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 greater than 0.90 to detect a slowing to 25% of the expected rate of progression of weakness at P less than 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 placebo-treated patients, no significant effect on IGF-I levels was observed. Mazindol doses not slow the progression of weakness in Duchenne dystrophy.
Griggs
R C
RC
Department of Neurology, University of Rochester, New York.
Moxley
R T
RT
Mendell
J R
JR
Fenichel
G M
GM
Brooke
M H
MH
Miller
P J
PJ
Mandel
S
S
Florence
J
J
Schierbecker
J
J
Kaiser
K K
KK
D001519:Behavior / Q000187:drug effects; D004311:Double-Blind Method; D001068:Feeding and Eating Disorders / Q000139:chemically induced; D006801:Humans; D008297:Male; D008454:Mazindol / Q000009:adverse effects / Q000627:therapeutic use*; D009119:Muscle Contraction / Q000187:drug effects; D009136:Muscular Dystrophies / Q000188:drug therapy*; D013997:Time Factors; D014987:Xerostomia / Q000139:chemically induced
D008454:Mazindol