Randomized, double‐blind trial of mazindol in Duchenne dystrophy

There is evidence that growth hormone may be related to the progression of weakness in Duchenne dystrophy. We conducted a 12‐month controlled trial of mazindol, a putative growth hormone secretion inhibitor, in 83 boys with Duchenne dystrophy. Muscle strength, contractures, functional ability and pulmonary function were tested at baseline, and 6 and 12 months after treatment with mazindol (3 mg/d) or placebo. The study was designed to have a power of >0.90 to detect a slowing to 25% of the expected rate of progression of weakness at P < 0.05. Mazindol did not benefit strength at any point in the study. Side effects attributable to mazindol included decreased appetite (36%), dry mouth (10%), behavioral change (22%), and gastrointestinal symptoms (18%); mazindol dosage was reduced in 43% of patients. The effect of mazindol on GH secretion was estimated indirectly by comparing the postabsorptive IGF‐I levels obtained following 3, 6, 9, and 12 months in the mazindol treated to those in the placebo groups. Although mazindol‐treated patients gained less weight and height than placebotreated patients, no significant effect on IGF‐I levels was observed. Mazindol does not slow the progression of weakness in Duchenne dystrophy.