Jennifer McNary, Head of Patient Advocacy and Engagement at Fulcrum Therapeutics, and mother to two boys with Duchenne muscular dystrophy, discusses the importance of clinically meaningful primary outcomes in clinical trials.
Fulcrum Therapeutics is enrolling patients with facioscapulohumeral muscular dystrophy (FSDH) to test their drug, losmapimod, as a possible treatment.
FSHD is a rare, progressive musculoskeletal disease in which persons initially lose muscle strength in the face, shoulders, arms, and trunk, but later the disease progresses throughout the lower body. FSHD is due to the mis-expression of DUX4 in skeletal muscles. There are currently no approved treatments for the disease.
Ms. McNary, who was instrumental in getting the first drug approved for Duchenne, (eteplirsen [Exondys 51] discusses how researchers are often “bullied” into using well-understood endpoint measures, (like the 6 minute walk test in the case of eteplirsen,) even if these measures are not particularly important to patients or caregivers. Additionally, it is not necessary for companies developing drugs to consult with patient advocacy groups, which would give them insight into what outcomes would be clinically significant. This is one reason why Fulcrum Therapeutics is using reachable workspace (RWS), which evaluates shoulder and proximal arm mobility, as the primary outcome measure for the REACH trial; FSHD causes progressive loss of range of motion in patients’ shoulders and arms, which impacts basic daily tasks such as cooking, getting dressed, opening the door, etc. and it was noted by FSHD patients Fulcrum Therapeutics spoke to that any drug approved in the future for the disease improve range of motion in the arms and shoulders.
To learn more about FSHD and other rare musculoskeletal diseases, visit checkrare.com/diseases/musculoskeletal-diseases/
