Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension)
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|ClinicalTrials.gov Identifier: NCT00993967|
Recruitment Status : Completed
First Posted : October 14, 2009
Results First Posted : March 5, 2018
Last Update Posted : March 5, 2018
|Condition or disease||Intervention/treatment||Phase|
|Freidreich's Ataxia||Drug: idebenone||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||200 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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 Start Date :||June 2007|
|Actual Primary Completion Date :||June 2012|
|Actual Study Completion Date :||June 2012|
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.
Idebenone 1350 mg/d, patients < or equal 45 kg Idebenone 2250 mg/d, patients > 45 kg
- Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs) [ Time Frame: 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) [ Time Frame: 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.
- Measures of Safety and Tolerability: Physical Examinations and Vital Signs [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ]Assessment of the head, eyes, ears, nose, throat, heart, chest, lungs, abdomen, extremities, peripheral pulses, skin and any other physical conditions of note.
- Measures of Safety and Tolerability: Electrocardiograms (ECGs) [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ]12-lead ECG recordings were performed at every visit. Each ECG was measured using 3 complexes: PR interval in lead II or V2, QRS and QT intervals and heart rate in lead II, corrected QT intervals QTcB and QTcF.
- Measures of Safety and Tolerability: Haematological and Biochemical Laboratory Parameters [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ]Safety haematological analysis were done at every visit. Analyses included red blood cell count, haemoglobin, haematocrit, red cell indices, white blood cell count including differential, platelet count Safety biochemistry were done at every visit. Analyses included sodium, potassium, chloride, bicarbonate, urea, creatinine, calcium, inorganic phosphate, glucose, total bilirubin, total protein, albumin, aspartate amiotransferase (AST), alanine aminotransferase (ALT), alkaline phosphotase, Gamma GT, creatine kinase (CK)^, cholesterol, triglycerides, uric acid.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00993967
|Hôpital Erasme - Univeristé Libre de Bruxelles|
|Bruxelles, Belgium, 1070|
|Hôpital de la Salpétrière - INSERM U679, Neurologie et Thérapeutique expérimentale|
|Paris, France, 75651|
|HELIOS Klinikum Berlin|
|Berlin, Germany, 13125|
|Neurologische Universitätsklinik und Ploklinik - Universitätsklimikum Bonn|
|Bonn, Germany, 53105|
|Klinik II, Neuropädiatrie und Muskelerkrankungen - Universitätsklinik Freiburg|
|Freiburg, Germany, 79106|
|Zentrum für Neurologische Medizin|
|Göttingen, Germany, 37073|
|UKE Hamburg Neuropädiatrie - Zentrum für Frauen, Kinder und Jugendmedizin|
|Hamburg, Germany, 20246|
|Neurologische Klinik - Klinikum Grosshadern|
|München, Germany, 81377|
|Neurologische Universitätsklinik und Poliklinik|
|Tübingen, Germany, 72076|
|University medical Center Groningen|
|Groningen, Netherlands, 9700 RB|
|Principal Investigator:||Nick Wood, Professor||Dept of Molecular Neuroscience, Institute of Neurology. The National Hospital, University College London|