The Rilutek ® (riluzole) Global Early Access Programme: An open-label safety evaluation in the treatment of amyotrophic lateral sclerosis

• Published in: Amyotrophic lateral sclerosis and other motor neuron disorders 2001, Vol.2 (3), p.153-158
• Main Authors: Debove, Catherine, Zeisser, Philippe, Salzman, Phyllis M, Powe, Larry K, Truffinet, Philippe
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Aged; Aged, 80 and over; Alanine Transaminase - blood; Amyotrophic Lateral Sclerosis - blood; Amyotrophic Lateral Sclerosis - drug therapy; Amyotrophic Lateral Sclerosis - mortality; Amyotrophic Lateral Sclerosis Early Access Programme Riluzole Rilutek Safety; Excitatory Amino Acid Antagonists - adverse effects; Excitatory Amino Acid Antagonists - therapeutic use; Female; Humans; Male; Middle Aged; Neutropenia - blood; Neutropenia - chemically induced; Riluzole - adverse effects; Riluzole - therapeutic use; Survival Analysis
• ISSN: 1466-0822
• DOI: 10.1080/146608201753275508

OBJECTIVES: This study had two main objectives: 1. To enable patients with amyotrophic lateral sclerosis (ALS) who had not participated in previous riluzole trials to receive riluzole therapy, and 2. To expand safety experience with the drug in a broad patient population. METHODS: This was a Phase IIIb multicentre, multinational, open-label, uncontrolled single treatment study of riluzole. Patients with diagnosed possible or probable ALS were administered 100 mg of riluzole/day (50 mg b.i.d. ). Clinical and laboratory adverse events were recorded every month for the first 3 months and thereafter at 3-monthly intervals. RESULTS: 8383 patients from 44 countries were entered into the study; 7916 of these patients with recorded data were administered the study drug. The mean duration of riluzole treatment was 202.1 days, with a range of 1-630 days. The most frequently reported serious and non-serious adverse events were common symptoms of ALS (respiratory symptoms and dysphagia), and only 1.9% of serious adverse events were considered to be related to the study drug. CONCLUSIONS: The safety results with this broad population (over 10% of the estimated ALS population worldwide) were consistent with those previously reported from placebo-controlled trials. No increase in adverse events and no unexpected adverse events were observed.