  • A Study of Clofarabine in Combination With Etoposide and Cyclophosphamide in Children With Acute Leukemias.

    Clofarabine (injection) is approved by the Food and Drug Administration (FDA) for the treatment of pediatric patients 1 to 21 years old with relapsed or refractory acute lymphoblastic leukemia (ALL) who have had at least 2 prior treatment regimens. This use is based on the induction of complete responses. Randomized trials demonstrating increased survival or other clinical benefit have not been conducted.

    The purpose of the phase 1 portion of this study was to determine if clofarabine added to a combination of etoposide and cyclophosphamide is safe in children with relapsed or refractory acute lymphoblastic leukemia (ALL) or acute myelogenous leukemia (AML). The purpose of the phase 2 portion of the study was to measure the effectiveness of the combination therapy in children with ALL.

    Investigators

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  • A Study of Ocrelizumab in Comparison With Interferon Beta-1a (Rebif) in Participants With Relapsing Multiple Sclerosis

    This randomized, double-blind, double-dummy, parallel-group study will evaluate the efficacy and safety of ocrelizumab in comparison with interferon beta-1a (Rebif) in participants with relapsing multiple sclerosis. Participants will be randomized to receive either ocrelizumab 600 mg or matching placebo intravenous (IV) as 300 mg infusions on Days 1 and 15 for the first dose and as a single infusion of 600 mg for all subsequent infusions every 24 weeks, with placebo injections matching interferon beta-1a SC three times per week; or interferon beta-1a 44 mcg SC injections three times per week (with placebo infusions matching ocrelizumab infusions every 24 weeks).

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  • A Phase 2 Study of CRG-022 in Patients With Relapsed/Refractory Large B-cell Lymphoma

    This is a prospective, open-label, multi-center clinical study designed to evaluate the safety, tolerability, efficacy, pharmacokinetics, pharmacodynamics, and immunogenicity of CRG-022, a CD22-directed autologous Chimeric Antigen Receptor (CAR) T-cell therapy for the treatment of relapsed or refractory large B-cell lymphoma (LBCL).

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  • An Expanded Access Study to Provide at Home Subcutaneous Administration of Pertuzumab and Trastuzumab Fixed-Dose Combination (PH FDC SC) for Patients With HER2-Positive Breast Cancer During the COVID-19 Pandemic

    This single arm, multicenter study provides the pertuzumab and trastuzumab fixed-dose combination formulation for subcutaneous injection (PH FDC SC) administered at home by a home health nursing provider for patients with human epidermal growth factor receptor 2-positive (HER2+) breast cancer who have completed concurrent chemotherapy with pertuzumab (Perjeta) and trastuzumab (Herceptin) by intravenous administration (P+H IV) and are currently receiving or will be receiving maintenance therapy with P+H IV, PH FDC SC, or trastuzumab SC in the clinic. The main objective is to enable continuity of care during the COVID-19 pandemic.

    This study will enroll approximately 200 patients in the United States.

    Participants with early or metastatic HER2+ breast cancer will be enrolled in this study. Participants with metastatic HER2+ breast cancer will receive treatment every 3 weeks and continue treatment unless early cessation is necessary due to disease recurrence, disease progression, unacceptable toxicity, participant withdrawal of consent, or per physician's recommendation. Participants with early HER2+ breast cancer will receive PH FDC SC to complete 1 year (up to 18 cycles) of dual blockade, including the P+H IV, PH FDC SC, or trastuzumab SC they received prior to enrolling in this study, unless early cessation is necessary due to disease recurrence, disease progression, unacceptable toxicity, participant withdrawal of consent, or per physician's recommendation.

    A remote cardiac surveillance substudy will be optional for patients enrolled at select sites.

    The Sponsor may decide to terminate the study when the COVID-19 pandemic is no longer a risk for this patient population.

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  • A Study to Evaluate the Safety of Rituximab Retreatment in Subjects With Systemic Lupus Erythematosus

    This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of rituximab compared with placebo when combined with a single stable background immunosuppressive medication in subjects with moderate to severe systemic lupus erythematosus (SLE). The primary efficacy endpoint of the trial will be evaluated at 52 weeks.

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  • A Placebo-Controlled Study of Saracatinib (AZD0530) in Patients With Recurrent Osteosarcoma Localized to the Lung

    The purpose of this study is to determine how long patients who undergo complete surgical removal of recurrent osteosarcoma in the lung will remain free of cancer after taking Saracatinib compared to patients taking placebo (a sugar pill).

    Investigator

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  • Alisertib in Treating Young Patients With Recurrent or Refractory Solid Tumors or Leukemia

    This phase II trial is studying the side effects of and how well alisertib works in treating young patients with relapsed or refractory solid tumors or leukemia. Alisertib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

    Investigators

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  • A Study Comparing Oral Buprenorphine and Injectable Buprenorphine for the Treatment of Opioid Use Disorder

    VA-BRAVE will determine whether a 28-day long-acting injectable sub-cutaneous (in the belly area) formulation of buprenorphine at a target dose of 300mg is superior in retaining Veterans in opioid treatment and in sustaining opioid abstinence compared to the daily sublingual (under the tongue) buprenorphine formulation at a target dose of 16-24 mg (standard of care). This is an open-label, randomized, controlled trial including 952 Veterans with opioid use disorder (OUD) recruited over 3 years and followed actively for 52 weeks. There are a number of secondary objectives that will be studied as well and include: comorbid substance use, both non-fatal and fatal opioid overdose, HIV and Hepatitis B (HBV) and C (HCV) testing results and risk behaviors, incarceration, quality of life, psychiatric symptoms of depression and posttraumatic stress disorder, housing status, dental health and utilization, and cost-effectiveness.

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  • A Study of Brain Aging in Vietnam War Veterans

    Traumatic brain injury (TBI) and post traumatic stress disorder (PTSD) are common combat related problems and may be associated with a greater risk of Alzheimer's disease (AD). The purpose of this study is to examine the possible connections between TBI and PTSD, and the signs and symptoms of AD on Veterans as they age.

    The information collected will help to learn more about how these injuries may affect Veterans of the Vietnam War as they grow older, as well as Veterans of the current wars in Iraq and Afghanistan, who also have these types of combat related injuries.

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  • An Intravenous Infusion Study of rHIgM22 in Patients With Multiple Sclerosis

    This is a Phase I, multi-center, double-blind, randomized, placebo-controlled, dose-escalation study designed to evaluate safety, tolerability, pharmacokinetics, and immunogenicity of single intravenous (IV) administrations of rHIgM22 in patients with all clinical presentations of MS.

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  • Aminophylline to Prevent Acute Kidney Injury in Children After Cardiac Surgery

    Children with congenital heart defects often need cardiac surgery with cardiopulmonary bypass (the "heart-lung machine"). Approximately 35 to 50% of these children will have "acute kidney injury," or damage to the kidneys, after the procedure. We currently have few medications to prevent this kidney injury. The hypothesis of this study is that giving aminophylline after heart surgery can decrease the acute kidney injury.

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  • A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A

    The objective of this study is to assess the therapeutic safety and preliminary efficacy of a hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells transduced with a lentiviral vector carrying the FANCA gene in subjects with Fanconi anemia subtype A (FA-A).

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  • Autologous Followed by Non-myeloablative Allogeneic Transplantation for Non-Hodgkin's Lymphoma

    The purpose of this trial is to develop an alternative treatment for patients with poor risk non-Hodgkin's lymphoma. This trial uses a combination of high dose chemotherapy with stem cell transplant using the patient's own cells. This is followed with non-myeloablative transplant using stem cells from a related or unrelated donor to try and generate an anti-lymphoma response from the new immune system.

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