Double-blind controlled trials of Cronassial in chronic neuromuscular diseases and ataxia.

We report three 12-month, double-blind, three-phase studies comparing the effect of placebo and 40 mg and 100 mg IM daily of purified bovine brain gangliosides (Cronassial) in chronic neuromuscular diseases. Thirty patients with Charcot-Marie-Tooth disease, 16 with idiopathic polyneuropathy, and 30 with spinocerebellar degeneration had neuromuscular function measured monthly by quantitative testing of motor and sensory function, coordination, and electrophysiologic factors. Analysis of these studies, and of longer term (up to 2 years) open studies of 100 mg daily of Cronassial in 67 patients failed to show therapeutic efficacy of Cronassial. Statistical power calculations indicated that five of the 37 measures had greater than a 70% chance of detecting a 20% difference in the rate of progression of the active-drug and placebo groups. A number of measures significantly improved during prolonged placebo treatment, suggesting that the placebo effect has a strong influence on "objective" measures of neuromuscular function.