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Effect of Pioglitazone Administered to Patients With Friedreich's Ataxia: Proof of Concept (ACTFRIE)

This study has been completed.
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris Identifier:
First received: December 18, 2008
Last updated: September 2, 2013
Last verified: August 2013

Friedreich's ataxia (FA) is a rare progressive neurological disorder affecting approximately 1/30, 000 individuals. No treatment is presently available to counteract the neurodegeneration of this extremely severe disease.

Pioglitazone, a well known PPAR gamma (peroxysome proliferators-activated receptor gamma) ligand induces the expression of many enzymes involved in the mitochondrial metabolism, including the superoxide dismutases. This agent may be therapeutic by counteracting the disabled recruitment of antioxidant enzymes in FA patients. This potential neuroprotective agent crosses the brain blood barrier in human.

Primary objective: To explore the effects of Pioglitazone on neurological function in FA patients. We expect neurological benefits taking into account the natural course of the disease.

Population: Subjects for this study will be limited to patients not older than 25 years Methodology: Prospective, randomized double-blind trial of Pioglitazone versus placebo in FA patients. Patients will be treated two years and will undergo clinical exams and testing during three days each six months at the clinical investigation centre.

Condition Intervention Phase
Friedreich's Ataxia Drug: pioglitazone Drug: Placebo Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: Effect of Pioglitazone Administered to Patients With Friedreich's ATAXIA:Proof of Concept

Resource links provided by NLM:

Further study details as provided by Assistance Publique - Hôpitaux de Paris:

Primary Outcome Measures:
  • evaluate the efficacy of Pioglitazone on the neurological function of FA patients. Success will be defined as a stabilisation or improvement on ICARS designed as no more than 2 points maximum increment on this scale in two year. [ Time Frame: 2 years ]

Secondary Outcome Measures:
  • tolerance of Pioglitazone [ Time Frame: 2 years ]
  • efficacy of Pioglitazone on neurological function [ Time Frame: 2 years ]
  • efficacy of Pioglitazone on functional handicap and quality of life [ Time Frame: 2 years ]
  • effect of Pioglitazone on cardiac parameters [ Time Frame: 2 years ]

Enrollment: 40
Study Start Date: December 2008
Study Completion Date: March 2013
Primary Completion Date: March 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Pioglitazone
Drug: pioglitazone
administered orally once a day after the first visit and for a total of 2 years. Initially, Pioglitazone will be started at 15mg /day. Dosage will then increase by 15mg /d/ week up to the maximal dose of 45mg /day
Placebo Comparator: Control
Drug: Placebo
a placebo administered orally once a day after the first visit and for a total of 2 years.
Other Name: Control

  Show Detailed Description


Ages Eligible for Study:   7 Years to 24 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of FA with confirmed FRDA mutations
  • GAA repeat length of the shorter allele of frataxin gene > 300
  • Age ≤ 24 years
  • Ambulatory (assistance devices permitted) or able to stand up without support
  • Neurologically symptomatic
  • All subjects agree and commit to the use of 2 reliable methods of birth control for the duration of the study if sexually active
  • Willing (and parents if minor) to participate in all aspects of trial design and follow-up
  • No modification of the usual treatment 6 months before inclusion and agree to stay with the same treatment during the trial (idebenone with a stable dosage, cardiologic therapeutic)

Exclusion Criteria:

  • Composite heterozygote
  • Patients unable to stand up even with support
  • Pregnant women
  • Cardiac insufficiency NYHA III to IV and heart ejection fraction> 50%
  • Alkaline phosphatase, SGOT or SGPT greater than 1.5 X the upper limit of normal
  • Patients with diabetes
  • Modification of the concomitant medications taken by the patient within the 6 months before inclusion or during the trial
  • Clinically significant medical disease that, in the judgment of the investigators, would expose the patient to undue risk of harm or prevent the patient from completing the study.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00811681

Hôpital Robert Debré
Paris, France, 75019
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Principal Investigator: Isabelle Husson Assistance Publique - Hôpitaux de Paris
  More Information

Responsible Party: Assistance Publique - Hôpitaux de Paris Identifier: NCT00811681     History of Changes
Other Study ID Numbers: P070123
Study First Received: December 18, 2008
Last Updated: September 2, 2013

Keywords provided by Assistance Publique - Hôpitaux de Paris:
Friedreich's ataxia

Additional relevant MeSH terms:
Friedreich Ataxia
Cerebellar Ataxia
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Hypoglycemic Agents
Physiological Effects of Drugs processed this record on September 21, 2017