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Enrolling by invitationPhase 1/2

Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy

Key facts

Who runs it
Sarepta Therapeutics, Inc.
Where
Australia, Belgium, Canada, France, Germany, Italy, New Zealand, Spain, Taiwan, Thailand, and United Kingdom35 sites in 11 countriesChoose a country to see its sites
Status
Enrolling by invitation
Phase
Phase 1/2
People taking part
78 people, planned
Started
4 Mar 2024
Expected to end
31 Dec 2026
Type of study
Interventional, 4 groupsOne group gets a placebo (a dummy treatment), for comparison.
Treatments tested
  • SRP-1003 IV Infusion
  • Placebo IV Infusion
  • SRP-1003 SC Injection
  • Placebo SC Injection
Record updated
5 October 2026

Who can take part

Age
18 years to 65 years
Sex
Women and men
Healthy volunteers
Cannot take part

The registry criteria, word for word

Show all 15 criteriaShow less

Who can take part

  • 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

Who cannot take part

  • Treatment with anti-myotonia medication within a period of 5 half-lives of the medication prior to screening
  • Inadequately controlled diabetes
  • 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
  • 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
  • Confirmed diagnosis of congenital DM1

Check if you might fit

Answer for yourself. This is not a decision: only the study team can say whether you can take part.

  • Are you between 18 and 65 years old?

This trial has no sites in United States.

It has sites in 11 countries. Choose a country to see its sites.

About the trial

What the trial wants to find out

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.

The 4 groups in the trial

  • SRP-1003 IV InfusionTreatment being tested

    Single or multiple doses of SRP-1003 by intravenous (IV) infusion

  • Placebo by IV InfusionPlacebo, for comparison

    Single or multiple doses of placebo by IV infusion

    Treatment: Placebo IV Infusion

  • SRP-1003 SC InjectionTreatment being tested

    Single or multiple doses of SRP-1003 by subcutaneous (SC) injection

  • Placebo by SC InjectionPlacebo, for comparison

    Single or multiple doses of placebo by SC injection

    Treatment: Placebo SC Injection

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
Official title
A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 (SRP-1003) in Subjects With Type 1 Myotonic Dystrophy Who Are ≥18 to ≤ 65 Years
Fit questions
Built from NCT06138743, who can take part
Record updated
5 October 2026