Iron-Chelating Therapy and Friedreich Ataxia - Full Text View

Sponsor:	Assistance Publique - Hôpitaux de Paris
Information provided by:	Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier:	NCT00224640

Purpose

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.

Condition	Intervention	Phase
Friedreich Ataxia	Drug: Iron chelating intervention	Phase 1 Phase 2

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Effect of Iron-Chelating Therapy in Friedreich Ataxia. Study Phase I/II

Resource links provided by NLM:

Genetics Home Reference related topics: ataxia neuropathy spectrum childhood myocerebrohepatopathy spectrum Friedreich ataxia infantile-onset spinocerebellar ataxia myoclonic epilepsy myopathy sensory ataxia spinocerebellar ataxia type 1 spinocerebellar ataxia type 2 spinocerebellar ataxia type 3 spinocerebellar ataxia type 6

MedlinePlus related topics: Friedreich's Ataxia Iron

U.S. FDA Resources

Further study details as provided by Assistance Publique - Hôpitaux de Paris:

Primary Outcome Measures:

assessment of iron overload at TO and month2 by imagery [ Time Frame: at months :0, 1 ,2 ,4 ,6 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Clinical (monthly) and biological parameter follow- up ( blood count, [ Time Frame: weekly ] [ Designated as safety issue: No ]
plasma iron, ferritin, transferrin and liver enzymes) [ Time Frame: every months ] [ Designated as safety issue: Yes ]

Enrollment:	15
Study Start Date:	March 2005
Study Completion Date:	March 2008
Primary Completion Date:	March 2008 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: 1 Iron chelating intervention	Drug: Iron chelating intervention Iron chelating intervention Other Name: Iron chelating intervention

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

Eligibility

Ages Eligible for Study:	13 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00224640

Locations

France
Necker Hospital
Paris, France, 75015

Sponsors and Collaborators

Assistance Publique - Hôpitaux de Paris

Investigators

Principal Investigator:

Arnold MUNNICH, Pr,MD,PhD

Assistance Publique - Hôpitaux de Paris

More Information

No publications provided

Responsible Party:	Amel Ouslimany, Department Clinical Research of Developpement
ClinicalTrials.gov Identifier:	NCT00224640 History of Changes
Other Study ID Numbers:	P041201, PCR 05001
Study First Received:	September 16, 2005
Last Updated:	March 3, 2009
Health Authority:	France: Ministry of Health

Keywords provided by Assistance Publique - Hôpitaux de Paris:

Iron-chelating treatment

Additional relevant MeSH terms:

Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinocerebellar Degenerations
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases

Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Iron Chelating Agents
Iron
Chelating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Trace Elements
Micronutrients
Growth Substances
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on May 24, 2012