Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy
ConditionMyotonic Dystrophy 1
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-65
SponsorSarepta Therapeutics, Inc.
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 requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of SRP-1003 or placebo.
Eligibility criteria
Qualifiers
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
Disqualifiers
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
Trial design
Treatments tested in this trial
- SRP-1003 IV Infusion
- Placebo IV Infusion
- SRP-1003 SC Injection
- Placebo SC Injection
Treatment groups
78 Participants
are divided into 4 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov