Status:
COMPLETED
Idebenone to Treat Friedreich's Ataxia
Lead Sponsor:
National Institute of Neurological Disorders and Stroke (NINDS)
Conditions:
Friedreich Ataxia
Eligibility:
All Genders
9-17 years
Phase:
PHASE2
Brief Summary
This study will determine whether a drug called idebenone is safe and effective in reducing the level of oxidants that are believed to damage the nervous system and hearts in patients with Friedreich'...
Detailed Description
Background: Friedreich's ataxia (FA) is a progressive, autosomal recessive, multisystem degenerative disease for which there is currently no effective treatment. Recent studies have suggested that lip...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- Diagnosis of FA with confirmed FRDA mutations.
- Age from nine up to but not over eighteen years.
- Weight between 30 to 80 kilograms.
- Ambulatory (assistance devices permitted).
- Willing to participate in all aspects of trial design and follow-up.
- All subjects agree and commit to the use of 2 reliable methods of birth control for the duration of the study if sexually active.
- Neurologically symptomatic.
- No exposure to idebenone, coenzyme Q10, or other dietary supplements for a period of at least one month before enrollment in the study.
- EXCLUSION CRITERIA:
- History of a hypersensitivity reaction to idebenone or coenzyme Q10.
- Pregnant or lactating women. All women of child-bearing potential must have negative serum pregnancy prior to the medication phase of the study. If a minor has a positive pregnancy test, we will inform her but not inform her parents unless we are asked to by the minor.
- Platelet count, white blood cell count or hemoglobin below the lower limit of normal.
- Alkaline phosphatase, SGOT, or SGPT greater than 1.5 times the upper limit of normal. Bilirubin greater than 1.5 g/dl.
- Creatinine greater than 1.5 times the upper limit of normal based upon the pediatric reference range provided by the testing laboratory.
- Clinically significant medical disease that, in the judgment of the investigators, would expose the patient to undue risk of harm or prevent the patient from completing the study.
Exclusion
Key Trial Info
Start Date :
September 27 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 17 2007
Estimated Enrollment :
51 Patients enrolled
Trial Details
Trial ID
NCT00229632
Start Date
September 27 2005
End Date
December 17 2007
Last Update
March 18 2019
Active Locations (1)
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1
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892