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

NCT06131983 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 60

Last updated 2026-02-06

No results posted yet for this 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.

Conditions

  • Facio-Scapulo-Humeral Dystrophy

Interventions

DRUG

ARO-DUX4 for Injection

single or multiple doses of ARO-DUX4 by intravenous (IV) infusion

DRUG

Placebo

calculated volume to match active treatment by IV infusion

Sponsors & Collaborators

  • Arrowhead Pharmaceuticals

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
16 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-02-22
Primary Completion
2026-12-31
Completion
2026-12-31
FDA Drug
Yes

Countries

  • Australia
  • Canada
  • Germany
  • Italy
  • Netherlands
  • New Zealand
  • Spain
  • Thailand

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06131983 on ClinicalTrials.gov