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 a type of Myotonic Dystrophy Type 1 (DM1) where the genetic repeat causing the condition is larger than 100 repeats.
- Your muscle symptoms from DM1 started when you were 12 years old or older.
- You have noticeable muscle stiffness (myotonia), meaning it takes you at least 2 seconds to fully open your hand, as assessed by the study doctor.
- You have measurable strength in your hand grip and in lifting your foot upward at the ankle.
- You are able to walk a short distance, climb and descend stairs (for certain groups in the study), and stand up from a chair 5 times in a row — all without using walking aids like canes, walkers, or leg braces.
Who may not be able to join:
- You have had a major surgery in the last 12 weeks, or you are expected to have one (such as having a heart device implanted) during the study.
- You have a history of a severe allergic reaction (anaphylaxis).
- You have another health condition, unrelated to DM1, that significantly affects your ability to walk or take part in physical assessments.
- You have recently taken medications that can reduce muscle stiffness (myotonia), within a certain timeframe before your screening visit (confirm with trial site).
- Your heart's electrical activity (measured by an ECG) shows certain abnormalities, including a reading above specific limits or a significant heart conduction problem, as judged by the study doctor.
- Your lung function test shows that your breathing capacity is below 50% of what is expected for someone your age and size.
- You have had a biopsy of a specific muscle in your lower leg (tibialis anterior) within the past 3 months, or you plan to have one during the study for reasons unrelated to this trial.
- You have had a history of suicidal behaviour, a suicide attempt, or certain levels of suicidal thoughts within the past 6 months, or the study doctor considers you to be at significant risk.
- You have recently taken certain diabetes or weight-management medications (such as semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide) within a specific timeframe before your screening visit.
- Significant weight loss during the study may affect your eligibility or continued participation, as it could impact how the medication is dosed and how results are measured (confirm with trial site).
Important: Always verify eligibility with the trial site directly before applying.
Note: This is not a complete list — additional criteria may apply. This information is provided for general understanding only and is not medical advice.
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: +1-781-317-1919
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
1 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
MAD Cohorts: Number of Participants With Treatment-Emergent Adverse Events (TEAEs); Dose Expansion Cohorts: Change From Baseline in Myotonia as Measured by Video Hand Opening Time (vHOT)
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.