Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy
NCT ID: NCT06138743
Last Updated: 2025-11-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
78 participants
INTERVENTIONAL
2024-03-04
2026-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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ARO-DM1 Intravenous (IV) Infusion
Single or multiple doses of ARO-DM1 by IV infusion
ARO-DM1 Intravenous (IV) Infusion
ARO-DM1 by intravenous (IV) infusion
Placebo by IV Infusion
Single or multiple doses of placebo by IV infusion
Placebo Intravenous (IV) Infusion
0.9% NaCl calculated volume to match active treatment by IV infusion
ARO-DM1 Subcutaneous SC Injection
Single or multiple doses of ARO-DM1 by sc injection
ARO-DM1 subcutaneous (SC) injection
ARO-DM1 by subcutaneous (SC) injection(s)
Placebo by SC Injection
Single or multiple doses of placebo by sc injection
Placebo Subcutaneous (SC) Injection
0.9% NaCl calculated volume to match active treatment by SC injection(s)
Interventions
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ARO-DM1 Intravenous (IV) Infusion
ARO-DM1 by intravenous (IV) infusion
Placebo Intravenous (IV) Infusion
0.9% NaCl calculated volume to match active treatment by IV infusion
ARO-DM1 subcutaneous (SC) injection
ARO-DM1 by subcutaneous (SC) injection(s)
Placebo Subcutaneous (SC) Injection
0.9% NaCl calculated volume to match active treatment by SC injection(s)
Eligibility Criteria
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Inclusion Criteria
* 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
* 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)
18 Years
65 Years
ALL
No
Sponsors
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Arrowhead Pharmaceuticals
INDUSTRY
Responsible Party
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Locations
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Research Site
Liverpool, New South Wales, Australia
Research Site
Birtinya, Queensland, Australia
Research Site
Herston, Queensland, Australia
Research Site
Melbourne, Victoria, Australia
Research Site
Christchurch, , New Zealand
Research Site
Taichung, , Taiwan
Research Site
Taipei, , Taiwan
Research Site
Taipei, , Taiwan
Research Site
Bangkok, Bangkok, Thailand
Research Site
Hat Yai, Changwat Songkhla, Thailand
Research Site
Lampang, , Thailand
Countries
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Central Contacts
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Other Identifiers
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2024-513579-42
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
ARODM1-1001
Identifier Type: -
Identifier Source: org_study_id