Education and Training

  • Children and Adolescents With Marfan Syndrome: 10,000 Healthy Steps and Beyond

    Marfan patients are at risk of sudden death due to weakening of the wall of the large blood vessel leading from the heart (aorta). The wall of the aorta weakens and dilates which can rupture, leading to death, and sometimes during intense exercise.

    There is some evidence in Marfan patients that a stiffer aorta increases risk for rupture.

    For some time, clinical care has focused on what type of exercise these patients should avoid due to risk for aortic dissection. Little clinical emphasis has been placed on encouraging patients to engage in routine and safe exercise such as walking. Informed by this evidence, the investigators propose to collaboratively investigate whether regular exercise improves aortic health in adolescent Marfan patients.

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  • Combined 18F-NaF/18F-FDG PET/MRI for Detection of Skeletal Metastases

    This clinical trial studies the use of sodium fluorine-18 (18F-NaF) plus fluorine-18 (18F) fluorodeoxyglucose (FDG) positron emission tomography (PET)/ whole body magnetic resonance imaging (WBMRI) to detect skeletal metastases in patients with stage III-IV breast cancer or stage II-IV prostate cancer.

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  • Dosing of Brentuximab Vedotin for Mycosis Fungoides, Sezary Syndrome Patients

    The purpose of this study is to test any good and bad effects of the study drug called brentuximab vedotin at a lower dose than is FDA-approved.

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  • GAIN Trial: Phase 2/3 Study of COR388 in Subjects With Alzheimer's Disease

    This is a randomized, double-blind, placebo-controlled study that assessed the efficacy, safety, and tolerability of 2 dose levels of COR388 in subjects with a clinical diagnosis of mild to moderate Alzheimer's disease (AD) dementia.

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  • Engaging Patients to Help Achieve Increased Patient Choice and Engagement for AFib Stroke Prevention

    A multi-center, randomized controlled 2-arm trial comparing the effectiveness of an innovative shared decision-making pathway and usual care for Atrial Fibrillation Stroke Prevention

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  • Genomic Profiling in Recommending Treatment for Patients With Metastatic Solid Tumors

    This research trial studies using genomic profiling to recommend anticancer treatment to patients with cancer that has spread beyond the original site of the tumor (metastatic cancer). Genomic profiling studies the deoxyribonucleic acid (DNA) of a tumor to detect genetic changes or abnormalities. This information can then be used to recommend treatments that may be more likely to result in a beneficial response. It is not yet known whether genomic profiling will detect abnormalities that can be used to make treatment recommendations and whether treatment based on genomic profiling is more effective than standard treatment.

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  • Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)

    Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in children with biliary atresia who have undergone a Kasai hepatoportoenterostomy.

    Investigator

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  • Cisplatin and Etoposide Plus Radiation Followed By Nivolumab/Placebo For Locally Advanced NSCLC

    Patients with Stage III unresectable non-small cell lung cancer will receive thoracic radiation, cisplatin and etoposide followed by nivolumab or placebo given every 2 weeks for a year.

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  • Evaluation of Alternative Oseltamivir (Tamiflu) Dosing Strategies.

    Objective 1: Determine the safety and toxicity profile of Tamiflu administered in combination with probenecid in healthy adults.Objective 2: Determine the pharmacokinetic profile of Tamiflu and probenecid in healthy adults.

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  • Evaluation of Pathwork Tissue of Origin (TOO) Test for Human Malignancies

    The pathworks tissue of origin test is a microarray-based test with the goal of identifying the tissue of origin in patients with metastatic tumors of unknown primary site.

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  • Evaluation of the Advanced Hybrid Closed Loop (AHCL) System in Type 1 Adults and Pediatrics Utilizing Lyumjev®

    This US study will evaluate the safety and effectiveness of utilizing insulin Lyumjev® lispro-aabc in the MiniMed™ 780G System in Type 1 adult and pediatric subjects in a home setting to support product and system labeling.

    Investigator

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  • Biopsy of Human Tumors for Cancer Stem Cell Characterization: a Feasibility Study

    To see if a limited sampling of tumor tissue from human subjects is a feasible way to gather adequate tissue for cancer stem cell quantification.

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  • Five or Ten Year Colonoscopy for 1-2 Non-Advanced Adenomatous Polyps

    This trial examines colorectal cancer incidence in participants with 1 to 2 non-advanced adenomas randomized to surveillance colonoscopy at 10 years compared to participants randomized to surveillance colonoscopy at 5 and 10 years.

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  • Chemoimmunotherapy With Epratuzumab in Relapsed Acute Lymphoblastic Leukemia (ALL)

    This Phase II trial is studying how well giving epratuzumab together with an established chemotherapy platform works in treating young patients with relapsed acute lymphoblastic leukemia. Monoclonal antibodies, such as epratuzumab, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread. Others find cancer cells and help kill them or carry cancer-killing substances to them. Chemotherapy drugs work in different ways to stop the growth of cancer cells, either by killing them or by stopping them from dividing. Giving monoclonal antibody therapy in combination chemotherapy may kill cancer cells more effectively.

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  • Bone Marrow Sparing Image-Guided Radiation Therapy (RT) Incorporating Novel Use of GCSF and FDG-PET Imaging

    This is a research study using Granulocyte Colony Stimulating Factor (GCSF) as a bone marrow stimulating agent for imaging to guide radiation treatment planning. G-CSF is a type of growth factor. Growth factors are proteins made in the body. G-CSF is a type of growth factor that makes the bone marrow produce white blood cells to reduce the risk of infection after some types of cancer treatment.

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  • (EXPLORER) Study of BLU-285 in Patients With Advanced Systemic Mastocytosis (AdvSM) and Relapsed or Refractory Myeloid Malignancies

    This is a Phase 1, open-label, dose-escalation study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and antineoplastic activity of avapritinib (also known as BLU-285), administered orally (PO), in adult patients with advanced systemic mastocytosis and other relapsed or refractory myeloid malignancies. The study consists of 2 parts:, dose-escalation (Part 1) and expansion (Part 2).

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  • Efficacy and Safety of Simtuzumab in Adults With Primary, Post Polycythemia Vera or Post Essential Thrombocythemia Myelofibrosis

    This study is to evaluate the efficacy and safety of simtuzumab (GS-6624) on bone marrow fibrosis either alone or in combination with ruxolitinib in participants with primary myelofibrosis (PMF) and post polycythemia vera or post essential thrombocythemia myelofibrosis (ET/PV MF).

    The study is designed as a two-stage trial. In the stage 1, participants will be randomized into two cohorts to receive either 200 or 700 mg of study drug. In the stage 2, participants on ruxolitinib will be randomized to receive either 200 or 700 mg of study drug.

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  • Clinical and Pathologic Studies in Non-Hodgkin's Lymphoma and Hodgkin's Disease

    The purpose of this study is to characterize the molecular and cell biology of the tumor cells in lymphoma.

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  • Efficacy, Safety and Tolerability of Serelaxin When Added to Standard Therapy in AHF

    The purpose of the study was to evaluate the efficacy, safety and tolerability of intravenous infusion of serelaxin, when added to standard therapy, in acute heart failure (AHF) patients.

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  • Chemotherapy for the Treatment of Patients With Newly Diagnosed Very Low-Risk and Low Risk Fusion Negative Rhabdomyosarcoma

    Rhabdomyosarcoma is a type of cancer that occurs in the soft tissues in the body. This phase III trial aims to maintain excellent outcomes in patients with very low risk rhabdomyosarcoma (VLR-RMS) while decreasing the burden of therapy using treatment with 24 weeks of vincristine and dactinomycin (VA) and examines the use of centralized molecular risk stratification in the treatment of rhabdomyosarcoma. Another aim of the study it to find out how well patients with low risk rhabdomyosarcoma (LR-RMS) respond to standard chemotherapy when patients with VLR-RMS and patients who have rhabdomyosarcoma with DNA mutations get separate treatment. Finally, this study examines the effect of therapy intensification in patients who have RMS cancer with DNA mutations to see if their outcomes can be improved.

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