Iron-Chelating Therapy and Friedreich Ataxia

• Sponsor: Assistance Publique - Hôpitaux de Paris (Other)
• Overall Status: Completed
• CT.gov ID: NCT00224640
• Collaborator: (none)
• Enrollment: 15
• Location: 1
• Arm: 1
• Duration (Months): 36
• Patients Per Site Per Month: 0.4

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Friedreich Ataxia	Drug: Iron chelating intervention	Phase 1/Phase 2

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

Study Design

• Study Type: Interventional
• Actual Enrollment: 15 participants
• Allocation: Non-Randomized
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Effect of Iron-Chelating Therapy in Friedreich Ataxia. Study Phase I/II
• Study Start Date: Mar 1, 2005
• Actual Primary Completion Date: Mar 1, 2008
• Actual Study Completion Date: Mar 1, 2008

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 1 Iron chelating intervention	Drug: Iron chelating intervention; Iron chelating intervention

Outcome Measures

Primary Outcome Measures

1. assessment of iron overload at TO and month2 by imagery [at months :0, 1 ,2 ,4 ,6]

Secondary Outcome Measures

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

2. plasma iron, ferritin, transferrin and liver enzymes) [every months]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 13 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• ClinicalTrials.gov Identifier: NCT00224640
• Other Study ID Numbers: P041201; PCR 05001
• First Posted: Sep 23, 2005
• Last Update Posted: Mar 4, 2009
• Last Verified: Mar 1, 2007

• Keywords provided by , ,: Iron-chelating treatment
• Additional relevant MeSH terms: Ataxia; Cerebellar Ataxia; Friedreich Ataxia; Iron Chelating Agents

Study Results

No Results Posted as of Mar 4, 2009