Iron-Chelating Therapy and Friedreich Ataxia

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Primary Purpose

Status

Completed

Phase

Phase 1

Locations

France

Study Type

Interventional

Intervention

Iron chelating intervention

About this trial

This is an interventional treatment trial for Friedreich Ataxia focused on measuring Iron-chelating treatment

Eligibility Criteria

13 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Necker Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

1

Arm Description

Iron chelating intervention

Outcomes

Primary Outcome Measures

assessment of iron overload at TO and month2 by imagery

Secondary Outcome Measures

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

plasma iron, ferritin, transferrin and liver enzymes)

Full Information

NCT ID

NCT00224640

First Posted

September 16, 2005

Last Updated

March 3, 2009

Sponsor

Assistance Publique - Hôpitaux de Paris

1. Study Identification

Unique Protocol Identification Number

NCT00224640

Brief Title

Iron-Chelating Therapy and Friedreich Ataxia

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

March 2007

Overall Recruitment Status

Completed

Study Start Date

March 2005 (undefined)

Primary Completion Date

March 2008 (Actual)

Study Completion Date

March 2008 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor

Assistance Publique - Hôpitaux de Paris

4. Oversight

Data Monitoring Committee

Yes

5. 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.

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Friedreich Ataxia

Keywords

Iron-chelating treatment

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

15 (Actual)

8. Arms, Groups, and Interventions

Arm Title

1

Arm Type

Experimental

Arm Description

Iron chelating intervention

Intervention Type

Drug

Intervention Name(s)

Iron chelating intervention

Intervention Description

Iron chelating intervention

Primary Outcome Measure Information:

Title

assessment of iron overload at TO and month2 by imagery

Time Frame

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

Secondary Outcome Measure Information:

Title

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

Time Frame

weekly

Title

plasma iron, ferritin, transferrin and liver enzymes)

Time Frame

every months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

13 Years

Accepts Healthy Volunteers

No

Eligibility 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Arnold MUNNICH, Pr,MD,PhD

Organizational Affiliation

Assistance Publique - Hôpitaux de Paris

Official's Role

Principal Investigator

Facility Information:

Facility Name

Necker Hospital

City

Paris

ZIP/Postal Code

75015

Country

France

Friedreich Ataxia

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

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