Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS Amyotrophic Lateral Sclerosis Lou Gehrig Lou Gehrig's Lou Gehrig's disease Motor Neuron Disease Nervous System Diseases KNS-760704 BIIB050
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 194
Healthy volunteers: no

Brief summary:
This was a 2-part study of dexpramipexole in patients with ALS.

Part 1 was a randomized, placebo-controlled, multi-center study to evaluate the safety, tolerability, and clinical effects of oral administration of 3 dosage levels of dexpramipexole vs. placebo for 12 weeks.

Part 2 was a randomized, double-blind, 2-arm, parallel group, extension study evaluating the safety, tolerability, and clinical effects of oral administration of 2 dosage levels of dexpramipexole for up to 72 weeks.

Detailed description:
This study was a two-part, multicenter, double-blind study in subjects with ALS to evaluate the safety and tolerability of dexpramipexole treatment, as well as the preliminary effects on measures of clinical function and mortality of dexpramipexole treatment.

In part 1, 102 subjects with ALS were randomized at 20 US sites to receive placebo, dexpramipexole at 50 mg/day; dexpramipexole at 150 mg/day; or dexpramipexole at 300 mg/day for 12 weeks. Participants who completed Part 1 were eligible to enroll into Part 2.

Part 2 was a randomized, double-blind, 2-arm, parallel-group, extension study evaluating the longer-term safety, tolerability, and clinical effects of oral administration of 2 dosage levels of dexpramipexole. In part 2, following a 4-week, placebo washout, continuing subjects received dexpramipexole at 50 mg/day or 300 mg/day as double-blind treatment for up to 72 additional weeks (Part 2 duration was up to a total of 76 weeks, including the 4 week placebo portion).

Arms:
arm 1: During Part 1, subjects received twice daily doses of dexpramipexole (50 mg/day, 150 mg/day, or 300 mg/day) or matching placebo for approximately 12 weeks. arm 2: At the beginning of Part 2, subjects received twice daily doses of placebo for approximately 4 weeks. arm 3: Following the Part 2 placebo washout, subjects received dexpramipexole (50 mg/day or 300 mg/day), subjects received twice daily doses of placebo for up to 18 months.

Interventions: intervention 1: Placebo intervention 2: Dexpramipexole 50 mg/day intervention 3: Dexpramipexole 150 mg/day intervention 4: Dexpramipexole 300 mg/day
Intervention descriptions: intervention 1: Placebo: 2 tablets taken orally twice daily intervention 2: Dexpramipexole: 2 x 12.5 mg tablets taken orally twice daily intervention 3: Dexpramipexole: 2 x 37.5 mg tablets taken orally twice daily intervention 4: Dexpramipexole: 2 x 75 mg tablets taken orally twice daily

Protocol document: