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Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy
Led by Arrowhead Pharmaceuticals · Updated on 2025-11-10
78
Participants Needed
11
Research Sites
143 weeks
Total Duration
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AI-Summary
What this Trial Is About
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.
CONDITIONS
Official 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 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
You will not qualify if you...
- 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 5 half-lives prior to Screening
- Abnormal coagulation parameters at Screening including platelet count, INR, prothrombin time, and APTT
- Note: Additional inclusion/exclusion criteria may apply per protocol
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
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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