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Completed

NCT Number: NCT01035671

Safety and Efficacy Study of A0001 in 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.

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Key information

Conditions

Friedreich's Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Friedreich Ataxia Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Spinal Cord Diseases Spinocerebellar Degenerations

Age range

18 year–60 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

alpha-tocopherolquinone (A0001)

Drug

28 days of low dose (1.0 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.

Placebo

Drug

28 days of placebo oral capsules. Treatment taken twice daily with meals.

Primary outcomes

  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 outcomes

  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

Sponsors and collaborators

Lead sponsor

Penwest Pharmaceuticals Co.

Industry

Registry information

Official study 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

Important dates

Study start
2009
Primary completion
2011
Study completion
2011
First posted
Dec 21, 2009
Registry last updated
Apr 22, 2011

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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