Condition: Facio-Scapulo-Humeral Dystrophy · Sponsor: Arrowhead Pharmaceuticals
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.
This description comes directly from the study's public registry record.
Medical Monitor · 626-304-3400 · ARO_DUX4@arrowheadpharma.com
Always discuss trial participation with your own doctor first.
| Research Site 2 | Liverpool, New South Wales, Australia | Recruiting |
| Research Site 3 | Auchenflower, Queensland, Australia | Recruiting |
| Research Site 1 | Birtinya, Queensland, Australia | Recruiting |
| Research Site 4 | Melbourne, Victoria, Australia | Recruiting |
| Research Site 2 | Calgary, Alberta, Canada | Not Yet Recruiting |
| Research Site 3 | Edmonton, Alberta, Canada | Recruiting |
| Research Site 1 | Montreal, Quebec, Canada | Recruiting |
| Research Site 2 | München, Germany | Recruiting |
| Research Site 1 | Ulm, Germany | Recruiting |
| Research Site 1 | Milan, Italy | Recruiting |
| Research Site 2 | Roma, Italy | Recruiting |
| Research Site 1 | Leiden, Netherlands | Recruiting |
+ 5 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT06131983