Clinical Trials Logo

Clinical Trial 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.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT06138743
Study type Interventional
Source Arrowhead Pharmaceuticals
Contact Medical Monitor
Phone 626-304-3400
Email ARO-DM1@arrowheadpharma.com
Status Recruiting
Phase Phase 1
Start date March 4, 2024
Completion date October 2026

See also
  Status Clinical Trial Phase
Recruiting NCT02880735 - Ventilatory Response After Non Invasive Ventilation in Type 1 Myotonic Dystrophy N/A
Recruiting NCT06378216 - Myotonic Dystrophy Type 1 Congenital and Juvenile Form: From Diagnosis to Rehabilitation [MDCJ-NeuBeRe]
Active, not recruiting NCT06089018 - Observational Study of Digital Biomarkers of Myotonia and Gait in Adults and Children With Myotonic Dystrophy
Completed NCT05662150 - Low-frequency Repetitive Nerve Stimulation in Myotonic Dystrophy Type 1 N/A
Active, not recruiting NCT04698551 - NIPD on cffDNA for Triplet Repeat Diseases
Completed NCT05027269 - Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients Phase 1/Phase 2
Completed NCT04712422 - Poor Neck Proprioception May Cause Balance Deficits in Myotonic Dystrophy 1
Recruiting NCT05006924 - Symptoms and Outcome Measures for Upper- Limb Function in Myotonic Dystrophy Type 1
Recruiting NCT04656210 - Myotonic Dystrophy - Vascular and Cognition
Completed NCT04634682 - Effect of MYODM on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1 N/A
Withdrawn NCT06270186 - Evaluation of Cognitive Functions in 20 Patients With Type 1 Myotonic Dystrophy With Virtual Reality Approach N/A
Recruiting NCT06411288 - Global Study of Del-desiran for the Treatment of DM1 Phase 3
Recruiting NCT06300307 - Study of ATX-01 in Participants With DM1 Phase 1/Phase 2
Completed NCT02729597 - Tracking the Brain in Myotonic Dystrophies: a 5-year Longitudinal Follow-up Study N/A
Not yet recruiting NCT05532813 - Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease) Phase 3
Active, not recruiting NCT04886518 - Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1 Phase 2
Completed NCT05036447 - Myotonic Dystrophy Type 1 and Resistance Exercise N/A
Recruiting NCT03424460 - Venous Thromboembolism in Myotonic Dystrophy Type 1 N/A
Recruiting NCT05865483 - Profile of Dysphagia in Myotonic Dystrophy Type 1 (DM1)
Completed NCT04001920 - Effects of a Multiple Component Training Program on Muscles in Adults With Myotonic Dystrophy Type 1 N/A