Iron-Chelating Therapy and Friedreich Ataxia

March 3, 2009 updated by: Assistance Publique - Hôpitaux de Paris

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

15

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Paris, France, 75015
        • Necker Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

13 years and older (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: 1
Iron chelating intervention
Drug: Iron chelating intervention
Iron chelating intervention

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
assessment of iron overload at TO and month2 by imagery
Time Frame: at months :0, 1 ,2 ,4 ,6
at months :0, 1 ,2 ,4 ,6

Secondary Outcome Measures

Outcome Measure
Time Frame
Clinical (monthly) and biological parameter follow- up ( blood count,
Time Frame: weekly
weekly
plasma iron, ferritin, transferrin and liver enzymes)
Time Frame: every months
every months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Assistance Publique - Hôpitaux de Paris

Investigators

  • Principal Investigator: Arnold MUNNICH, Pr,MD,PhD, Assistance Publique - Hôpitaux de Paris

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2005

Primary Completion (ACTUAL)

March 1, 2008

Study Completion (ACTUAL)

March 1, 2008

Study Registration Dates

First Submitted

September 16, 2005

First Submitted That Met QC Criteria

September 16, 2005

First Posted (ESTIMATE)

September 23, 2005

Study Record Updates

Last Update Posted (ESTIMATE)

March 4, 2009

Last Update Submitted That Met QC Criteria

March 3, 2009

Last Verified

March 1, 2007

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • P041201
  • PCR 05001

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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