randomized_placebo_controlled_trial · 2017
Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy: results of a randomized, placebo-controlled clinical trial
Campbell C, McMillan HJ, Mah JK, et al. · Muscle and Nerve
moderatePMID · 27462804Trial · nct01099761Verified
Plain-English summary
The trial showed body-composition signals but was stopped early because of nosebleeds and small dilated surface blood vessels, preventing reliable conclusions about function.
Population
Ambulatory boys with Duchenne muscular dystrophy
Intervention
Subcutaneous ACE-031 every two to four weeks versus placebo
Outcomes measured
Safety|Lean mass|Bone mineral density|Fat mass|Six-minute walk distance
Key findings
Pharmacodynamic body-composition trends were seen, but development was halted after epistaxis and telangiectasia safety findings.
Funding & disclosures
Commercially sponsored ACE-031 development programme with investigator and company-author involvement.
Limitations
Premature termination, small sample, short duration and exploratory functional outcomes.Risk of bias
Moderate to high — randomized and blinded, but early termination and sponsor involvement substantially limit efficacy interpretation.Citation
Campbell C, McMillan HJ, Mah JK, et al. (2017). Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy: results of a randomized, placebo-controlled clinical trial. Muscle and Nerve
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