NCT01035671

Safety and Efficacy Study of A0001 in Subjects With Friedreich's Ataxia

A Phase 2a, Double-Blind, Randomized, Placebo-Controlled, 28 Day, Three-arm, Parallel Group Study of A0001 in the Treatment of Subjects With Friedreich's Ataxia

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 42 (estimated)
• Sponsor: Penwest Pharmaceuticals Co. · Industry
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2a double-blind, placebo-controlled study with two dose levels of A0001 given twice daily for 28 days. Potential subjects will be screened first to determine eligibility, after which they will be randomized to receive either a high dose of A0001, a low dose of A0001 or placebo for 28 days. Eligible subjects will return within 21 days of screening for the baseline visit and randomization to one of three potential treatments. The subjects will be required to take 3 capsules of study medication in the morning with a morning meal and 3 capsules of study medication at night with an evening meal for 28 days. Additional visits to the clinic are planned for Day 14 and Day 28, at which time a number of clinical and biochemical assessments will be done.

Conditions

• Friedreich's Ataxia

Interventions

Type	Name	Description
DRUG	alpha-tocopherolquinone (A0001)	28 days of low dose (1.0 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
DRUG	alpha-tocopherolquinone (A0001)	28 days of high dose (1.5 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
DRUG	placebo	28 days of placebo oral capsules. Treatment taken twice daily with meals.

Timeline

• Start date: 2009-12-01
• Primary completion: 2011-02-01
• Completion: 2011-03-01
• First posted: 2009-12-21
• Last updated: 2011-04-22

Locations

1 site across 1 country: United States