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

NCT06138743 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 78

Last updated 2026-08-11

No results posted yet for this study

Summary

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.

Conditions

  • Myotonic Dystrophy 1

Interventions

DRUG

SRP-1003 IV Infusion

SRP-1003 by IV infusion

DRUG

Placebo IV Infusion

0.9% sodium chloride (NaCl) calculated volume to match active treatment by IV infusion

DRUG

SRP-1003 SC Injection

SRP-1003 by SC injection(s)

DRUG

Placebo SC Injection

0.9% NaCl calculated volume to match active treatment by SC injection(s)

Sponsors & Collaborators

  • Sarepta Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Medical Director · Sarepta Therapeutics, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-03-04
Primary Completion
2026-12-31
Completion
2026-12-31
FDA Drug
Yes

Countries

  • Australia
  • Belgium
  • Canada
  • France
  • Germany
  • Italy
  • New Zealand
  • Spain
  • Taiwan
  • Thailand
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06138743 on ClinicalTrials.gov