Iron-Chelating Therapy and Friedreich Ataxia

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-03-31

Study Completion Date

2008-03-31

Brief Summary

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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 radicals with short-chain quinones (Idebenone) protects the patients against cardiomyopathy but not CNS involvement. Removing CNS iron should limit the impact of the neurological symptoms of the disease.

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Detailed Description

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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: minimum age: 13 years Follow up in the Dept of Genetics, Hospital Necker-Enfants Malades, Paris, France

Conditions

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Friedreich Ataxia

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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1

Iron chelating intervention

Group Type EXPERIMENTAL

Iron chelating intervention

Intervention Type DRUG

Iron chelating intervention

Interventions

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Iron chelating intervention

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Minimum age: 13 years
2. Molecular confirmation of frataxin gene mutation
3. Iron overload evaluation
4. Presence of lactate
5. Echography response to Idebenone treatment
6. Urinary test of pregnancy for girls
7. Sexual abstinence for men
8. Information consent

Exclusion Criteria

1. No disturbance of iron metabolism
2. No response to Idebenone
3. Friedreich not confirmed
4. Polynuclear neutrophils \<2 x 109/L or hemoglobin \< 8g/dL
5. No participation to other trial
6. Doubt regarding the compliance of the patient to protocol
7. Impossibility to undergo X-ray examination or presence of iron material in the backbone
8. Pregnant women
9. Absence of social insurance.

Minimum Eligible Age

13 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No