clinical trial · NCT06138743
Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy
Myotonic Dystrophy 1SRP-1003 IV InfusionPlacebo IV InfusionSRP-1003 SC InjectionPlacebo SC Injection
brief 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.
started
Mar 4, 2024
primary completion
Dec 31, 2026
completion
Dec 31, 2026
last updated
Aug 11, 2026
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
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol