NCT00647296

Safety and Tolerability Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS)

A 2-Part, Randomized, Double-Blind, Safety and Tolerability Study Evaluating KNS-760704 in Patients With Amyotrophic Lateral Sclerosis (ALS)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 194 (actual)
• Sponsor: Knopp Biosciences · Industry
• Sex: All
• Age: 21 Years – 80 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	Placebo	Placebo: 2 tablets taken orally twice daily
DRUG	Dexpramipexole 50 mg/day	Dexpramipexole: 2 x 12.5 mg tablets taken orally twice daily
DRUG	Dexpramipexole 150 mg/day	Dexpramipexole: 2 x 37.5 mg tablets taken orally twice daily
DRUG	Dexpramipexole 300 mg/day	Dexpramipexole: 2 x 75 mg tablets taken orally twice daily

Timeline

• Start date: 2008-04-09
• Primary completion: 2009-07-31
• Completion: 2009-09-04
• First posted: 2008-03-31
• Last updated: 2021-07-08
• Results posted: 2021-07-08

Locations

21 sites across 1 country: United States