Education and Training

  • Natural History Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)

    The rod-cone dystrophies (often referred to as retinitis pigmentosa (RP)) are a clinically and genetically heterogeneous group of disorders in which there is progressive loss of rod and later cone photoreceptor function leading to severe visual impairment. RP usually occurs as an isolated retinal disorder, but it may also be seen in association with systemic abnormalities.

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  • Feasibility Study of Intraoperative Detection of Residual Cancer in Breast Cancer Patients

    This is a prospective, multi-center, randomized, clinical trial evaluating patients undergoing breast conserving surgery using the LUM Imaging System.

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  • Lenvatinib/Everolimus or Lenvatinib/Pembrolizumab Versus Sunitinib Alone as Treatment of Advanced Renal Cell Carcinoma

    The primary purpose of the study is to demonstrate that lenvatinib in combination with everolimus (Arm A) or pembrolizumab (Arm B) is superior compared to sunitinib alone (Arm C) in improving progression-free survival (PFS) (by independent imaging review [IIR] using Response Evaluation Criteria in Solid Tumors [RECIST 1.1]) as first-line treatment in participants with advanced renal cell carcinoma (RCC).

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  • Glaser Obesity Study

    This study will determine if the drug metformin, coupled with diet and exercise counseling, will help obese adolescents lose weight.

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  • Innate and Acquired Immunity to Influenza Infection and Immunization (SLVP029)

    The purpose of the study is to get a better understanding of the natural and adaptive immune response to the flu virus and to compare the immune cell responses to FDA-licensed flu vaccines in nasal mucosal cells and in blood.

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  • Neonatal Erythropoietin And Therapeutic Hypothermia Outcomes in Newborn Brain Injury (NEATO)

    Hypoxic-ischemic encephalopathy (HIE), a condition of reduced blood and oxygen flow to a baby's brain near the time of birth, may cause death or neurologic disability. Cooling therapy (hypothermia) provides some protection, but about half of affected infants still have a poor outcome. This clinical trial will determine if the drug erythropoietin, given with hypothermia, is safe to use as a treatment that may further reduce the risk of neurologic deficits after HIE.

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  • Fexofenadine Use in Gastroesophageal Reflux Symptoms

    The investigators wish to study the effectiveness of Fexofenadine (an over the counter allergy pill) for the treatment of gastroesophageal reflux symptoms in patients who still have symptoms despite being on a proton pump inhibitor. The investigators will do this by giving participants both Fexofenadine (an H1 blocker) for 2 weeks and placebo (sugar pill) for 2 weeks. The participants will not know which drug they are getting at a particular time. This will help the investigators better assess the true effectiveness of Fexofenadine.

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  • Health and Recovery Program in Increasing Physical Activity Level in Stage IA-IIIA Endometrial Cancer Survivors

    This randomized phase II trial studies how well a health and recovery program works in increasing physical activity level in stage IA-IIIA endometrial cancer survivors. Health and recovery program which includes exercise counseling, Fitbit tracker, and phone or email/text communication may increase the level of physical activity in endometrial cancer survivors and promote and maintain behavior change at a lower cost.

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  • Genetics of Symptomatology and Treatment Response in Psychotic Major Depression

    We hope to learn more about the biology of psychiatric illness with the hope of improving the diagnosis and treatment of such psychiatric conditions as major depression.

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  • Molecular Evaluation of AML Patients After Stem Cell Transplant to Understand Relapse Events

    Prospective determination of the clinical utility of measurable residual disease (MRD) testing for relapse and survival of patients with acute myeloid leukemia (AML) undergoing allogeneic hematopoietic cell transplantation (alloHCT).

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  • HDR Brachytherapy 68-Ga-RM2 PET, 68-Ga-PSMA-11 PET &Multi Parametric MRI in Prostate Cancer

    This study is being conducted to determine whether the combination of imaging agents 68-Ga RM2 and 68-Ga PMSA11 is better at assessing response to high dose rate (HDR) local therapy than standard imaging or biopsy in patients with known prostate cancer (PC)

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  • First in Human Trial of Topical VT30 in Pts With Venous/Lymphatic Malformations Assoc With PIK3CA or TEK Gene Mutations

    VT30-101 is a 2-part first-in-human trial of topically administered VT30 to subjects with cutaneous venous malformations, lymphatic malformations, or mixed venolymphatic malformations associated with PIK3CA or TEK mutations.

    Part 1 is a 4-week treatment, open-label, 4-sequence, escalating repeat-application cohort study, with intra-subject and inter-cohort dose escalation.

    Part 2 is a 12-week treatment, randomized, placebo-controlled, double-blind, safety and exploratory efficacy study. Part 2 will be initiated only after the successful completion of Part 1 with results that demonstrate the general safety and tolerability of topically applied VT30. Up to 12 subjects who complete Part 1 may be enrolled into Part 2 of the study.

    The primary objective is to evaluate the safety and tolerability of VT30. The study will also determine the dose and regimen of VT30 to be carried into Part 2 of the protocol. Other aims include documenting plasma drug levels of VT30 and VT10 and, on an exploratory basis, examining pharmacologic target engagement and change in potential efficacy readouts.

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  • Optimal Timing of BR55 CEUS of the Ovaries

    This is an exploratory, single center, open label, prospective study of BR55 to determine the optimal phase of the menstrual cycle for performing BR55 CEUS of the target ovary in premenopausal women scheduled to undergo preventative surgery because of high familial/hereditary or genetic risk for ovarian cancer.

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  • HIIT vs. MICT Training Study

    The purpose of this study is to compare the impact of high-intensity interval training (HIIT) versus moderate-intensity continuous training (MICT) on human health outcomes in healthy sedentary subjects, over 12 weeks of exercise training.

    The investigators will compare several health parameters, such as changes in multiomics profile, cardiorespiratory fitness, muscle strength, and body composition, before and after 12-week interventions of either HIIT or MICT.

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  • Genetics of Charcot Marie Tooth (CMT) - Modifiers of CMT1A, New Causes of CMT2

    This project includes two projects. One is looking for new genes that cause Charcot Marie Tooth disease (CMT). The other is looking for genes that do not cause CMT, but may modify the symptoms a person has.

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  • Focal MR-Guided Focused Ultrasound Treatment of Localized Intermediate Risk Prostate Lesions

    The hypothesis of this study is that focal treatment with ExAblate MRgFUS has the potential to be an effective non-invasive treatment for intermediate risk, organ-confined prostate lesions, with a low incidence of morbidity. The study hypothesis will be tested by measuring treatment-related safety and initial effectiveness parameters in the ExAblate MRgFUS treated patients, as described above.

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  • Management of the PDA Trial

    Estimate the risks and benefits of active treatment versus expectant management of a symptomatic patent ductus arteriosus (sPDA) in premature infants.

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  • Efficacy and Safety of ON 01910.Na in Myelodysplastic Syndrome (MDS) Patients With Trisomy 8 or Classified as Intermediate-1, -2 or High Risk

    This study will explore the efficacy and safety of a regimen of ON 01910.Na as a 48-hour continuous intravenous infusion once a week for 3 weeks of a 4-week cycle in MDS patients with Trisomy 8 or classified as Intermediate-1, -2 or High Risk who are not responding to current therapeutic options. The rationale for this trial is based upon data from laboratory studies with ON 01910.Na and upon activity that has been observed in other clinical trials with ON 01910.Na in patients with MDS.

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  • HORNBILL: A Study to Test Different Doses of BI 764524 in Patients Who Have Had Laser Treatment for a Type of Diabetic Eye Disease Called Diabetic Retinopathy With Diabetic Macular Ischemia

    This is a study in people with a type of diabetic eye disease called diabetic retinopathy with diabetic macular ischemia. People who have had laser treatment for their diabetic retinopathy can participate in the study. The laser treatment is called panretinal photocoagulation.

    The purpose of the study is to find out how well different doses of a medicine called BI 764524 are tolerated. BI 764524 is injected into the eye. The study has 2 parts. In the first part, participants get different doses of BI 764524 only once. Participants are in the first part for about 5 months and visit the study site about 8 times. In the second part, participants are put into different groups by chance. Some participants get BI 764524 injections every 4 weeks. Other participants get sham injections every 4 weeks. A sham injection means that it is not a real injection and contains no medicine. Participants cannot tell whether they get the real injection or a sham injection. For the second part, participants are in the study for about 7 months. During this time, they visit the study site about 7 times. In this study, BI 764524 is given to humans for the first time.

    The doctors compare how well people tolerate the BI 764524 injections and the sham injections.

    The doctors also regularly check the general health of the participants.

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  • Phase 1-2 MAHCT w/ TCell Depleted Graft w/ Simultaneous Infusion Conventional and Regulatory T Cell

    For patients with hematologic malignancies undergoing allogeneic myeloablative (MA) HCT with a T cell depleted graft, the infusion of naturally occurring regulatory T cells with conventional T cells (T cell add back) in pre-defined doses and ratios will reduce the incidence of acute graft vs host disease while augmenting the graft vs leukemia effect and improving immune reconstitution.

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