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

Phase 2

Completed

• Conditions: Friedreich's Ataxia
• Interventions: Drug: alpha-tocopherolquinone (A0001); Drug: placebo

• Registration Number: NCT01035671
• Lead Sponsor: Penwest Pharmaceuticals Co.

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2009-12
• Study First Submitted: 2009-12-17
• Study First Submitted QC: 2009-12-18
• Study First Posted: 2009-12-21
• Primary Completion: 2011-02
• Study Completion: 2011-03
• Status Verified: 2011-04
• Last Update Submitted: 2011-04-21
• Last Update Posted: 2011-04-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 42

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Low Dose	alpha-tocopherolquinone (A0001)	A0001 (0.5 g BID)
Placebo	placebo	Placebo
High Dose	alpha-tocopherolquinone (A0001)	A0001 (0.75 g BID)

Primary Outcome Measures

Name	Time	Method
Change in Disposition Index of Glucose Regulation, determined by Frequent Sampling IV Glucose Tolerance Test	Baseline, Day 14 and Day 28

Secondary Outcome Measures

Name	Time	Method
HbA1C	Baseline, Day 14 and Day 28
Plasma 1,5-anhydroglucitol (1,5-AG) (Glycomark)	Baseline, Day 14 and Day28
Timed 25 Foot Walk Test	Baseline and Day 28
9-Hole Peg Test	Baseline, Day 14 and Day 28
Activities of Daily Living	Baseline and Day 28
SF-36®	Baseline and Day 28
Specific Activity of Complex 1 in whole blood	Baseline, Day 14 and Day 28
Sensitivity index (SI) calculated from IVGTT	Baseline, Day 14 and Day 28
Glucose effectiveness (SG) calculated from IVGTT	Baseline, Day 14 and Day 28
AIRg for glucose and insulin during IVGTT	Baseline, Day 14 and Day 28
Fasting Glucose, Insulin and Lactate	Baseline, Day 14 and Day 28
Vision Low Contrast Letter Acuity Test	Baseline and Day 28
Modified Fatigue Impact Scale	Baseline and Day 28
FARS/neurological exam	Baseline and Day 28
Global Impression of Clinical Severity	Baseline, Day 14 and Day 28

Trial Locations

Locations (1)

The Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States