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

Study Overview
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Brief Summary
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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.

Official Title
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A Phase III Open-Label, Single-Group, Extension Study to Obtain Long-Term Safety and Tolerability Data of Idebenone in the Treatment of Friedreich's Ataxia Patients.

Conditions
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Freidreich's Ataxia

Intervention / Treatment
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* Drug: idebenone


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 9 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Idebenone<br>1350 mg/day or 2250 mg/day for patients weighing ≤45 kg or >45 kg, respectively.In case of poor tolerability, dose reduction to 450 mg/day or 900 mg/day, respectively, were allowed. | Drug: idebenone<br>* Idebenone 1350 mg/d, patients < or equal 45 kg Idebenone 2250 mg/d, patients > 45 kg<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 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. | Baseline, Month 12 and month 24 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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FRDA, idebenone, FRDA disease