randomized_phase_2_trial · 2022
Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy
Statland JM|Campbell C|Desai U|Karam C|Díaz-Manera J|Guptill JT|Korngut L|Genge A|Tawil RN|Elman L|Joyce NC|Wagner KR|Manousakis G|Amato AA|Butterfield RJ|Shieh PB|Wicklund M|Gamez J|Bodkin C|Pestronk A|Weihl CC|Vilchez-Padilla JJ|Johnson NE|Mathews KD|Miller B|Leneus A|Fowler M|van de Rijn M|Attie KM · Muscle & Nerve
moderateDOI · 10.1002/mus.27558PMID · 35428982Verified
Plain-English summary
ACE-083 increased treated-muscle volume, but the larger muscles did not produce consistent improvements in strength, function or patient-reported outcomes.
Population
Adults with FSHD1 or FSHD2
Sample size
95
Intervention
ACE-083 injected bilaterally into biceps brachii or tibialis anterior every 3 weeks
Outcomes measured
Muscle volume|Contractile muscle volume|Fat fraction|Strength|Functional tests|Patient-reported outcomes|Safety
Key findings
Muscle volume increased significantly versus placebo, while functional and patient-reported benefits were inconsistent.
Funding & disclosures
Acceleron Pharma funded the study; several authors were employees or stockholders and multiple investigators reported industry relationships.
Limitations
Disease-specific population|Local intramuscular administration|No consistent functional benefit|Development programme discontinuedRisk of bias
Moderate — randomized and blinded in part 2, but sponsor involvement and endpoint limitations apply.Citation
Statland JM|Campbell C|Desai U|Karam C|Díaz-Manera J|Guptill JT|Korngut L|Genge A|Tawil RN|Elman L|Joyce NC|Wagner KR|Manousakis G|Amato AA|Butterfield RJ|Shieh PB|Wicklund M|Gamez J|Bodkin C|Pestronk A|Weihl CC|Vilchez-Padilla JJ|Johnson NE|Mathews KD|Miller B|Leneus A|Fowler M|van de Rijn M|Attie KM (2022). Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy. Muscle & Nerve. https://doi.org/10.1002/mus.27558
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