Randomized, double-blind trial of mazindol in Duchenne dystrophy

Area of Science:

• Neurology
• Endocrinology
• Pediatrics

Background:

• Growth hormone's potential role in Duchenne muscular dystrophy (DMD) progression is under investigation.
• Mazindol, a potential inhibitor of growth hormone secretion, was explored as a therapeutic agent.

Purpose of the Study:

• To evaluate the efficacy of mazindol in slowing the progression of weakness in Duchenne muscular dystrophy.
• To assess the impact of mazindol on muscle strength, functional ability, and pulmonary function in DMD patients.

Main Methods:

• A 12-month, randomized, placebo-controlled trial involving 83 boys with Duchenne muscular dystrophy.
• Participants received either mazindol (3 mg/d) or a placebo, with assessments at baseline, 6 months, and 12 months.
• Evaluated outcomes included muscle strength, contractures, functional ability, pulmonary function, and Insulin-like Growth Factor-I (IGF-I) levels.

Main Results:

• Mazindol did not demonstrate any significant benefit in improving muscle strength at any time point during the study.
• No significant effect on Insulin-like Growth Factor-I (IGF-I) levels was observed, despite mazindol-treated patients gaining less weight and height.
• Reported side effects included decreased appetite, dry mouth, behavioral changes, and gastrointestinal symptoms, leading to dosage reduction in 43% of patients.

Conclusions:

• Mazindol, at the tested dosage, does not slow the progression of weakness in Duchenne muscular dystrophy.
• The drug did not show a significant effect on growth hormone secretion markers (IGF-I) in this patient population.
• Further research may be needed to explore other therapeutic avenues for managing Duchenne muscular dystrophy progression.