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Objective: To replicate the beneficial effect of brain-derived neurotrophic factor (BDNF) in 1,135 ALS patients in a multicenter trial.

Background: In a phase I through II study, BDNF appeared to increase survival and retard loss of pulmonary function in ALS patients.

Methods: Patients were randomized to placebo, or 25 or 100 μg/kg BDNF for 9 months.

Results: The study failed to show benefit of BDNF treatment for the primary end points. Survival in patients treated with 25 μg/kg BDNF was identical to placebo, but there was a trend toward increased survival in the 100-μg/kg group. As a whole, survival was better than anticipated when planning the study. The 9-month probability of survival was approximately 85% across all groups. This diminished the power of the study. Among the 60% of patients with baseline forced vital capacity of ≤91%, survival was significantly greater for 100 μg/kg BDNF versus …