Experimental drug AOC 1001 tested for Long-Term safety in rare muscle disease
NCT ID NCT05479981
First seen Jun 27, 2026 · Last updated Sep 11, 2026 · Updated 3 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.
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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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37 people
The number who actually took part.
- Started
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Aug 2022
- Finished
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Sep 2025
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 to 66 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Key Inclusion Criteria: * Completion of AOC 1001-CS1 (MARINA) study with satisfactory compliance and no significant tolerability issues Key Exclusion Criteria: * Pregnancy, intent to become pregnant, or active breastfeeding * Unwilling or unable to continue to comply with contraceptive requirements * Any new conditions or worsening of existing conditions that in the opinion of the investigator or sponsor would make the participant unsuitable for the study or could interfere with participation or completion of the study
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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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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
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