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 Myotonic Dystrophy Type 1 (DM1) confirmed by genetic testing
- A doctor has observed signs of DM1, including noticeable muscle stiffness (myotonia)
- Your DM1 symptoms first appeared after the age of 12
- You are able to walk at least 10 meters (about 33 feet) on your own at the time of your screening appointment
- If you are able to have children, you must agree to use highly effective birth control plus a condom throughout the study and for at least 90 days after it ends, and agree not to donate sperm or eggs during this time
Who may not be able to join:
- You have diabetes that is not well managed or controlled
- You have been diagnosed with the congenital (present-from-birth) form of DM1
- You have high blood pressure that is not well controlled
- You have had a biopsy of a specific lower leg muscle (tibialis anterior) in the past 3 months, or plan to have one during the study
- You have a significant heart, liver, or kidney condition
- You tested positive for HIV at your screening appointment
- You tested positive for Hepatitis B or Hepatitis C at your screening appointment
- You have epilepsy that is untreated or not well controlled
- You have taken medication specifically for muscle stiffness (myotonia) recently, within a timeframe based on how long that medication stays in your body (confirm with trial site)
- Your blood clotting test results were abnormal at your screening appointment
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: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.