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A Phase 1b, Open-label, Multi-cohort Study of AZD0120, an Autologous CD19/BCMA Targeting Chimeric Antigen Receptor T-cell, in Adults With Autoimmune Diseases
This trial is a Phase 1b, open-label, multi-center, clinical study of AZD0120, a BCMA/CD19 dual targeting CAR+ T-cell therapy, to evaluate the safety and toler…
Identification of Novel Genetic Variants and Biomarkers of Disease Progression in Arrhythmogenic Cardiomyopathy
Arrhythmogenic ventricular cardiomyopathy (AVC) is a genetic condition which affects the heart and can lead to heart failure and rhythm problems, of which, sud…
Balance4Mobility: Effects of Walkasins Use on Clinical Outcomes of Gait and Balance Function in Individuals With Peripheral Neuropathy and Balance Problems - A Randomized Control Trial
The goal of this clinical trial is to is to test whether Walkasins can help people with peripheral neuropathy maintain their balance better. The main question …
Cord Blood Transplantation in Children and Young Adults With Hematologic Malignancies and Non-malignant Disorders
This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic di…
INTEGRATED CHILDHOOD ACTIVITY AND NUTRITION (ICAN) STUDY
This study is a multi-functional integrated research and education project to prevent losses in academic achievement, cognitive function, and behavioral health…
Natural History of Familial Dysautonomia
The study will collect clinical information from patients with FD and allow them to give blood to help develop biological markers of the disease to aid diagnos…
The Natural History of Arrhythmogenic Cardiomyopathy With Pathogenic Plakophilin-2 Variants (PKP2-ACM): An Observational Cohort Study
The goal of this study is to describe the natural history and clinical events for patients who have Arrhythmogenic Cardiomyopathy with Pathogenic Plakophilin-2…
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…
Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis
To evaluate the efficacy of Pozelimab/Cemdisiran combination therapy in patients with sIBM
Genetics of Neonatal Encephalopathy and Related Disorders
Investigators at Boston Children's Hospital are conducting research in order to better understand the genetic factors which may contribute to neonatal encephal…
A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Dose Comparison and Exploratory Efficacy Study of Orally Administered SAT-3247 in Ambulatory DMD Patients
Phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and \< 10 years. The trial will study two doses of SAT-3247 in a randomized, double-blind, place…
Prophylactic Intra-Operative Ventricular Arrhythmia Ablation in High-Risk LVAD Candidates (PIVATAL)
To investigate the effect of VTA ablation at the time of LVAD implant to see if it can reduce the incidence of VTA after surgery