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

Phase 3

Completed

• Conditions: Freidreich's Ataxia
• Interventions: Drug: idebenone

• Registration Number: NCT00993967
• Lead Sponsor: Santhera Pharmaceuticals

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2007-06
• Study First Submitted: 2009-10-13
• Study First Submitted QC: 2009-10-13
• Study First Posted: 2009-10-14
• Primary Completion: 2012-06
• Study Completion: 2012-06
• Results First Submitted: 2016-10-14
• Status Verified: 2017-08
• Results First Submitted QC: 2017-08-21
• Last Update Submitted: 2017-08-21
• Results First Posted: 2018-03-05
• Last Update Posted: 2018-03-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 200

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Idebenone	idebenone	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.

Primary Outcome Measures

Name	Time	Method
Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)	overall study, up to 24 months	Global Overvbiew of accurance of AEs-Safety population. The Safety population included all subjects who received at least one dose of the study medication.
Absolute Change in The International Cooperative Ataxia Rating Scale (ICARS)	Baseline, Month 12 and month 24	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.

Trial Locations

Locations (11)

Hôpital de la Salpétrière - INSERM U679, Neurologie et Thérapeutique expérimentale; 🇫🇷 Paris, France

Zentrum für Neurologische Medizin; 🇩🇪 Göttingen, Germany

HELIOS Klinikum Berlin; 🇩🇪 Berlin, Germany

UKE Hamburg Neuropädiatrie - Zentrum für Frauen, Kinder und Jugendmedizin; 🇩🇪 Hamburg, Germany

Universitätsklinik Innsbruck; 🇦🇹 Innsbruck, Austria

Hôpital Erasme - Univeristé Libre de Bruxelles; 🇧🇪 Bruxelles, Belgium

Neurologische Universitätsklinik und Ploklinik - Universitätsklimikum Bonn; 🇩🇪 Bonn, Germany

Neurologische Universitätsklinik und Poliklinik; 🇩🇪 Tübingen, Germany

Klinik II, Neuropädiatrie und Muskelerkrankungen - Universitätsklinik Freiburg; 🇩🇪 Freiburg, Germany

Neurologische Klinik - Klinikum Grosshadern; 🇩🇪 München, Germany

University medical Center Groningen; 🇳🇱 Groningen, Netherlands