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Study identifier: NCT06138743 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy

Condition: Myotonic Dystrophy 1  ·  Sponsor: Arrowhead Pharmaceuticals

PhasePhase 1/Phase 2
Planned participants78
Who can joinAll sexes, 18 Years to 65 Years
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Medical Monitor  ·  626-304-3400  ·  ARO-DM1@arrowheadpharma.com

Always discuss trial participation with your own doctor first.

Locations (11)

Research SiteLiverpool, New South Wales, AustraliaRecruiting
Research SiteBirtinya, Queensland, AustraliaRecruiting
Research SiteHerston, Queensland, AustraliaRecruiting
Research SiteMelbourne, Victoria, AustraliaRecruiting
Research SiteChristchurch, New ZealandRecruiting
Research SiteTaichung, TaiwanRecruiting
Research SiteTaipei, TaiwanRecruiting
Research SiteTaipei, TaiwanRecruiting
Research SiteBangkok, Bangkok, ThailandRecruiting
Research SiteHat Yai, Changwat Songkhla, ThailandRecruiting
Research SiteLampang, ThailandRecruiting

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Source record: clinicaltrials.gov/study/NCT06138743