Evaluating ARO-DM1 for treating Type 1 Myotonic Dystrophy
A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy Who Are ≥18 to ≤ 65 Years
This study is testing a new drug called ARO-DM1 to see if it can safely help people with Type 1 Myotonic Dystrophy feel better compared to those who receive a placebo.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 78 (estimated) |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Sponsor | Arrowhead Pharmaceuticals Industry-sponsored |
| Locations | 11 sites (Liverpool, New South Wales and 10 other locations) |
| Trial ID | NCT06138743 on ClinicalTrials.gov |
What this trial studies
This is a Phase 1/2a double-blinded, placebo-controlled study designed to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-DM1 in individuals diagnosed with Type 1 Myotonic Dystrophy. Participants will be randomly assigned to receive either ARO-DM1 or a placebo, with the study involving both single and multiple ascending doses. The trial aims to gather data on how the drug affects the condition compared to no treatment.
Who should consider this trial
Good fit: Ideal candidates are individuals with a genetically confirmed diagnosis of Type 1 Myotonic Dystrophy who are at least 12 years old and can walk independently.
Not a fit: Patients with inadequately controlled diabetes, congenital DM1, or significant comorbid conditions may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the management of Type 1 Myotonic Dystrophy symptoms.
How similar studies have performed: While this approach is novel, similar studies targeting genetic conditions have shown promise in the past.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Genetically confirmed diagnosis of DM1 * Clinician-assessed signs of DM1 including clinically apparent myotonia * Onset of DM1 symptoms occurred after the age of 12 years * Walk for at least 10 meters independently at Screening * Subjects of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of study or last dose of study drug, whichever is later. Subjects must not donate sperm or eggs during the study and for at least 90 days following the end of study or last dose of study drug whichever is later. Exclusion Criteria: * Inadequately controlled diabetes * Confirmed diagnosis of congenital DM1 * Uncontrolled hypertension * History of tibialis anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during the study period * Clinically significant cardiac, liver or renal disease * HIV infection (seropositive) at Screening * Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at screening * Untreated or poorly controlled epilepsy * Treatment with anti-myotonia medication within a period of 5 half-lives of the medication prior to Screening. * Abnormal coagulation parameters at Screening including platelet count, international normalized ratio (INR), prothrombin time, and activated partial thromboplastin time (APTT) Note: Additional inclusion/exclusion criteria may apply per protocol
Where this trial is running
Liverpool, New South Wales and 10 other locations
- Research Site — Liverpool, New South Wales, Australia (Recruiting)
- Research Site — Birtinya, Queensland, Australia (Recruiting)
- Research Site — Herston, Queensland, Australia (Recruiting)
- Research Site — Melbourne, Victoria, Australia (Recruiting)
- Research Site — Christchurch, New Zealand (Recruiting)
- Research Site — Taichung, Taiwan (Recruiting)
- Research Site — Taipei, Taiwan (Recruiting)
- Research Site — Taipei, Taiwan (Recruiting)
- Research Site — Bangkok, Bangkok, Thailand (Recruiting)
- Research Site — Hat Yai, Changwat Songkhla, Thailand (Recruiting)
- Research Site — Lampang, Thailand (Recruiting)
Study contacts
- Study coordinator: Medical Monitor
- Email: ARO-DM1@arrowheadpharma.com
- Phone: 626-304-3400
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.