Education and Training

  • Diabetes Assistant (DiAs) Control-to-Range (CTR) Nocturnal Closed-Loop Camp Study

    The primary goal is to test the function of the Diabetes Assistant (DiAs) enhanced control-to-range (CTR) controller in a closely monitored diabetes camp setting. The camp setting will allow us to obtain pilot efficacy data.

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  • Detection of Graft Versus Host Disease With [18F]F-AraG

    This is a single-center imaging study to determine utility of in vivo imaging with [18F]F-AraG to identify sites of Graft Versus Host Disease (GVHD) in patients highly suspected of having acute GVHD who require systemic therapy, and patients at high risk for developing acute GVHD. [18F]F-AraG PET scans will be compared to biopsy results to correlate T cell accumulation which is implicated in the disease. High risk patients will be followed to verify predictive potential of [18F]F-AraG.

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  • A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec

    The primary objective of this study is to evaluate the clinical outcomes following treatment with nusinersen in participants with spinal muscular atrophy (SMA) who previously received onasemnogene abeparvovec.

    The secondary objectives of this study are to evaluate the safety and tolerability; clinical outcomes and pharmacodynamics (PD) of nusinersen treatment in participants with SMA who previously received onasemnogene abeparvovec.

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  • Efficacy, Tolerability and Pharmacokinetics of Subcutaneous Exendin (9-39) in Patients With Post Bariatric Hypoglycemia

    This study is designed to evaluate the efficacy, safety and pharmacokinetics of subcutaneous exendin (9-39) in subjects with post-bariatric hypoglycemia. Development of this subcutaneous formulation of exendin (9-39) would represent a targeted therapeutic approach for this rare disease with unmet clinical need.

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  • Erwinase Master Treatment Protocol

    The purpose of this study is to make Erwinase available to patients with acute lymphoblastic leukemia (ALL) who have had previous allergic reactions to certain formulations of L-asparaginase.

    Investigator

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  • Determining the Utility of a Behavioral Intervention in Chronic Migraine

    This proposal will involve a pilot study to evaluate the feasibility, acceptability, and outcomes of a lifestyle behavior protocol in managing chronic migraine. Additionally, the proposal will investigate biomolecules that are uniquely involved in chronic migraine patients who respond to the protocol. Successful completion of this proposal will inform the design of a future full-scale behavioral clinical trial to control chronic migraine.

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  • Dose Escalation Study of AST-OPC1 in Spinal Cord Injury

    The purpose of this study is to evaluate the safety of cross sequential escalating doses of AST-OPC1 administered among 5 cohorts at a single time-point between 21 and 42 days post injury, inclusively, to subjects with subacute cervical spinal cord injuries (SCI).

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  • A Study of JNJ-40346527 in Patients With Relapsed or Refractory Hodgkin Lymphoma

    The purpose of this study is to determine the safety, pharmacokinetics, and preliminary efficacy information of JNJ-40346527 in patients with relapsed or refractory Hodgkin lymphoma.

    Investigator

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  • Comparison of Staples Versus Prolene Suture for Skin Closure at Cesarean Delivery

    Currently different materials are used to close the skin after a cesarean delivery, including absorbable suture, non-absorbable suture and staples. It is not known what is the best choice of material to close the skin after a cesarean section, but commonly staples or dissolvable suture is used. Recently plastic surgeons have found that non-dissolvable suture may have a better cosmetic outcome than staples. The investigators hope to learn if there is a difference in pain both at suture/staple removal and 6 weeks postoperatively between Prolene suture, Absorbable suture (monocryl or vicryl) or staples. The investigators also plan to look for differences in wound complications and patient satisfaction, as well as operating and removal times. This knowledge will be important in helping practitioners choose the closure technique at cesarean delivery.

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  • Donor Atorvastatin Treatment for Preventing Severe Acute Graft-Versus-Host Disease in Patients Undergoing Myeloablative Peripheral Blood Stem Cell Transplantation

    This phase II trial studies donor atorvastatin treatment for the prevention of severe acute graft-versus-host disease (GVHD) in patients undergoing myeloablative peripheral blood stem cell (PBSC) transplantation. Giving chemotherapy and total-body irradiation (TBI) before a donor PBSC transplant helps stop the growth of cancer cells. It may also prevent the patient's immune system reject the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving atorvastatin to the donor before transplant may prevent this from happening.

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  • Dose-escalation Study of Lupartumab Amadotin (BAY1129980)

    The purpose of this study is to evaluate:

    - The side effects of BAY1129980 when given every 21 days different dose levels.

    - Determine the dose level of BAY1129980 that should be tested in future clinical research studies.

    - Measure how much BAY1129980 is in the blood at specific times after administration.

    - If treatment with BAY1129980 shows any effect on reducing the tumor growth.

    - If there are specific biomarkers that might be able to explain why some patients respond to treatment and others do not.

    - If treatment with BAY1129980 causes an immune response from the body against the drug (immunogenicity).

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  • A Safety and Tolerability Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness.

    This is a Long-Term, Single-Arm, Open-Label, Multicenter Phase 3 follow-on trial of the ARGX-113-1704 study to evaluate the safety and tolerability of ARGX-113 in patients with gMG. Patients who have completed at least 1 cycle of treatment and at least 1 year of trial ARGX-113-1705 and have started Part B are eligible to enroll in the open-label trial ARGX-113-2002 to receive efgartigimod by SC administration.

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  • Effect of Proton Pump Inhibitors on Endothelial Function

    In this randomized controlled crossover study, the investigators propose to test the hypothesis that proton pump inhibitors (PPIs) increase plasma levels of asymmetric dimethylarginine (ADMA), which is a marker of endothelial dysfunction. The authors propose to evaluate ADMA concentrations and vascular function analysis in healthy volunteers and adults with a history of cardiovascular disease given PPI vs placebo for four weeks each.

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  • Clinical Study of Spinal Muscular Atrophy

    The investigators propose to prepare for clinical trials where SMA patients are asked to join the research effort. The visits will include questions, physical exam, blood drawing, and sometimes X-rays and a skin biopsy. The investigators will use modern computer methods to process the information during which the investigators will plan a clinical trial. Once the clinical trial begins, the investigators will offer SMA patients participation if they meet the criteria for that trial.

    Identifying an effective SMA treatment is very important because there is currently none. Clinical trials are the only way to decide whether a new treatment works in SMA patients or not.

    Investigator

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  • Efficacy and Safety of Allogeneic Mesenchymal Precursor Cells (Rexlemestrocel-L) for the Treatment of Heart Failure.

    The primary objective of this study is to determine whether transendocardial delivery of allogeneic human bone marrow-derived mesenchymal precursor cells (MPCs [rexlemestrocel-L]) is effective in the treatment of chronic heart failure (HF) due to left ventricular (LV) systolic dysfunction.

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  • EPVent 2- A Phase II Study of Mechanical Ventilation Directed by Transpulmonary Pressures

    This phase II multi-centered, randomized controlled trial of mechanical ventilation directed by esophageal pressure measurement will test the primary hypothesis that using a strategy of maintaining a minimal but positive transpulmonary pressure (Ptp = airway pressure minus pleural pressure) throughout the ventilatory cycle will lead to an improvement in patient survival.

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  • EAP 177Lu-DOTA0-Tyr3-Octreotate for Inoperable, SSR+, NETs, Progressive Under SSA Tx

    Advanced Accelerator Applications is currently pursuing marketing approval for 177Lu-DOTA0-Tyr3-Octreotate (Lutathera). This expanded access therapeutic protocol aims to allow patients suffering from inoperable, somatostatin receptor positive, neuroendocrine tumors, progressive under somatostatin analogue therapy to access the investigational product, 177Lu-DOTA0-Tyr3-Octreotate (Lutathera), prior to its commercial availability.

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  • Cancer Biology of Retinoblastoma

    Many children with the childhood cancer, Retinoblastoma, have surgery to remove the tumor and sometimes the entire eye. The purpose of this study is to collect the extra tissue from patients who undergo tumor removal for laboratory experiments that will help us understand not only what occurs in retinoblastoma cells but also how cells normally function. Some of these studies will include an evaluation of how cells control the way that genes are expressed, how cells "know" to become retinal cells, how cells remain retinal cells, how cells lose their identity as retinal cells, what changes make retinoblastoma cells different from normal retinal cells, and what changes make some retinoblastomas worse than others.

    Investigator

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  • Digital Symptom Tracking, Patient Engagement and Quality of Life in Advanced Cancer

    The purpose of this study is to (1) describe patient and clinician engagement in web-based symptom self-monitoring, (2) identify differences in symptom management between intervention and usual care groups, and (3) identify potential outcomes of real-time symptom tracking and management.

    With the assistance of the study coordinator, participants randomized to the intervention will create an account with Noona. Patients will be instructed to log symptoms as often as relevant using their own personal devices. Patients will also be prompted once per week for 24 weeks to log any recent symptoms. These participants will be sent a Symptom Questionnaire (SQ) via the Noona tool that summarizes their symptoms and distress one week prior to each oncology clinic visit. Symptoms designated as clinically severe either during regular symptom logging or via the SQ will trigger a prompt to contact the clinical team for immediate follow-up.

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  • A Trial to Assess Efficacy and Safety of Octreotide Subcutaneous Depot in Patients With Acromegaly

    The purpose of this trial is to assess the efficacy and safety of CAM2029 in patients with acromegaly. Patients will be randomized to either CAM2029 or placebo administered subcutaneously once monthly during 6 months.

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