Experimental drug AOC 1001 tested for Long-Term safety in rare muscle disease
NCT ID NCT05479981
First seen Nov 01, 2025 · Last updated Jun 23, 2026 · Updated 27 times
Summary
This study is a follow-up to an earlier trial, testing the long-term safety and effects of a drug called AOC 1001 in adults with myotonic dystrophy type 1 (DM1), a genetic muscle disease. 37 participants who completed the first study received multiple doses of AOC 1001 by IV infusion. Researchers are monitoring side effects and checking how the drug affects muscle tissue and disease markers.
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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.
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.
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Contacts and locations
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Locations
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Kansas University Medical Center
Kansas City, Kansas, 66205, United States
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Ohio State University
Columbus, Ohio, 43221, United States
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Stanford University
Palo Alto, California, 94304, United States
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University of California Los Angeles
Los Angeles, California, 90095, United States
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University of Colorado
Denver, Colorado, 80045, United States
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University of Florida
Gainesville, Florida, 32608, United States
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University of Rochester Medical Center
Rochester, New York, 14642, United States
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Virginia Commonwealth University
Richmond, Virginia, 23298, United States
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
AOC 1001
What this could lead to
If successful, this could point toward a treatment that slows or improves muscle function in people with DM1.
What could go wrong
This is a small, early-phase extension study with only 37 participants, so results may not apply to everyone. Long-term safety and effectiveness are still being evaluated.
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.