Status:
COMPLETED
Effect of Pioglitazone Administered to Patients With Friedreich's Ataxia: Proof of Concept
Lead Sponsor:
Assistance Publique - Hôpitaux de Paris
Conditions:
Friedreich's Ataxia
Eligibility:
All Genders
7-24 years
Phase:
PHASE3
Brief Summary
Friedreich's ataxia (FA) is a rare progressive neurological disorder affecting approximately 1/30, 000 individuals. No treatment is presently available to counteract the neurodegeneration of this extr...
Detailed Description
State of the art Friedreich's ataxia (FA) is a rare progressive neurological disorder affecting approximately 1/30, 000 individuals. No treatment is presently available to counteract the neurodegenera...
Eligibility Criteria
Inclusion
- Diagnosis of FA with confirmed FRDA mutations
- GAA repeat length of the shorter allele of frataxin gene \> 300
- Age ≤ 24 years
- Ambulatory (assistance devices permitted) or able to stand up without support
- Neurologically symptomatic
- All subjects agree and commit to the use of 2 reliable methods of birth control for the duration of the study if sexually active
- Willing (and parents if minor) to participate in all aspects of trial design and follow-up
- No modification of the usual treatment 6 months before inclusion and agree to stay with the same treatment during the trial (idebenone with a stable dosage, cardiologic therapeutic)
Exclusion
- Composite heterozygote
- Patients unable to stand up even with support
- Pregnant women
- Cardiac insufficiency NYHA III to IV and heart ejection fraction\> 50%
- Alkaline phosphatase, SGOT or SGPT greater than 1.5 X the upper limit of normal
- Patients with diabetes
- Modification of the concomitant medications taken by the patient within the 6 months before inclusion or during the trial
- 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.
Key Trial Info
Start Date :
December 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2013
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT00811681
Start Date
December 1 2008
End Date
March 1 2013
Last Update
September 4 2013
Active Locations (1)
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1
Hôpital Robert Debré
Paris, France, 75019