Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

70 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-12-31

Study Completion Date

2009-04-30

Brief Summary

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This study is meant to assess the effectiveness of idebenone on neurological outcome measures in patients with Friedreich's Ataxia over a 6 months period.

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Detailed Description

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The study involves 6 clinic visits and upon completion the possibility to join a 12 months extension study where all patients will receive high dose Idebenone.

Conditions

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Friedreich's Ataxia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Arm 1 - 900 mg/day Idebenone

mid dose Idebenone

Group Type EXPERIMENTAL

Idebenone

Intervention Type DRUG

Patients ≤ 45 kg/99 lbs: idebenone 450 mg/day; Patients \> 45 kg/99 lbs: idebenone 900 mg/day

Arm 2 - up to 2250 mg/day Idebenone

high dose Idebenone

Group Type EXPERIMENTAL

Idebenone

Intervention Type DRUG

Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients \> 45 kg/99 lbs: idebenone 2250 mg/day

Arm 3 - Placebo

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets.

Interventions

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Idebenone

Patients ≤ 45 kg/99 lbs: idebenone 450 mg/day; Patients \> 45 kg/99 lbs: idebenone 900 mg/day

Intervention Type DRUG

Idebenone

Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients \> 45 kg/99 lbs: idebenone 2250 mg/day

Intervention Type DRUG

Placebo

Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Documented diagnosis of Friedreich's Ataxia (FRDA) with confirmed FRDA mutations
* Patients 8 - 17 years of age at baseline
* Patients with a body weight greater than 25 kg/55 lbs at baseline
* Patients able to walk at least 10 meters without accompanying person (ICARS Walking Capacities score ≤6) at screening and baseline
* Patients who in the opinion of the investigator are able to comply with the requirements of the study, including swallowing the study medication
* Negative urine pregnancy test at screening and baseline (female patients of childbearing potential)

Exclusion Criteria

* Treatment with idebenone, Coenzyme Q10 or vitamin E (if taken at a dose 5 times above the daily requirement) within the past month
* Patients with International Cooperative Ataxia Rating Scale (ICARS) score of greater than 54 or less than 10 at screening
* Pregnancy and/or breast-feeding
* Clinically significant abnormalities of clinical hematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of normal of Aspartate Aminotransferase (AST; also known as GOT), Alanine Aminotransferase (ALT; also known as GPT) or creatinine
* History of abuse of drugs or alcohol
* Participation in the previous (Phase II) study of idebenone at the National Institutes of Health (NIH)
* Participation in a trial of another investigational drug within the last 3 months

Minimum Eligible Age

8 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No