Study of ARO-DUX4 in Adult and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1
Recruiting
Phase 1
Phase 2
Interventional Study
Facio-Scapulo-Humeral Dystrophy
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 16 – 70
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 60 (estimated)
- Sponsor
- Arrowhead Pharmaceuticals · Industry
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About This Trial
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…
Trial Locations
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Eligibility Criteria
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 electrocardiog…
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