Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy
Recruiting
Phase 1
Phase 2
Interventional Study
Myotonic Dystrophy 1
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 18 – 65
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 78 (estimated)
- Sponsor
- Sarepta Therapeutics, Inc. · Industry
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About This Trial
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 requiremen…
Trial Locations
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Eligibility Criteria
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…
Contacts
Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4
1-888-SAREPTA (1-888-727-3782)
CONTACT