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Clinical Genetics Branch (CGB) Eligibility Screening Survey
Background: Clinical Genetics Branch (CGB) researchers study individuals and populations at high genetic risk of cancer in order to improve our understanding …
A Pilot Study to Evaluate the Safety and Tolerability of Baricitinib in Patients With Job s Syndrome With Lupus-like Disease and/or Atopic Dermatitis
Background: Autosomal dominant hyper-IgE syndrome (HIES), also called Job syndrome, is a genetic disorder that affects the immune system. It can cause skin an…
Using Behavioral Economics and Implementation Science to Advance the Use of Genomic Medicine Utilizing an EHR Infrastructure Across a Diverse Health System
Given the expansion of indications for genetic testing and our understanding of conditions for which the results change medical management, it is imperative to…
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 has two parts. In Part 1, the trial will study two doses of SAT-3247 …
Risk Stratification of Ischemic and Non-ischemic Cardiomyopathies in Racial and Ethnic Minority Groups in the Bronx - Cardiovascular Multimodality Imaging Study
Determining the etiology of cardiomyopathy is of high clinical importance for optimal treatment strategy and prediction of prognosis. There is increased risk f…
Multicenter Trial to Evaluate the Safety and Effectiveness of the Cardiohelp System for up to 30 Days of Support in Children With Severe Cardiac Failure
There are two primary goals of this multicenter clinical trial that combines an FDA device trial and a phase II drug trial in the same study cohort. These two …
A Phase 1/2 Study of NKX019, a CD19 Chimeric Antigen Receptor Natural Killer (CAR NK) Cell Therapy, in Subjects With Immune-Mediated Diseases
This is a Phase 1/2, open-label, multi-center, multi-cohort, non-randomized dose escalation and dose expansion basket study to determine the safety and tolerab…
A Phase 2 Randomized, Placebo-Controlled Clinical Trial to Assess the Safety and Efficacy of Donanemab in Participants With Early Cognitive Decline, at Least One Core Clinical Feature of Dementia With Lewy Bodies, and Confirmation of Alpha-Synuclein and Amyloid Co-pathology
The main purpose of this study is to evaluate whether treatment with donanemab slows the progression of cognitive (how we think, learn, remember, pay attention…
A Multicenter, Phase I/II, Clinical Study to Evaluate the Safety, Tolerability and Efficacy of Intrastromal Injection of Genome-editing GEB-101 in Participants With TGFBI-related Corneal Dystrophy
The goal of this clinical trial is to evaluate the safety and efficacy of GEB-101 injection in subjects with TGFBI-related Corneal Dystrophy
Electrical Impedance Myography: Natural History Studies in Neuromuscular Disorders and Healthy Volunteers
Background: \- Electrical impedance myography (EIM) is a new technique being studied to see if it is helpful in evaluating muscle disorders and nerve disorder…
A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque-like subs…
Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of WAL0921 in Patients With Glomerular Kidney Diseases and Proteinuria
This is an adaptive prospective, multi-center, randomized, double-blind, placebo-controlled study to evaluate the safety, efficacy, pharmacokinetics, and pharm…