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

NCT00811681 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 40

Last updated 2013-09-04

No results posted yet for this study

Summary

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.

Conditions

Interventions

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

DRUG

Placebo

a placebo administered orally once a day after the first visit and for a total of 2 years.

Sponsors & Collaborators

  • Assistance Publique - Hôpitaux de Paris

    lead OTHER

Principal Investigators

  • Isabelle Husson · Assistance Publique - Hôpitaux de Paris

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
7 Years
Max Age
24 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-12-31
Primary Completion
2013-03-31
Completion
2013-03-31

Countries

  • France

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00811681 on ClinicalTrials.gov