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

Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age16-70
SponsorArrowhead Pharmaceuticals

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 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

Qualifiers

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

Disqualifiers

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

Trial design

Treatments tested in this trial

  • ARO-DUX4 for Injection
  • Placebo

Treatment groups

60 Participants
are divided into 2 treatment groups

Locations

17
Australia
Research Site 22170, LiverpoolNew South Wales, Australia
Research Site 34066, AuchenflowerQueensland, Australia
Research Site 14575, BirtinyaQueensland, Australia
Research Site 43004, MelbourneVictoria, Australia

Sponsors and collaborators

Arrowhead Pharmaceuticals

Lead sponsor