Iron-Chelating Therapy and Friedreich Ataxia

Effect of Iron-Chelating Therapy in Friedreich Ataxia. Study Phase I/II

Status

Completed

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00224640

Enrollment

Registered

2005-09-23

Start date

2005-03-31

Completion date

2008-03-31

Last updated

2009-03-04

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Friedreich Ataxia

Keywords

Iron-chelating treatment

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 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.

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

Interventions

DRUGIron chelating intervention

Iron chelating intervention

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

13 Years to No maximum

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame
assessment of iron overload at TO and month2 by imagery	at months :0, 1 ,2 ,4 ,6

Secondary

Measure	Time frame
Clinical (monthly) and biological parameter follow- up ( blood count,	weekly
plasma iron, ferritin, transferrin and liver enzymes)	every months

Countries

France

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026