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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


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
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