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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01035671
Recruitment Status : Completed
First Posted : December 21, 2009
Last Update Posted : April 22, 2011
Information provided by:

Study Description
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.

Condition or disease Intervention/treatment Phase
Friedreich's Ataxia Drug: alpha-tocopherolquinone (A0001) Drug: placebo Phase 2

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 42 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Study Start Date : December 2009
Primary Completion Date : February 2011
Study Completion Date : March 2011

Arms and Interventions

Arm Intervention/treatment
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.
Experimental: High Dose
A0001 (0.75 g BID)
Drug: alpha-tocopherolquinone (A0001)
28 days of high dose (1.5 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
Placebo Comparator: Placebo
Drug: placebo
28 days of placebo oral capsules. Treatment taken twice daily with meals.

Outcome Measures

Primary Outcome Measures :
  1. Change in Disposition Index of Glucose Regulation, determined by Frequent Sampling IV Glucose Tolerance Test [ Time Frame: Baseline, Day 14 and Day 28 ]

Secondary Outcome Measures :
  1. Sensitivity index (SI) calculated from IVGTT [ Time Frame: Baseline, Day 14 and Day 28 ]
  2. Glucose effectiveness (SG) calculated from IVGTT [ Time Frame: Baseline, Day 14 and Day 28 ]
  3. AIRg for glucose and insulin during IVGTT [ Time Frame: Baseline, Day 14 and Day 28 ]
  4. Fasting Glucose, Insulin and Lactate [ Time Frame: Baseline, Day 14 and Day 28 ]
  5. HbA1C [ Time Frame: Baseline, Day 14 and Day 28 ]
  6. Plasma 1,5-anhydroglucitol (1,5-AG) (Glycomark) [ Time Frame: Baseline, Day 14 and Day28 ]
  7. Specific Activity of Complex 1 in whole blood [ Time Frame: Baseline, Day 14 and Day 28 ]
  8. Timed 25 Foot Walk Test [ Time Frame: Baseline and Day 28 ]
  9. FARS/neurological exam [ Time Frame: Baseline and Day 28 ]
  10. 9-Hole Peg Test [ Time Frame: Baseline, Day 14 and Day 28 ]
  11. Vision Low Contrast Letter Acuity Test [ Time Frame: Baseline and Day 28 ]
  12. Global Impression of Clinical Severity [ Time Frame: Baseline, Day 14 and Day 28 ]
  13. Modified Fatigue Impact Scale [ Time Frame: Baseline and Day 28 ]
  14. Activities of Daily Living [ Time Frame: Baseline and Day 28 ]
  15. SF-36® [ Time Frame: Baseline and Day 28 ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01035671

United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Penwest Pharmaceuticals Co.
Principal Investigator: David Lynch, MD, PhD Children's Hospital of Philadelphia
More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Thomas Sciascia, MD/Chief Medical Officer and VP Clinical Operations and Regulatory Affairs, Penwest Pharmaceuticals Co.
ClinicalTrials.gov Identifier: NCT01035671     History of Changes
Other Study ID Numbers: FRD02
First Posted: December 21, 2009    Key Record Dates
Last Update Posted: April 22, 2011
Last Verified: April 2011

Additional relevant MeSH terms:
Cerebellar Ataxia
Friedreich Ataxia
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Vitamin E
Antihypertensive Agents
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Growth Substances