Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy
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
Sarepta Therapeutics, Inc.
78 participants
Mar 4, 2024
INTERVENTIONAL
Conditions
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.
Eligibility
Plain Language Summary
Simplified for easier understanding
This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.
Interested in this trial?
Get notified about updates and connect with the research team.
Interventions
SRP-1003 by IV infusion
0.9% sodium chloride (NaCl) calculated volume to match active treatment by IV infusion
SRP-1003 by SC injection(s)
0.9% NaCl calculated volume to match active treatment by SC injection(s)
Locations(35)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06138743