Gene therapy trial hopes to treat rare muscle disease
NCT ID NCT04174105
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage study tests a gene therapy called AT845 for adults with late-onset Pompe disease, a condition that weakens muscles. The treatment aims to deliver a working copy of the GAA gene to muscle cells. Eleven participants who have been on standard enzyme replacement therapy for at least two years are enrolled. The study focuses on safety and whether the therapy can improve muscle function.
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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
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Locations
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Newcastle Upon Tyne Hospitals Foundation Trust Clinical Research Facility
Newcastle upon Tyne, NE1 4LP, United Kingdom
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Stanford University
Palo Alto, California, 94304, United States
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University of California Irvine, Department of Neurology
Orange, California, 92868, United States
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University of Utah, Division of Medical Genetics
Salt Lake City, Utah, 84108, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Pompe disease study aims to clear path for gene therapy