Researchers are looking for people with facioscapulohumeral muscular dystrophy (FSHD) to participate in a phase 2 clinical trial, being conducted by Acceleron Pharma, to test the investigational drug ACE-083.
ACE-083 is a drug that inhibits selected proteins that are involved in the regulation of muscle size and strength. It is administered via intramuscular injection and is designed to increase muscle size and strength specifically in the muscles into which the drug is administered. Acceleron is developing ACE-083 for diseases, like FSHD, in which improved muscle strength in a specific set of muscles may provide clinical benefit.
The goals of the study are to evaluate the safety, tolerability and efficacy of ACE-083 in people with FSHD.
The study will take place in two parts, the first of which is a dose-escalation study to evaluate the safety and tolerability of ACE-083 in up to 36 FSHD patients. In this part of the study, all participants will receive ACE-038. In the second part of the study, up to 40 additional participants will be randomly assigned to take either ACE-083 or a placebo. This part of the study will help investigators assess whether treatment with the drug increases muscle size, strength and function.
Part one of the study will take place at multiple sites in the United States. Part two will involve sites in the United States, Canada and the European Union. Travel expenses for mileage, tolls and parking will be covered. Depending on distance to the site, participants may also be covered for overnight hotel stays.
Participants must be 18 years or older, have genetically confirmed FSHD or have a clinical diagnosis of FSHD and a first-degree relative with genetically confirmed FSHD, and meet additional criteria.
MDA is not involved with this trial, but has supported foundational research in FSHD with an investment since inception of more than $21 million.
For additional information on this trial, including complete eligibility criteria, visit ClinicalTrials.gov and enter NCT02927080 into the search box. If you are interested in participating in the study, email firstname.lastname@example.org