Trial | NCT01035671  Report issue

Title

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

  • Phase

    Phase 2

  • Study Type

    Interventional

  • Status

    Completed No Results Posted

  • Intervention/Treatment

    vitamin e ...

  • Study Participants

    42
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.
Study Started
Dec 31
2009
Primary Completion
Feb 28
2011
Study Completion
Mar 31
2011
Last Update
Apr 22
2011
Estimate

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.

Low Dose Experimental

A0001 (0.5 g BID)

High Dose Experimental

A0001 (0.75 g BID)

Placebo Placebo Comparator

Placebo

Criteria 

Inclusion Criteria:

Individuals with genetically confirmed Friedreich's Ataxia (GAA or point mutation)
Impaired Glucose Tolerance, measured by Oral GTT

Exclusion Criteria:

Overt Diabetes Mellitus
Presence of clinically significant cardiovascular disease
No Results Posted