Clinical investigation in Duchenne muscular dystrophy: penicillamine and vitamin E.

Author(s): Fenichel GM, Brooke MH, Griggs RC, Mendell JR, Miller JP, Moxley RT 3rd, Park JH, Provine MA, Florence J, Kaiser KK

Affiliation(s): Department of Neurology, Vanderbilt University School of Medicine, Nashville, TN 37212.

Publication date & source: 1988-11, Muscle Nerve., 11(11):1164-8.

Publication type: Clinical Trial; Randomized Controlled Trial

A double-blind controlled trial of penicillamine and vitamin E against placebo was conducted in 106 boys with Duchenne muscular dystrophy. Nine dropped out of the study after being randomized. Patients were evaluated using an established protocol that measures muscle strength, joint contractures, functional grade, and pulmonary function. The trial lasted for 18 months. The power of the study to detect a 75% slowing of the disease was 0.95 at a P less than 0.05. No therapeutic effect was observed in the patients taking penicillamine and vitamin E as compared with those taking placebo.