Friedreich's Ataxia
Conditions
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.
Interventions
DRUGalpha-tocopherolquinone (A0001)
28 days of low dose (1.0 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
DRUGplacebo
28 days of placebo oral capsules. Treatment taken twice daily with meals.
Sponsors
Penwest Pharmaceuticals Co.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 60 Years
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in Disposition Index of Glucose Regulation, determined by Frequent Sampling IV Glucose Tolerance Test | Baseline, Day 14 and Day 28 |
Secondary
| Measure | Time frame |
|---|---|
| Glucose effectiveness (SG) calculated from IVGTT | Baseline, Day 14 and Day 28 |
| AIRg for glucose and insulin during IVGTT | Baseline, Day 14 and Day 28 |
| Fasting Glucose, Insulin and Lactate | Baseline, Day 14 and Day 28 |
| HbA1C | Baseline, Day 14 and Day 28 |
| Plasma 1,5-anhydroglucitol (1,5-AG) (Glycomark) | Baseline, Day 14 and Day28 |
| Specific Activity of Complex 1 in whole blood | Baseline, Day 14 and Day 28 |
| Timed 25 Foot Walk Test | Baseline and Day 28 |
| Sensitivity index (SI) calculated from IVGTT | Baseline, Day 14 and Day 28 |
| 9-Hole Peg Test | Baseline, Day 14 and Day 28 |
| Vision Low Contrast Letter Acuity Test | Baseline and Day 28 |
| Global Impression of Clinical Severity | Baseline, Day 14 and Day 28 |
| Modified Fatigue Impact Scale | Baseline and Day 28 |
| Activities of Daily Living | Baseline and Day 28 |
| SF-36® | Baseline and Day 28 |
| FARS/neurological exam | Baseline and Day 28 |
Countries
United States
Outcome results
None listed