One-Shot gene therapy tested in kids with debilitating muscle disease

NCT ID NCT03381729

Terminated Disease control Sponsor: Novartis Gene Therapies Source: ClinicalTrials.gov ↗

Summary

This study tested a single injection of an experimental gene therapy called AVXS-101 in young children with spinal muscular atrophy (SMA) who could sit but not stand or walk. The main goal was to find a safe dose and see if the treatment could help children gain motor skills like standing or walking. The trial was terminated early and enrolled 32 children between 6 months and 5 years old.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Contacts and locations

Show contact details

Locations

Ann & Robert H. Lurie Children's Hospital

Chicago, Illinois, 60611, United States
Boston Children's Hospital

Boston, Massachusetts, 02115, United States
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Johns Hopkins

Baltimore, Maryland, 21287, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Nemours Children's Hospital

Orlando, Florida, 32827, United States
Stanford University

Stanford, California, 94305, United States
UCLA

Los Angeles, California, 90095, United States
UT Southwestern

Dallas, Texas, 75390, United States
University of Utah

Salt Lake City, Utah, 84112, United States
Washington University

St Louis, Missouri, 63130, United States

Conditions

Explore the condition pages connected to this study.

SPINAL MUSCULAR ATROPHY