First-in-Human gene therapy trial hopes to help boys with rare muscle disease breathe easier

NCT ID NCT07052929

Recruiting now Disease control Sponsor: Astellas Gene Therapies Source: ClinicalTrials.gov ↗

First seen Nov 01, 2025 · Last updated May 12, 2026 · Updated 27 times

Summary

This study tests a new gene therapy called ASP2957 for boys with X-linked myotubular myopathy (XLMTM), a rare and serious muscle disease present at birth that often requires a breathing machine. The therapy delivers a healthy copy of the MTM1 gene using a harmless virus to help muscles work better. Up to 9 boys under 3 years old will receive a single infusion, and researchers will monitor safety and find the best dose over one year.

Disclaimer Read more

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

Sign up to get updates when this study changes or when new studies for X-LINKED MYOTUBULAR MYOPATHY are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Contacts and locations

Show contact details

Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Boston Children's Hospital
RECRUITING

Boston, Massachusetts, 02115, United States
Lurie Children's Hospital
RECRUITING

Chicago, Illinois, 60611, United States
Oregon Health & Science University
RECRUITING

Portland, Oregon, 97239, United States
The Hospital for Sick Children
RECRUITING

Toronto, Ontario, Canada

Conditions

Explore the condition pages connected to this study.

X-LINKED MYOTUBULAR MYOPATHY