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

Friedreich's Ataxia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01035671
• Other study ID #: FRD02
• Status: Completed
• Phase: Phase 2
• First received: December 17, 2009
• Last updated: April 21, 2011
• Start date: December 2009
• Est. completion date: March 2011

Study information

• Verified date: April 2011
• Source: Penwest Pharmaceuticals Co.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 42
• Est. completion date: March 2011
• Est. primary completion date: February 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 60 Years

Eligibility

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

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Friedreich Ataxia
• Friedreich's Ataxia

Intervention

Drug:
• alpha-tocopherolquinone (A0001)
28 days of low dose (1.0 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
• alpha-tocopherolquinone (A0001)
28 days of high dose (1.5 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
• placebo
28 days of placebo oral capsules. Treatment taken twice daily with meals.

Locations

Country	Name	City	State
United States	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Penwest Pharmaceuticals Co.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Disposition Index of Glucose Regulation, determined by Frequent Sampling IV Glucose Tolerance Test		Baseline, Day 14 and Day 28	No
Secondary	Sensitivity index (SI) calculated from IVGTT		Baseline, Day 14 and Day 28	No
Secondary	Glucose effectiveness (SG) calculated from IVGTT		Baseline, Day 14 and Day 28	No
Secondary	AIRg for glucose and insulin during IVGTT		Baseline, Day 14 and Day 28	No
Secondary	Fasting Glucose, Insulin and Lactate		Baseline, Day 14 and Day 28	No
Secondary	HbA1C		Baseline, Day 14 and Day 28	No
Secondary	Plasma 1,5-anhydroglucitol (1,5-AG) (Glycomark)		Baseline, Day 14 and Day28	No
Secondary	Specific Activity of Complex 1 in whole blood		Baseline, Day 14 and Day 28	No
Secondary	Timed 25 Foot Walk Test		Baseline and Day 28	No
Secondary	FARS/neurological exam		Baseline and Day 28	No
Secondary	9-Hole Peg Test		Baseline, Day 14 and Day 28	No
Secondary	Vision Low Contrast Letter Acuity Test		Baseline and Day 28	No
Secondary	Global Impression of Clinical Severity		Baseline, Day 14 and Day 28	No
Secondary	Modified Fatigue Impact Scale		Baseline and Day 28	No
Secondary	Activities of Daily Living		Baseline and Day 28	No
Secondary	SF-36®		Baseline and Day 28	No