Friedreich's ataxia Nervous System Diseases Friedreich's ataxia
Conditions
Interventions
High doses of coenzyme Q10 / vitamin E vs Low doses of coenzyme Q10 / vitamin E
Sponsors
Royal Free Hospital Trust (UK)
Eligibility
Inclusion criteria
Inclusion criteria: 1. Homozygous for the GAA expansion in the FRDA gene characteristic of the disease 2. Able and willing to attend the assessments over the trial period 3. Over 10 years of age at the start of the trial
Exclusion criteria
Exclusion criteria: 1. Planned surgery during the trial period 2. An ICARS score greater than 80% of the maximum 3. Taking coenzyme Q10, vitamin E or other antioxidants 4. Planned pregnancy
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Significant improvement in the rate of change in the International Cooperative Ataxia Ratings Scale (ICARS) scores over two years of the trial in the high dose versus the low dose treatment groups | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Rate of change in the kinetic, posture and gait, speech, ocular subscores of the ICARS 2. Rate of change in the following: a. speech tests (PaTa repetition, speed of reading) b. upper limb coordination tests (hand clicker, modified peg board, hand target, BRAIN kinesia) c. lower limb coordination tests (foot target, box) d. cardiac tests (IVS, PWd and fraction shortening) e. activities of daily living scale | — |
Countries
United Kingdom
Outcome results
None listed