Experimental gene therapy aims to help boys with rare muscle disease breathe easier
NCT ID NCT07052929
First seen Jun 24, 2026 · Last updated Aug 07, 2026 · Updated 4 times
Summary
This early-phase trial tests a new gene therapy called ASP2957 for X-linked myotubular myopathy (XLMTM), a rare muscle disease present at birth that often requires a ventilator to breathe. The therapy delivers a healthy copy of the MTM1 gene using a modified virus. Nine boys up to 3 years old will receive a single infusion, and researchers will monitor safety and find the best dose.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- ASP2957 (a gene therapy using a modified virus to deliver a healthy MTM1 gene)
- What this could lead to
- If it works, this could point toward a treatment that improves muscle function and reduces the need for a ventilator in boys with XLMTM.
- What could go wrong
- This is the first time ASP2957 is tested in humans, so safety and effectiveness are unknown. The trial is very small (9 boys), and results may not apply to all patients. There are risks from the gene therapy and immune-suppressing drugs.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
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Lurie Children's Hospital
RECRUITINGChicago, Illinois, 60611, United States
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Oregon Health & Science University
RECRUITINGPortland, Oregon, 97239, United States
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The Hospital for Sick Children
RECRUITINGToronto, Ontario, Canada
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The University of Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75390, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.