This is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple ascending doses of SRP-1003 compared to placebo in male and female participants with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of SRP-1003 or placebo.
Who can participate
Age range
18 Years – 65 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Key Inclusion Criteria:
* Genetically confirmed diagnosis of DM1
* Clinician-assessed signs of DM1 including clinically apparent myotonia
* Onset of DM1 symptoms occurred after the age of 12 years
* Walk for at least 10 meters independently at screening
* Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of study or last dose of study drug, whichever is later. Participants must not donate sperm or eggs during the study and for at least 90 days following the end of study or last dose of study drug whichever is later.
Key Exclusion Criteria:
* Inadequately controlled diabetes
* Confirmed diagnosis of congenital DM1
* Uncontrolled hypertension
* History of tibialis anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during the study period
* Clinically significant cardiac, liver or renal disease
* Human immunodeficiency virus infection (seropositive) at screening
* Seropositive for hepatitis B or hepatitis C at screening
* Untreated or poorly controlled epilepsy
* Treatment with anti-myotonia medication within a period of 5 half-lives of the medication prior to screening.
* Abnormal coagulation parameters at screening including platelet count, international normalized ratio, prothrombin time, and activated partial thromboplastin time
Note: Additional inclusion/exclusion criteria may apply per protocol
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Participants with Treatment-emergent Adverse Events Over Time Through End of Study (EOS)
Timeframe: Single-dose phase (Part 1): Up to Day 90 (EOS); multiple-dose phase (Part 2): Up to Day 180 (EOS)
Trial details
NCT IDNCT06138743
SponsorSarepta Therapeutics, Inc.
Sponsor typeINDUSTRY
Study typeINTERVENTIONAL
Primary completion2026-12-31
Contact for this trial
Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4