Study of ARO-DUX4 in Adult Patients With Facioscapulohumeral Muscular Dystrophy Type 1
Status:
Recruiting
Trial end date:
2025-06-01
Target enrollment:
Participant gender:
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 a single dose of ARO-DUX4
or placebo. In Part 2 of the study, participants will receive 2 or 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.