Status:
RECRUITING
Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy
Lead Sponsor:
Arrowhead Pharmaceuticals
Conditions:
Myotonic Dystrophy 1
Eligibility:
All Genders
18-65 years
Phase:
PHASE1
PHASE2
Brief 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 do...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
March 4 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2026
Estimated Enrollment :
78 Patients enrolled
Trial Details
Trial ID
NCT06138743
Start Date
March 4 2024
End Date
December 1 2026
Last Update
November 7 2025
Active Locations (11)
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1
Research Site
Liverpool, New South Wales, Australia, 2170
2
Research Site
Birtinya, Queensland, Australia, 4575
3
Research Site
Herston, Queensland, Australia, 4029
4
Research Site
Melbourne, Victoria, Australia, 3004