The study to test whether patients can tell if they feel better when they are treated with Idebenone

Phase 1

Active, not recruiting

Conditions: Friedreich's Ataxia
MedDRA version: 14.1Level: PTClassification code 10017374Term: Friedreich's ataxiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Registration Number: EUCTR2007-001646-40-GB

Lead Sponsor: Santhera Pharmaceuticals (Switzerland) Ltd

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Not Recruiting

Sex: All

Target Recruitment: 232

Inclusion Criteria

• Completion of 52 weeks in study SNT-III-001
• Body weight = 25kg
• Negative urine pregnancy test
• Eligibility to participate in the present extension study as confirmed by the investigator
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Primary end point(s): • Measures of safety and tolerability<br>- nature and frequency of AEs<br>- haematological and biochemical parameters<br>- physical examinations and vital signs<br>- ECGs<br>;Timepoint(s) of evaluation of this end point: From Baseline, Week 4, 12, Month 6, 12 and 24;Main Objective: • To gather long-term data on the safety and tolerability of idebenone in Friedreich’s Ataxia patients<br>;Secondary Objective: • To explore the effect of idebenone after longer term administration on neurological symptoms and signs as assessed by the International Cooperative Ataxia Rating Scale (ICARS) and the Friedreich’s Ataxia Rating Scale (FARS)

Secondary Outcome Measures