Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy
Status:
Recruiting
Trial end date:
2026-10-01
Target enrollment:
Participant gender:
Summary
This is a Phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate
the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single and
multiple ascending doses of ARO-DM1 compared to placebo in male and female subjects 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 ARO-DM1 or placebo.