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Phase 1/2 Study of AOC 1020 in Adults with Facioscapulohumeral Muscular Dystrophy (FSHD)
EU CTIS ID: 2022-502096-32-00
What this study is testing
To evaluate the safety and tolerability of ascending doses of AOC 1020 in participants with FSHD
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- FSHD1 or FSHD2 diagnosis confirmed by documented genetic testing
- Ambulatory and able to walk 10 meters (with or without assistive devices such as one cane, walking stick or braces)
- At least 1 muscle region suitable for biopsy
- Muscle weakness in both upper and lower body, as determined by Investigator
You likely can't join if
- Any contraindication to MRI
- Blood or plasma donation within 16 weeks of Study Day 1
- Pregnancy, intent to become pregnant within 9 months after last planned dose of Study Drug, or active breastfeeding
- Any abnormal lab values, conditions or diseases 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
- Treatment with any investigative medication within 1 month (or 5 half-lives of the drug, whichever is longer) of Screening
- Unwilling or unable to comply with the contraceptive requirements
See the full eligibility criteria
- FSHD1 or FSHD2 diagnosis confirmed by documented genetic testing
- Ambulatory and able to walk 10 meters (with or without assistive devices such as one cane, walking stick or braces)
- At least 1 muscle region suitable for biopsy
- Muscle weakness in both upper and lower body, as determined by Investigator
- Any contraindication to MRI
- Blood or plasma donation within 16 weeks of Study Day 1
- Pregnancy, intent to become pregnant within 9 months after last planned dose of Study Drug, or active breastfeeding
- Any abnormal lab values, conditions or diseases 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
- Treatment with any investigative medication within 1 month (or 5 half-lives of the drug, whichever is longer) of Screening
- Unwilling or unable to comply with the contraceptive requirements
- Body mass index (BMI) >35.0 kg/m2 at Screening. Body weight of > 120 kg at Screening ineligible for participation in cohorts with planned siRNA dose levels > 4 mg/kg.
- History of muscle biopsy within 30 days of the screening biopsy or planning to undergo any nonstudy muscle biopsies over the duration of the study
- History of bleeding disorders, significant keloid, or other skin or muscle conditions (e.g., severe muscle wasting) that, in the opinion of the Investigator, makes the participant unsuitable for serial muscle biopsy
- Anticipated survival less than 2 years
The study team makes the final eligibility decision.
Where it's taking place
- United States
- Canada
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; Canada; United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.