Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy

Last updated: March 23, 2025
Sponsor: Arrowhead Pharmaceuticals
Overall Status: Active - Recruiting

Phase

1/2

Condition

Muscular Dystrophy

Treatment

Placebo

ARO-DM1 for Injection

Clinical Study ID

NCT06138743
ARODM1-1001
2024-513579-42
  • Ages 18-65
  • All Genders

Study 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 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.

Eligibility Criteria

Inclusion

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 contraceptionin addition to a condom during the study and for at least 90 days following the endof study or last dose of study drug, whichever is later. Subjects must not donatesperm or eggs during the study and for at least 90 days following the end of studyor last dose of study drug whichever is later.

Exclusion

Exclusion Criteria:

  • Inadequately controlled diabetes

  • Confirmed diagnosis of congenital DM1

  • Uncontrolled hypertension

  • History of thromboembolic events

  • History of Tibialis Anterior (TA) biopsy within 3 months of Day 1 or planning toundergo 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 themedication prior to Screening.

  • Abnormal coagulation parameters at Screening including platelet count, InternationalNormalized Ratio (INR), prothrombin time, and activated partial thromboplastin time (APTT)

  • History or presence of any of the following: hypercoagulable state, nephrotic rangeproteinuria, antiphospholipid antibody syndrome or myeloproliferative diseases,inability to ambulate, use of hormone-based contraceptives and peri/post- menopausalhormone replacement therapy ≤ 16 weeks prior to Day 1

Note: Additional inclusion/exclusion criteria may apply per protocol

Study Design

Total Participants: 36
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 1/2
Study Start date:
March 04, 2024
Estimated Completion Date:
June 30, 2025

Connect with a study center

  • Research Site

    Birtinya, Queensland 4575
    Australia

    Active - Recruiting

  • Research Site

    Herston, Queensland 4006
    Australia

    Active - Recruiting

  • Research Site

    Melbourne, Victoria 3004
    Australia

    Active - Recruiting

  • Research Site

    Christchurch, 8011
    New Zealand

    Active - Recruiting

  • Research Site

    Taichung, 40447
    Taiwan

    Active - Recruiting

  • National Taiwan University Hospital

    Taipei, 10002
    Taiwan

    Active - Recruiting

  • Research Site

    Taipei, 112201
    Taiwan

    Active - Recruiting

  • Research Site

    Hat Yai, Songkhla 90110
    Thailand

    Active - Recruiting

  • Research Site

    Bangkok, 10700
    Thailand

    Active - Recruiting

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