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

April 21, 2011 updated by: Penwest Pharmaceuticals Co.

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

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 Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

42

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • The Children's Hospital of Philadelphia

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 60 years (Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Drug: placebo
28 days of placebo oral capsules. Treatment taken twice daily with meals.
Experimental: Low Dose
A0001 (0.5 g BID)
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.
Experimental: High Dose
A0001 (0.75 g BID)
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change in Disposition Index of Glucose Regulation, determined by Frequent Sampling IV Glucose Tolerance Test
Time Frame: Baseline, Day 14 and Day 28
Baseline, Day 14 and Day 28

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Penwest Pharmaceuticals Co.

Investigators

  • Principal Investigator: David Lynch, MD, PhD, Children's Hospital of Philadelphia

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2009

Primary Completion (Actual)

February 1, 2011

Study Completion (Actual)

March 1, 2011

Study Registration Dates

First Submitted

December 17, 2009

First Submitted That Met QC Criteria

December 18, 2009

First Posted (Estimate)

December 21, 2009

Study Record Updates

Last Update Posted (Estimate)

April 22, 2011

Last Update Submitted That Met QC Criteria

April 21, 2011

Last Verified

April 1, 2011

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • FRD02

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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