Gene therapy trial offers hope for rare muscle disease
NCT ID NCT05230459
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a single dose of a gene therapy called AB-1003 in 10 adults with a rare genetic muscle disease (LGMD2I/R9). The goal is to see if it is safe and can help improve muscle function. Participants must be able to walk or run 10 meters in under 30 seconds.
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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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Kennedy Krieger Institute
RECRUITINGBaltimore, Maryland, 21205, United States
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University of California - Irvine
RECRUITINGIrvine, California, 92697, United States
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University of Iowa
RECRUITINGIowa City, Iowa, 52242, United States
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University of Kansas Medical Center
RECRUITINGKansas City, Kansas, 66160, United States
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University of Washington Medical Center
RECRUITINGSeattle, Washington, 98195, United States
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VCU
RECRUITINGRichmond, Virginia, 23298, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can MRI scans reveal the hidden pattern of muscle damage in a rare muscular dystrophy?
- Experimental drug targets root cause of rare muscle-wasting disease
- Virtual group therapy aims to boost social skills in kids with rare muscle disease
- Robotic arm could help people with paralysis regain independence at home
- Paving the way: new study aims to sharpen tools for LGMD R1 trials