Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension)

Santhera Pharmaceuticals200 enrolled

Overview

This is an Extension study of the MICONOS main randomised placebo-controlled trial (NCT00905268), and open to those patients completing the main study. The scientific aim of this extension study is to monitor safety and tolerability of idebenone over two years in patients with Friedreich's Ataxia.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

200

Conditions

Freidreich's Ataxia

Interventions

idebenoneDRUG

Idebenone 1350 mg/d, patients \< or equal 45 kg Idebenone 2250 mg/d, patients \> 45 kg

Eligibility

Sex: ALLMin age: 9 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Completion of 52 weeks in study SNT-III-001 * Body weight ≥ 25 kg * Negative urine pregnancy test * Eligibility to participate in the present extension study as confirmed by investigator Exclusion Criteria: * Safety or tolerability issues arising during the course of SNT-III-001 which in the opinion of the investigator preclude further treatment with idebenone * Clinically significant abnormalities of haematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of SGOT, SGPT or creatinine * Parallel participation in another clinical drug trial * Pregnancy or breast-feeding * Abuse of drugs or alcohol

Locations (11)

Universitätsklinik Innsbruck

Innsbruck, Austria

Hôpital Erasme - Univeristé Libre de Bruxelles

Brussels, Belgium

Hôpital de la Salpétrière - INSERM U679, Neurologie et Thérapeutique expérimentale

Paris, France

Outcomes

Primary Outcomes

Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)

Global Overvbiew of accurance of AEs-Safety population. The Safety population included all subjects who received at least one dose of the study medication.

Time frame: overall study, up to 24 months

Absolute Change in The International Cooperative Ataxia Rating Scale (ICARS)

The International Cooperative Ataxia Rating Scale (ICARS) is a commonly used evaluation and is composed of four clinical sub-scores involving the following: posture and gait, limb coordination, speech and oculomotor function.The ICARS score is the total sum of the sub scores and ranges from 0 to 100, with 100 indicative of the most severely affected outcome.

Time frame: Baseline, Month 12 and month 24

Data from ClinicalTrials.gov

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