Status:
COMPLETED
Iron-Chelating Therapy and Friedreich Ataxia
Lead Sponsor:
Assistance Publique - Hôpitaux de Paris
Conditions:
Friedreich Ataxia
Eligibility:
All Genders
13+ years
Phase:
PHASE1
PHASE2
Brief Summary
Friedreich ataxia, an autosomal recessive condition, ascribed to frataxin gene expansion, has been shown to result from an iron- induced injury to the mitochondrial respiratory chain. Buffering free r...
Detailed Description
The current clinical trial is a monocentric open phase1-2 trial in the context of rare diseases framework, aimed to the goal of defining the tolerance/efficacy of the treatment. Inclusion criteria: m...
Eligibility Criteria
Inclusion
- Minimum age: 13 years
- Molecular confirmation of frataxin gene mutation
- Iron overload evaluation
- Presence of lactate
- Echography response to Idebenone treatment
- Urinary test of pregnancy for girls
- Sexual abstinence for men
- Information consent
Exclusion
- No disturbance of iron metabolism
- No response to Idebenone
- Friedreich not confirmed
- Polynuclear neutrophils \<2 x 109/L or hemoglobin \< 8g/dL
- No participation to other trial
- Doubt regarding the compliance of the patient to protocol
- Impossibility to undergo X-ray examination or presence of iron material in the backbone
- Pregnant women
- Absence of social insurance.
Key Trial Info
Start Date :
March 1 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2008
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT00224640
Start Date
March 1 2005
End Date
March 1 2008
Last Update
March 4 2009
Active Locations (1)
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1
Necker Hospital
Paris, France, 75015