Study of ARO-DUX4 in Adult Patients With Facioscapulohumeral Muscular Dystrophy Type 1

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

Phase

1/2

Condition

N/A

Treatment

Placebo

ARO-DUX4 for Injection

Clinical Study ID

NCT06131983
ARODUX4-1001
  • Ages 18-70
  • All Genders

Study Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ARO-DUX4 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In Part 1 of the study, participants will receive one dose of ARO-DUX4 or placebo. In Part 2 of the study, participants will receive 4 doses of ARO-DUX4 or placebo. Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2. All participants will undergo pre- and post-dose MRI-guided muscle biopsies (a total of 2 biopsies). Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy. Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Genetically confirmed FSHD1 based on Screening evaluation or source verifiablemedical record

  • Clinical severity score between 3 and 8 (scale, 0 to 10)

  • Must have eligible lower extremity muscle for biopsy as determined from MRI by acentral reader

  • A 12-lead electrocardiogram (ECG) at Screening with no abnormalities that maycompromise participant's safety in the study

  • Participants of childbearing potential and their partners must use highly effectivecontraception during the study and for at least 12 weeks following the end of studyor last dose of study medication, whichever is later. Males must not donate spermduring the study from Day 1 until at least 12 weeks following the end of study orlast dose of study medication, whichever is later.

Exclusion

Exclusion Criteria:

  • Human Immunodeficiency Virus (HIV) infection as shown by presence of anti-HIVantibody (seropositive) at Screening

  • Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at Screening

  • Uncontrolled hypertension

  • Severe cardiovascular disease

  • History of thrombolic events

  • Platelet count less that the lower limit of normal at Screening

  • History or presence of: a hypercoagulable state, nephrotic range proteinuria,antiphospholipid antibody syndrome, myeloproliferative disease, inability toambulate, use of hormone-based contraceptives.

  • Any contraindication to muscle biopsy or MRI

Note: additional inclusion/exclusion criteria may apply per protocol

Study Design

Total Participants: 60
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 1/2
Study Start date:
February 26, 2024
Estimated Completion Date:
September 30, 2025

Connect with a study center

  • Research Site

    Liverpool, New South Wales 2170
    Australia

    Active - Recruiting

  • Research Site

    Auchenflower, Queensland 4066
    Australia

    Active - Recruiting

  • Research Site

    Birtinya, Queensland 4575
    Australia

    Active - Recruiting

  • Research Site

    Auckland, 1010
    New Zealand

    Active - Recruiting

  • Research Site

    Bangkok, 10700
    Thailand

    Active - Recruiting

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