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TRANSPIRE: Lung Injury in a Longitudinal Cohort of Pediatric HSCT Patients
Hematopoietic stem cell transplant (HSCT) is an effective but toxic therapy and pulmonary morbidity affects as many as 25% of children receiving transplant. Ea…
Pediatric Cardiomyopathy Mutation Analysis
The goal of this protocol is to obtain information from individuals with cardiomyopathy and from their families in order to elucidate the molecular genetics of…
A Phase II Prospective Study Evaluating Selective Depletion of CD45RA+ (Naïve) T Cells (TND) From Peripheral Blood Stem Cell (PBSC) Grafts for Prevention of Graft Versus Host Disease (GVHD) in Non-Malignant Diseases (NMDs)
This phase II trial investigates how well a naive T cell depleted graft work for the reduction of graft versus host disease in patients with non-malignant dise…
Hematopoietic Cell Transplant and Gene Therapy for Non-Malignant Blood Disorders Biobank Resource (BMT CTN 2402)
A prospective, multicenter study that will establish a repository of biospecimens and clinical data from patients undergoing hematopoietic stem cell transplant…
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of DYNE-251 Administered Intravenously in Ambulatory Male Participants 4 to 18 Years of Age With Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks…
Prospective Observational Study of Smartphone-Based Self-Monitoring Using an Artificial Intelligence Solution During Non-Surgical Treatment for Thyroid Eye Disease
This prospective observational study evaluates the feasibility and clinical utility of a smartphone-based artificial intelligence (AI) self-monitoring system i…
Natural History Study of Synucleinopathies
Synucleinopathies are a group of rare diseases associated with worsening neurological deficits and the abnormal accumulation of the protein α-synuclein in the …
Single Neuron Population Dynamics in Human Speech Motor Cortex for a Speech Prosthesis
The purpose of this study is to obtain preliminary device safety information and demonstrate proof of principle (feasibility) of the ability of people with tet…
Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders
A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent diso…
A Phase I/II Trial of Reduced Intensity Conditioning and Familial HLA-Mismatched Bone Marrow Transplantation in Children With Non-Malignant Disorders
This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are …
A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime
The hypothesis for this study is that a preparative regimen that maximizes host immunosuppression without myeloablation will be well tolerated and sufficient f…
Center Without Walls for Imaging Proteinopathies With PET (CW2IP2): Phase I Pilot Study of Biodistribution, Metabolism, Excretion and Brain Uptake of 11C-M503
The current protocol is to determine the biodistribution, metabolism, excretion and brain uptake of 11C-M503. The goal of this radiotracer is to quantify alpha…