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Completed

NCT Number: NCT00224640

Iron-Chelating Therapy and Friedreich Ataxia

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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Key information

Conditions

Friedreich Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Spinal Cord Diseases Spinocerebellar Degenerations

Age range

13 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Necker Hospital

Paris, 75015, France

About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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 criteria

  • 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.

Treatment and study plan

Iron chelating intervention

Drug

Iron chelating intervention

Primary outcomes

  1. assessment of iron overload at TO and month2 by imagery

    Time frame: at months :0, 1 ,2 ,4 ,6

Secondary outcomes

  1. Clinical (monthly) and biological parameter follow- up ( blood count,

    Time frame: weekly

  2. plasma iron, ferritin, transferrin and liver enzymes)

    Time frame: every months

Sponsors and collaborators

Lead sponsor

Assistance Publique - Hôpitaux de Paris

Other

Registry information

Official study title

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

Important dates

Study start
2005
Primary completion
2008
Study completion
2008
First posted
Sep 23, 2005
Registry last updated
Mar 4, 2009

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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