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Neurological Disorders Clinical Trials

Open clinical trials for neurological disorders

Recruiting
DYNEDMD: Measuring Safety and How Well DYNE-251 Works in Boys with Duchenne Muscular Dystrophy

Full title:

A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy and Pharmacokinetics of DYNE-251 Administered to Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Study goal:

The main goal of this study is to see if DYNE-251 is safe to use in young males with DMD. Researchers also want to find out how well DYNE-251 treats the disease and how it affects the body in other ways.

Age:

Male, age 4 to 16 years old

Recruiting
FACOMS: Friedreich's Ataxia Clinical Outcome Measures

Full title:

Friedreich’s Ataxia Clinical Outcome Measures Study

Study goal:

To better understand how Friedreich’s ataxia progresses and to develop tests that can be used in other clinical trials to measure changes in the disease over time.

Diagnosis:

Friedreich's ataxia

Age:

Birth to 22 years old

Clinical trial categories:

Neurological Disorders Friedreich’s Ataxia
Recruiting
GENETX: Assessing Gene Therapy Communication Needs

Study goal:

To learn more about participants beliefs, attitudes, and questions about gene therapy to help make web-based resources to share information. This could help patients make treatment decisions.

Age:

18 to 35 years old with rare genetic diseases

Recruiting
INC: Pediatric Study of Charcot-Marie-Tooth Disease

Full title:

 Inherited Neuropathies Consortium Rare Disease Clinical Research Network

Study goal:

This long-term study aims to better understand the genetic causes of Charcot-Marie-Tooth disease (CMT) and to learn how different forms of the rare hereditary disease affect people over time.

Age:

Birth to 22 years

Recruiting
TBANK: Collecting, Banking, and Distributing Human Tissue Samples in St. Jude Biorepository

Full title:

TBANK: Protocol for Collecting, Banking, and Distributing Human Tissue Samples: St. Jude Children’s Research Hospital Biorepository

Study goal:

To provide a high-quality repository of tumor and normal samples to facilitate translational research performed by St Jude faculty and their collaborators

Diagnosis:

All diseases