Clinical trial NCT00993967: Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension)
Official title: 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.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Freidreich's Ataxia
Keywords: FRDA
idebenone
FRDA disease
Lead sponsor: Santhera Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2009-10-14
Enrollment: 200
Sex: ALL
Minimum age (years): 9
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)
timeFrame: overall study, up to 24 months
measure: Absolute Change in The International Cooperative Ataxia Rating Scale (ICARS)
timeFrame: Baseline, Month 12 and month 24
Secondary outcomes:
Other outcomes:
measure: Measures of Safety and Tolerability: Physical Examinations and Vital Signs
timeFrame: Month 1, 3, 6, 12, 18 and 24
measure: Measures of Safety and Tolerability: Electrocardiograms (ECGs)
timeFrame: Month 1, 3, 6, 12, 18 and 24
measure: Measures of Safety and Tolerability: Haematological and Biochemical Laboratory Parameters
timeFrame: Month 1, 3, 6, 12, 18 and 24
Locations:
city: Innsbruck
country: Austria
facility: Universitätsklinik Innsbruck
city: Bruxelles
country: Belgium
facility: Hôpital Erasme - Univeristé Libre de Bruxelles
city: Paris
country: France
facility: Hôpital de la Salpétrière - INSERM U679, Neurologie et Thérapeutique expérimentale
city: Berlin
country: Germany
facility: HELIOS Klinikum Berlin
city: Bonn
country: Germany
facility: Neurologische Universitätsklinik und Ploklinik - Universitätsklimikum Bonn
city: Freiburg
country: Germany
facility: Klinik II, Neuropädiatrie und Muskelerkrankungen - Universitätsklinik Freiburg
city: Göttingen
country: Germany
facility: Zentrum für Neurologische Medizin
city: Hamburg
country: Germany
facility: UKE Hamburg Neuropädiatrie - Zentrum für Frauen, Kinder und Jugendmedizin
city: München
country: Germany
facility: Neurologische Klinik - Klinikum Grosshadern
city: Tübingen
country: Germany
facility: Neurologische Universitätsklinik und Poliklinik
city: Groningen
country: Netherlands
facility: University medical Center Groningen
Study officials:
affiliation: Dept of Molecular Neuroscience, Institute of Neurology. The National Hospital, University College London
name: Nick Wood, Professor
role: PRINCIPAL_INVESTIGATOR
References: