Status:
COMPLETED
Phase 1 Trial of Idebenone to Treat Patients With Friedreich's Ataxia
Lead Sponsor:
National Institute of Neurological Disorders and Stroke (NINDS)
Conditions:
Friedreich Ataxia
Eligibility:
All Genders
Phase:
PHASE1
Brief Summary
This study will determine the highest amount of idebenone that can be taken without harmful side effects in children, teenagers, and adults with Friedreich's ataxia, a progressive degenerative disease...
Detailed Description
Background: Friedreich's ataxia (FRDA) is a progressive, autosomal recessive, multisystem degenerative disease for which there is currently no effective treatment. Recent studies have demonstrated tha...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- Diagnosis of FRDA with confirmed FRDA mutations.
- Age greater than or equal to five years.
- No exposure to idebenone or coenzyme Q(10) for a period of at least one week before onset of the medication phase of the study.
- Written, informed consent (and assent, if applicable).
- EXCLUSION CRITERIA:
- History of hypersensitivity reaction to idebenone or coenzyme Q(10).
- 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.
- Lactose intolerant individuals (because of the lactose content in the tablet ingredients).
- Age less than five years old.
- Platelet count, lymphocyte count or hemoglobin below the lower limit of normal.
- Alkaline phosphatase, SGOT, or SGPT greater than 1.5 x the upper limit of normal. Bilirubin greater than 1.2 g/dl.
- Creatinine greater than 1.5 x the upper limit of normal.
- 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 :
February 1 2004
Trial Type :
INTERVENTIONAL
End Date :
April 1 2006
Estimated Enrollment :
16 Patients enrolled
Trial Details
Trial ID
NCT00078481
Start Date
February 1 2004
End Date
April 1 2006
Last Update
March 4 2008
Active Locations (1)
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1
National Institute of Neurological Disorders and Stroke (NINDS)
Bethesda, Maryland, United States, 20892