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

Study Overview
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Brief Summary
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This study is meant to assess the safety and tolerability of idebenone in patients with Friedreich's Ataxia over a 12 months period.

Detailed Description
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The study involves 6 clinic visits.

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

Conditions
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Friedreich's Ataxia

Intervention / Treatment
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* Drug: Idebenone


Participation Criteria
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Eligibility Criteria
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Inclusion criteria: Friedreich's ataxia patients completing core study SNT-III-002 (NCT00537680) and presenting at Week 24 (Visit 5) of that study Body weight ≥ 25kg/55 lbs Negative urine pregnancy test Patients who in the opinion of the investigator are able to comply with the requirements of this study Exclusion criteria: Adverse events during the course of SNT-III-002(NCT00537680)which in the opinion of the investigator are attributable to idebenone and preclude further treatment with idebenone Clinically significant abnormalities of clinical hematology or biochemistry including, but not limited to, elevations greater than 2 times the upper limit of normal of AST, ALT or creatinine Treatment with coenzyme Q10, vitamin E (if taken at a dose 5 times above the daily requirement) or other sources of idebenone within the past month Parallel participation in another clinical drug trial Past or present history of abuse of drugs or alcohol Pregnancy or breast-feeding

Ages Eligible for Study
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Minimum Age: 8 Years
Maximum Age: 18 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: 1<br>high dose Idebenone | Drug: Idebenone<br>* Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients > 45 kg/99 lbs: idebenone 2250 mg/day<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in ICARS | 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. A total score of 0 points represents the best possible score, and a value of 100 points is the worst possible score. Therefore, a negative change in ICARS score represents an improvement. A higher score also indicates more disability. | baseline and 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| FARS (Friedreich's Ataxia Rating Scale) | 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 consists of three subscales, comprising a general score for ataxia, a score for activities of daily living (ADL) and a neurological examination. The scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability. | baseline and 12 Months | 
| Nature of Adverse Events |  | 12 Months | 
| Frequency/Number of Mild, Moderate, and Severe Adverse Events |  | 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Friedreich's Ataxia, Idebenone, ICARS