Iron-Chelating Therapy and Friedreich Ataxia

Friedreich Ataxia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00224640
• Other study ID #: P041201
• Secondary ID: PCR 05001
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: September 16, 2005
• Last updated: March 3, 2009
• Start date: March 2005
• Est. completion date: March 2008

Study information

• Verified date: March 2007
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Ministry of Health
• Study type: Interventional

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: March 2008
• Est. primary completion date: March 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 13 Years and older

Eligibility

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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Friedreich Ataxia

Intervention

Drug:
• Iron chelating intervention
Iron chelating intervention

Locations

Country	Name	City	State
France	Necker Hospital	Paris

Sponsors (1)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	assessment of iron overload at TO and month2 by imagery		at months :0, 1 ,2 ,4 ,6	No
Secondary	Clinical (monthly) and biological parameter follow- up ( blood count,		weekly	No
Secondary	plasma iron, ferritin, transferrin and liver enzymes)		every months	Yes