New drug AOC 1001 tested for rare muscle disease
NCT ID NCT05027269
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested a new drug called AOC 1001 in 39 adults with myotonic dystrophy type 1, a genetic muscle disorder. Participants received either the drug or a placebo by IV infusion. The goal was to check safety and how the drug moves through the body. Results will help decide if larger studies are warranted.
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 controls or reduces symptoms of myotonic dystrophy type 1.
What could go wrong
This is an early phase 1/2 trial with only 39 participants, so results may not apply to everyone. The drug may cause side effects or fail to improve symptoms.
Disclaimer
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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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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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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