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

Santhera Pharmaceuticals70 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Friedreich's Ataxia

Interventions

IdebenoneDRUG

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

IdebenoneDRUG

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

PlaceboDRUG

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

Eligibility

Sex: ALLMin age: 8 YearsMax age: 17 Years

Medical Language ↔ Plain English

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

Locations (2)

David Geffen School of Medicine, UCLA

Los Angeles, California, United States

The Children's Hopsital of Philadelphia

Philadelphia, Pennsylvania, United States

Outcomes

Primary Outcomes

Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24

International Cooperative Ataxia Rating Scale (ICARS): ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability. Minimum score: 0, maximum score 100, higher score indicates greater impairment Calculation details: ICARS score at week 24 minus ICARS score baseline

Time frame: baseline and 6 months

Secondary Outcomes

Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24

To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on neurological function as assessed by the Friedreich's Ataxia Rating Scale (FARS) FARS consists of a 25 manoeuvre exam along with 4 quantitative performance measures. The neurological exam covers bulbar function, upper limb coordination, lower limb coordination, peripheral nervous system function, deep tendon reflexes, stability and gait. The use of FARS has been recently validated as a sensitive scale for this population. The FARS exam scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability. Calculation details FARS score at week 24 minus FARS score baseline

Time frame: baseline and 6 months

Activities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24

To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on Activities of Daily Living (ADL) assessed by the ADL scale of the Friedreich's Ataxia Rating Scale (FARS) score 0 to 36, higher score indicates greater impairment

Time frame: baseline and 6 months

Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12

Data from ClinicalTrials.gov

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