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A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 in Adults 18 to 65 Years With Type 1 Myotonic Dystrophy
Led by Arrowhead Pharmaceuticals · Updated on 2025-11-10
78
Participants Needed
11
Research Sites
N/A
Total Duration
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AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-DM1 in adults aged 18 to 65 with type 1 myotonic dystrophy (DM1). This phase 1/2a study compares single and multiple doses of ARO-DM1 to placebo. The study includes male and female participants with genetically confirmed DM1 who show clinical signs of the disease. Participants will receive ARO-DM1 or placebo either by intravenous infusion or subcutaneous injection. The study is divided into two parts: Part 1 involves single doses, and Part 2 involves multiple doses with dose escalation. Both parts are randomized and double-blinded to compare the effects of the drug and placebo. During the study, participants will be monitored for treatment-emergent adverse events up to 90 days in Part 1 and up to 180 days in Part 2. Assessments include blood tests to measure drug levels, physical tests such as the Timed Up and Go Test, 10-Meter Walk/Run Test, hand function evaluations, and patient-reported health scales. The study lasts up to 180 days with regular visits for safety and effectiveness monitoring.
CONDITIONS
Brief Title
Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Genetically confirmed diagnosis of type 1 myotonic dystrophy (DM1)
- Clinician-assessed signs of DM1 including clear myotonia
- Onset of DM1 symptoms after age 12 years
- Ability to walk at least 10 meters independently at screening
- Use of highly effective contraception plus condom during study and for 90 days after, for subjects of childbearing potential
- No sperm or egg donation during study and for 90 days after last dose
You will not qualify if you...
- Uncontrolled diabetes
- Confirmed diagnosis of congenital DM1
- Uncontrolled hypertension
- Tibialis anterior biopsy within 3 months before Day 1 or planned during study
- Clinically significant heart, liver, or kidney disease
- HIV infection at screening
- Positive for hepatitis B or C at screening
- Untreated or poorly controlled epilepsy
- Use of anti-myotonia medication within 5 half-lives before screening
- Abnormal blood clotting tests at screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 180 days
Participants receive single or multiple doses of ARO-DM1 or placebo by intravenous infusion or subcutaneous injection.
Multiple visits during dosing and assessments through Day 180
Trial Site Locations
Total: 11 locations
1
Research Site
Liverpool, New South Wales, Australia, 2170
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2
Research Site
Birtinya, Queensland, Australia, 4575
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3
Research Site
Herston, Queensland, Australia, 4029
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4
Research Site
Melbourne, Victoria, Australia, 3004
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5
Research Site
Christchurch, New Zealand, 8011
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6
Research Site
Taichung, Taiwan, 40447
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7
Research Site
Taipei, Taiwan, 10041
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8
Research Site
Taipei, Taiwan, 11217
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9
Research Site
Bangkok, Bangkok, Thailand, 10700
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10
Research Site
Hat Yai, Changwat Songkhla, Thailand, 90110
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11
Research Site
Lampang, Thailand, 52000
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Research Team
M
Medical Monitor
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
4
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