NCT00224640

Iron-Chelating Therapy and Friedreich Ataxia

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

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: Assistance Publique - Hôpitaux de Paris · Academic / Other
• Sex: All
• Age: 13 Years
• Healthy volunteers: Not accepted

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

Conditions

• Friedreich Ataxia

Interventions

Type	Name	Description
DRUG	Iron chelating intervention	Iron chelating intervention

Timeline

• Start date: 2005-03-01
• Primary completion: 2008-03-01
• Completion: 2008-03-01
• First posted: 2005-09-23
• Last updated: 2009-03-04

Locations

1 site across 1 country: France