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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.
Diagnosis:
Age:
Male, age 4 to 16 years old
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
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.
Diagnosis:
Age:
Birth to 22 years
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:
Clinical trial categories:
Childhood Cancer Blood Disorders Immune Disorders Infectious Diseases Neurological Disorders Genetic SyndromesFull title:
Friedreich’s Ataxia Global Clinical Consortium UNIFIED Natural History Study (UNIFAI)
Study goal:
To collect data about how Friedreich’s ataxia progresses over time and affects the daily life of patients.
Diagnosis:
Age:
Any