Education and Training

  • Improving the Diagnosis of Common Variable Immune Deficiency

    This is an observational, case-control study with a single blood draw among two cohorts, patients with antibody deficiency (e.g., CVID) and healthy controls. Samples will be analyzed by mass cytometry (CyTOF) to examine the major signaling pathways of all circulating innate and adaptive immune cell types, as well as whole exome sequencing. The goal is to improve our general understanding of the human immune response to infections and the diagnosis of CVID.

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  • Post-Traumatic Stress Symptoms (PTSS) in Transplant Recipients

    This study is conducted to better understand Post-Traumatic Stress Symptoms (PTSS) in adolescent transplant recipients and their parent/guardian and to see if PTSS play a role in the way adolescent transplant recipients take their prescribed medicine.

    Target population: medically stable adolescent solid organ (e.g., heart, kidney, liver, lung, small bowel) transplant recipients and their parent(s)/guardian.

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  • Investigation of Mechanisms of Action in Superpower Glass

    The following study aims to understand the mechanism of action at work in a novel artificial intelligence (AI) tool that runs on Google Glass through an Android app to deliver social emotion cues to children with autism during social interactions. This study will examine 2 versions of software on the Google Glass based wearable intervention system. Participants will receive 1 of 2 versions of the software and use the device at home for 4 weeks. This novel device will use a camera, microphone, head motion tracker to analyze the behavior of the subject during interactions with other people. The system is designed to give participants non-interruptive social cues in real-time and will record social responses that can later be used to help aid behavioral therapy. It is hypothesized that both mechanisms under investigation will contribute to social gains in children over the 4 week period of use.

    Investigator

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  • Longer-term Recurrence Rates in Patients With Bladder Cancer After Hexvix (Cysview)Fluorescence Cystoscopy/TURB

    The study is intended to investigate whether the improved initial detection and resection of bladder cancer lesions in patients with non-muscle invasive bladder cancer with Hexvix (Cysview) fluorescence cystoscopy/TURB will lead to a longer-term reduction in recurrences compared to standard white light cystoscopy/TURB.

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  • MM CAR-T to Upgrade Response BMTCTN1902

    This study is designed as a Phase II, multicenter, single arm trial to assess anti-B Cell Maturation Antigen (BCMA) chimeric antigen receptor (CAR) T-cells (bb2121) to improve post autologous hematopoietic cell transplant (HCT) responses among patients with multiple myeloma (MM).

    Investigator

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  • Rates of Apnea in Patients Undergoing Vitreoretinal Surgery

    The purpose of this study is to determine if apnea (the stopping of breathing) occurs in patients undergoing undergoing monitored anesthesia care (MAC) for vitreoretinal surgery

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  • Raloxifene for Women With Alzheimer's Disease

    This is a multisite pilot randomized trial of raloxifene or placebo for the treatment of women with Alzheimer's disease.

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  • Phase II Cont. IV of ON 01910.Na in MDS w/ Trisomy 8/Intermed-1, 2/High Risk

    This study is under Molecular and Cellular Characterization of Myelodysplastic Syndromes (MDS) (eProtocol 15369). The purpose of this proposed study is to analyze existing samples taken from participants participating in a clinical trial evaluating the efficacy and safety of investigational agent ON 01910.Na (eProtocol 16214). This study will use existing blood and marrow samples to determine the rate and duration of objective hematologic and marrow responses, and duration of progression-free survival in ON01910.Na-treated MDS patients.

    This study will use existing blood and marrow samples to determine the rate and duration of objective hematologic and marrow responses, and duration of progression-free survival in ON01910.Na-treated MDS patients.

    Investigator

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  • Pilot 3D Contrast-Enhanced Ultrasound Imaging to Predict Treatment Response in Liver Metastases

    Patients are invited to participate in a research study of liver perfusion (how blood flows to the liver over time). Researchers hope to learn whether perfusion characteristics of liver metastases may be predictive of response to treatment and whether liver perfusion characteristics can be used to follow response to treatment. Patients were selected as a possible participant in this study because they are identified as having liver metastases

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  • INCB000928 Administered as a Monotherapy or in Combination With Ruxolitinib in Participants With Anemia Due to Myeloproliferative Disorders

    This Phase 1/2, open-label, dose-finding study is intended to evaluate the safety and tolerability, PK, PD, and efficacy of INCB000928 administered as monotherapy or in combination with ruxolitinib in participants with MF who are transfusion-dependent or presenting with symptomatic anemia. This study will consist of 2 parts: dose escalation and expansion.

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  • Left Atrial Pressure Monitoring to Optimize Heart Failure Therapy

    The purpose of this clinical study is to evaluate the safety and clinical effectiveness of use of a physician-directed, patient self-management system, guided by left atrial pressure measurements, for use in patients with heart failure. The system allows patients to adjust their HF medications daily based on a physician-directed prescription plan and their current HF status, similar to the manner in which diabetes patients manage their insulin therapy. The goal of the LAPTOP-HF study is to demonstrate reductions in episodes of worsening heart failure (HF) and hospitalizations in patients who are managed with the left atrial pressure (LAP) management system (treatment group) versus those who receive only the current standard of care (control group).

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  • Master Protocol to Assess the Safety and Antitumor Activity of Genetically Engineered T Cells in NY-ESO-1 and/or LAGE-1a Positive Solid Tumors

    This trial will evaluate safety and efficacy of human engineered T-cell therapies, in participants with advanced tumors.

    Investigator

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  • Mixed Chimera Allogeneic Transplantation From Matched Unrelated Donors For The Treatment Of Multiple Myeloma

    The purpose of the study is to determine the toxicity and feasibility of non-myeloablative allogeneic hematopoietic cell transplants for multiple myeloma from unrelated donors.

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  • Magnetic Resonance Guided High Intensity Focused Ultrasound in Advanced Pancreatic Adenocarcinoma Treatment

    The primary purpose of this protocol is to assess the ExAblate 2100 MR guided high intensity focused ultrasound device as an intervention for treatment of advanced stage pancreatic adenocarcinoma.

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  • Pazopanib in Imatinib Refractory or Intolerant Gastrointestinal Stromal Tumors (GIST)

    This study is being done to gather information about the safety (any harmful effects) and effectiveness (usefulness) of Pazopanib in the treatment of Gastrointestinal Stroma Tumors (GIST) that cannot be treated by surgery or has spread to other organs. The Food and Drug Administration (FDA) have approved Pazopanib for the treatment of advanced kidney cancer but it is not approved for the treatment of GIST. The investigators hope to learn about the safety and usefulness (effectiveness) of Pazopanib for patients with GIST.

    Primary Objective:

    Non-progression rate based on RECIST criteria (CR+PR+SD)

    Secondary Objectives:

    - Response per Choi criteria

    - 6 month progression-free survival

    - Safety and tolerability

    Investigator

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  • Pembrolizumab, Dabrafenib, and Trametinib Before Surgery for the Treatment of BRAF-Mutated Anaplastic Thyroid Cancer

    This phase II trial studies the effect of pembrolizumab, dabrafenib, and trametinib before surgery in treating patients with BRAF V600E-mutated anaplastic thyroid cancer. BRAF V600E is a specific mutation (change) in the BRAF gene, which makes a protein that is involved in sending signals in cells and in cell growth. It may increase the growth and spread of tumor cells. Dabrafenib and trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Pembrolizumab, dabrafenib, and trametinib may help to control BRAF V600E-mutated anaplastic thyroid cancer when given before surgery.

    Investigator

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  • Maintenance Chemotherapy or Observation Following Induction Chemotherapy and Radiation Therapy in Treating Patients With Newly Diagnosed Ependymoma

    The primary aim of this randomized phase III trial was to study whether the addition of maintenance chemotherapy delivered after surgical resection and focal radiation would be better than surgery and focal radiation alone. The trial also studied if patients who received induction chemotherapy and then either achieved a complete response or went on to have a complete resection would also benefit from maintenance chemotherapy. Children ages 1-21 years with newly diagnosed intracranial ependymoma were included. There were 2 arms that were not randomized. One arm studied patients with Grade II tumors located in the supratentorial compartment that were completely resected. One arm studied patients with residual tumor and those patients all received maintenance chemotherapy after focal radiation. Chemotherapy drugs, such as vincristine sulfate, carboplatin, cyclophosphamide, etoposide, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving more than one drug (combination chemotherapy) may kill more tumor cells. Radiation therapy uses high-energy x-rays to kill tumor cells. Specialized radiation therapy that delivers a high dose of radiation directly to the tumor may kill more tumor cells and cause less damage to normal tissue. Giving chemotherapy in combination with radiation therapy may kill more tumor cells and allow doctors to save the part of the body where the cancer started.

    Investigators

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  • Low-Dose Naltrexone for the Treatment of Complex Regional Pain Syndrome

    The investigators are testing treatment with low-dose naltrexone (LDN) for symptom relief of complex regional pain syndrome (CRPS). Study participants will be randomly assigned to receive either LDN or placebo for a period of several weeks. During this period participants will be asked to attend either in-person or virtual study visits and complete questionnaires.

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  • Liraglutide and a Calorie Restricted Diet Augments Weight Loss and Decreases Risk of Type 2 Diabetes and CVD.

    The goal of this study is to evaluate the hypothesis that the addition of liraglutide, a long-acting glucagon-like peptide 1 (GLP-1) analogue, to a calorie-restricted diet will lead to greater weight loss than will a calorie-restricted diet alone in subjects who are older (50 to 60 years of age), overweight/obese, and prediabetic. These individuals have been selected for study because they are at greatly increased risk to develop type 2 diabetes (2DM) and cardiovascular disease (CVD), and it is hypothesized that the addition of liraglutide to a calorie-restricted diet will significantly decrease risk of these adverse outcomes.

    There is considerable evidence that GLP-I compounds, including liraglutide, improve glycemic control in patients with manifest 2DM. However, there is relatively little information as to the potential utility of these compounds in nondiabetic individual at greatly increased risk of 2DM and CVD. This research proposal is aimed at providing some of this information by quantifying the effects of liraglutide, a long-acting GLP-1 analogue, on weight loss, insulin secretion, insulin action, and multiple CVD risk factors in a very high risk group-older, overweight/obese, prediabetic individuals. Furthermore, by using specific methods, not surrogate estimates, and avoiding the confounding effects of glucotoxicity, it will be possible to gain new insights into the effects of GLP-1 on insulin secretion and insulin action.

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  • Maveropepimut-S (MVP-S) and Low-Dose CPA in Patients With Platinum-Resistant Ovarian Cancer

    Phase 2, single arm, study to assess the efficacy and safety of maveropepimut-S (MVP-S) and low-dose cyclophosphamide (CPA) in subjects with recurrent, platinum resistant ovarian cancer.

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