NCT00224640

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Mar 2005

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2005

Completed
7 months until next milestone

First Submitted

Initial submission to the registry

September 16, 2005

Completed
7 days until next milestone

First Posted

Study publicly available on registry

September 23, 2005

Completed
2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2008

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2008

Completed
Last Updated

March 4, 2009

Status Verified

March 1, 2007

Enrollment Period

3 years

First QC Date

September 16, 2005

Last Update Submit

March 3, 2009

Conditions

Friedreich Ataxia

Keywords

Iron-chelating treatment

Outcome Measures

Primary Outcomes (1)

  • assessment of iron overload at TO and month2 by imagery

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

Secondary Outcomes (2)

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

    weekly

  • plasma iron, ferritin, transferrin and liver enzymes)

    every months

Study Arms (1)

1

EXPERIMENTAL

Iron chelating intervention

Drug: Iron chelating intervention

Interventions

Iron chelating interventionDRUG

Iron chelating intervention

1

Eligibility Criteria

Age13 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Necker Hospital

Paris, 75015, France

Location

MeSH Terms

Conditions

Friedreich Ataxia

Condition Hierarchy (Ancestors)

Spinocerebellar DegenerationsCerebellar DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesSpinal Cord DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMitochondrial DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Arnold MUNNICH, Pr,MD,PhD

    Assistance Publique - Hôpitaux de Paris

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

September 16, 2005

First Posted

September 23, 2005

Study Start

March 1, 2005

Primary Completion

March 1, 2008

Study Completion

March 1, 2008

Last Updated

March 4, 2009

Record last verified: 2007-03

Locations

FR(1)