New hope for FSHD: Long-Term drug safety trial underway
NCT ID NCT06547216
First seen Jun 27, 2026 · Last updated Jul 15, 2026 · Updated 2 times
Summary
This study is for people with FSHD, a genetic disease that causes muscle weakness. It tests the long-term safety and how well the body tolerates a drug called AOC 1020, given through a vein. About 84 adults who completed a previous study will take part. The main goal is to check for side effects over time.
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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About 84 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jul 2024
- Expected to finish
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Jun 2030
An estimate. End dates often move.
- Lead sponsor
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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
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16 to 72 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.
Inclusion Criteria: 1. Ability to provide written informed consent (signed and dated) and any authorizations required by local law and be willing and able to comply with all study requirements. When enrolling participants who are minors, it is necessary to also obtain consent from a legally designated representative and the participant will receive information in a way adapted to their age and mental maturity. 2. Completion of AOC 1020-CS1 with the following as judged by the Investigator and Sponsor: 1. No significant tolerability issues 2. Satisfactory compliance with the AOC 1020-CS1 protocol requirements Exclusion Criteria: 1. Pregnancy, intent to become pregnant during the clinical study, or active breastfeeding. 2. Unwilling or unable to continue to comply with contraceptive requirements for the length of AOC 1020-CS2. 3. Any new conditions or worsening of existing condition(s) 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. (Note: FSHD progression is not exclusionary, even if the participant no longer has the ability to walk 10 meters without a walker or two canes.)
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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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Duke University
Durham, North Carolina, 27708, United States
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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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Rare Disease Research
Atlanta, Georgia, 30329, United States
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Stanford University
Palo Alto, California, 94304, United States
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University College London
London, WIT 7HA, United Kingdom
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University of California Los Angeles
Los Angeles, California, 90095, United States
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University of California San Diego
San Diego, California, 92093, 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 Ottawa
Ottawa, Ontario, K1Y 4E9, Canada
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University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
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University of Rochester Medical Center
Rochester, New York, 14642, United States
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University of Sheffield
Sheffield, S10 2TN, United Kingdom
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University of Texas Southwestern
Dallas, Texas, 75390, United States
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University of Washington
Seattle, Washington, 98104, United States
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Virginia Commonwealth University
Richmond, Virginia, 23298, United States
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