Try our beta test site
IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...

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

This study has been completed.
Information provided by:
Penwest Pharmaceuticals Co. Identifier:
First received: December 17, 2009
Last updated: April 21, 2011
Last verified: April 2011

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 Intervention Phase
Friedreich's Ataxia
Drug: alpha-tocopherolquinone (A0001)
Drug: placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (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

Resource links provided by NLM:

Further study details as provided by Penwest Pharmaceuticals Co.:

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

Estimated Enrollment: 42
Study Start Date: December 2009
Study Completion Date: March 2011
Primary Completion Date: February 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.


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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: 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 Identifier (NCT Number):
Responsible Party: Thomas Sciascia, MD/Chief Medical Officer and VP Clinical Operations and Regulatory Affairs, Penwest Pharmaceuticals Co. Identifier: NCT01035671     History of Changes
Other Study ID Numbers: FRD02
Study First Received: December 17, 2009
Last Updated: April 21, 2011

Additional relevant MeSH terms:
Friedreich Ataxia
Cerebellar 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 processed this record on May 24, 2017