Friedreich AtaxiaPhase 1/2NCT00224640
Iron-Chelating Therapy and Friedreich Ataxia
A Phase 1/2 study testing Iron chelating intervention in people with Friedreich Ataxia, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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. Read more about this study
- Who is it for?
- Ages 13 years and older
- Conditions the study is for
- Friedreich Ataxia
- What is being tested?
- Iron chelating intervention
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteFrance
- Study dates
- Mar 2005to Mar 2008
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 13 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- Impossibility to undergo X-ray examination or presence of iron material in the backbone
- Pregnant women
- Absence of social insurance.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
Read the 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
- Conditions
- Friedreich Ataxia
- Being tested
- Iron chelating intervention
- Keywords
- Iron-chelating treatment
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Iron chelating interventionDrug
Iron chelating intervention
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Iron chelating intervention Receives: Drug: Iron chelating intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
assessment of iron overload at TO and month2 by imagery
Measured: at months :0, 1 ,2 ,4 ,6
Secondary outcomes (2)
Clinical (monthly) and biological parameter follow- up ( blood count,
Measured: weekly
plasma iron, ferritin, transferrin and liver enzymes)
Measured: every months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2005 (estimated)
- Study startMar 2005
- Primary completionMar 2008
- Study completionMar 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich AtaxiaStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Investigators
- Arnold MUNNICH, Pr,MD,PhD, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00224640
- Sponsor's protocol ID
- P041201
- Other IDs
- PCR 05001
- Status last verified
- Mar 2007
- First submitted
- 16 Sep 2005
- Last updated
- 4 Mar 2009
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Iron-Chelating Therapy in Friedreich Ataxia. Study Phase I/II
- ClinicalTrials.gov ID
- NCT00224640
- Acronym / sponsor ID
- P041201
- Conditions
- Friedreich Ataxia
- Keywords
- Iron-chelating treatment
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- Mar 2005
- Primary completion
- Mar 2008 (actual)
- Study completion
- Mar 2008 (actual)
- First posted
- 23 Sep 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: Iron chelating interventionIron chelating intervention |
Who may qualify
- Ages
- 13 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Minimum age: 13 years
- Molecular confirmation of frataxin gene mutation
- Iron overload evaluation
- Presence of lactate
- Echography response to Idebenone treatment
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Iron chelating interventionIron chelating intervention | Drug | 1 |
Primary outcome
assessment of iron overload at TO and month2 by imagery
Time frame: at months :0, 1 ,2 ,4 ,6
2 secondary outcomes
Clinical (monthly) and biological parameter follow- up ( blood count,
Time frame: weekly
plasma iron, ferritin, transferrin and liver enzymes)
Time frame: every months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2009.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Arnold MUNNICH, Pr,MD,PhD principal investigator
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Iron chelating intervention in people with Friedreich Ataxia, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Mar 2009. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
assessment of iron overload at TO and month2 by imagery (time frame: at months :0, 1 ,2 ,4 ,6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Mar 2009; AzTrials last refreshed its copy on 6 Nov 2026.