Phase 2 Muscular Dystrophy Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You have been diagnosed with FSHD1 (a type of muscular dystrophy) confirmed by genetic testing
- Your disease severity falls in the middle range of a 0–10 scale (between 3 and 8)
- A scan (MRI) shows that you have a suitable leg muscle that can be safely used for a biopsy (small tissue sample)
- Your heart tracing (ECG) at the screening appointment shows no problems that could put you at risk during the study
- If you or your partner could become pregnant, you must be willing to use highly effective birth control during the study and for at least 12 weeks after it ends; males must also agree not to donate sperm during this period
Who may not be able to join:
- You have tested positive for HIV at your screening appointment
- You have tested positive for hepatitis B or hepatitis C at your screening appointment
- You have high blood pressure that is not well controlled
- You have serious heart disease
- You have a history of blood clots (thrombotic events)
- Your platelet count (cells that help blood clot) is below the normal range at screening
- You have a history of conditions that affect blood clotting, kidney protein loss, certain immune or blood disorders, or you are unable to walk, or you currently use hormone-based contraception (confirm with trial site)
- There is any medical reason why you cannot safely have a muscle biopsy or an MRI scan
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Phone: 626-304-3400
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
4 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Over Time Through End of Study (EOS)
Can't join this trial?
Data last synced from ClinicalTrials.gov: 23 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.