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

Phase 1/2

Recruiting

• Conditions: Facioscapulohumeral muscular dystrophy (FSHD)

• Registration Number: 2023-509748-89-00
• Lead Sponsor: Arrowhead Pharmaceuticals Inc.

Brief Summary

To evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ARO-DUX4 using escalating single and multiple doses in subjects with facioscapulohumeral muscular dystrophy Type 1 (FSHD1).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-11-08
• Study First Submitted QC: 2023-11-13
• Study First Posted: 2023-11-15
• Study Start: 2024-02-26
• Status Verified: 2025-10
• Last Update Submitted: 2025-10-02
• Last Update Posted: 2025-10-07
• Primary Completion: 2026-12
• Study Completion: 2026-12-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 44

Inclusion Criteria

• Genetically confirmed FSHD1 based on Screening evaluation or source verifiable medical 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 a central reader
• A 12-lead electrocardiogram (ECG) at Screening with no abnormalities that may compromise participant's safety in the study
• Participants of childbearing potential and their partners must use highly effective contraception during the study and for at least 12 weeks following the end of study or last dose of study medication, whichever is later. Males must not donate sperm during the study from Day 1 until at least 12 weeks following the end of study or last dose of study medication, whichever is later.

Exclusion Criteria

• Human Immunodeficiency Virus (HIV) infection as shown by presence of anti-HIV antibody (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 to ambulate, use of hormone-based contraceptives.
• Any contraindication to muscle biopsy or MRI

Note: additional inclusion/exclusion criteria may apply per protocol

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Primary Outcome Measures

Name	Time	Method
Incidence, frequency, and severity of treatment-emergent adverse events (TEAEs) in subjects with FSHD1 over time through end of study (EOS) at Day 90 (Part 1) or Day 360 (Part 2)		Incidence, frequency, and severity of treatment-emergent adverse events (TEAEs) in subjects with FSHD1 over time through end of study (EOS) at Day 90 (Part 1) or Day 360 (Part 2)

Secondary Outcome Measures

Name	Time	Method
Plasma PK of ARO-DUX4 in subjects with FSHD1		Plasma PK of ARO-DUX4 in subjects with FSHD1
Urinary excretion of ARO-DUX4 in subjects with FSHD1		Urinary excretion of ARO-DUX4 in subjects with FSHD1

Trial Locations

Locations (5)

Research Site 2; 🇪🇸 Madrid, Spain

Research Site 3; 🇪🇸 Barcelona, Spain

Research Site 1; 🇪🇸 Valencia, Spain

Research Site 4; 🇦🇺 Melbourne, Victoria, Australia

Research Site; 🇹🇭 Bangkok, Thailand