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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
Sponsor:
Information provided by (Responsible Party):
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.

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)
Primary Purpose: Treatment
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


Arm Intervention/treatment
Experimental: 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.
Drug: idebenone
Idebenone 1350 mg/d, patients < or equal 45 kg Idebenone 2250 mg/d, patients > 45 kg



Primary Outcome Measures :
  1. 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.

  2. 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.


Other Outcome Measures:
  1. 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.

  2. 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.

  3. 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.



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Ages Eligible for Study:   9 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

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


Locations
Austria
Universitätsklinik Innsbruck
Innsbruck, Austria
Belgium
Hôpital Erasme - Univeristé Libre de Bruxelles
Bruxelles, Belgium, 1070
France
Hôpital de la Salpétrière - INSERM U679, Neurologie et Thérapeutique expérimentale
Paris, France, 75651
Germany
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
Netherlands
University medical Center Groningen
Groningen, Netherlands, 9700 RB
Sponsors and Collaborators
Santhera Pharmaceuticals
Investigators
Principal Investigator: Nick Wood, Professor Dept of Molecular Neuroscience, Institute of Neurology. The National Hospital, University College London

Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00993967     History of Changes
Other Study ID Numbers: SNT-III-001-E
First Posted: October 14, 2009    Key Record Dates
Results First Posted: March 5, 2018
Last Update Posted: March 5, 2018
Last Verified: August 2017

Keywords provided by Santhera Pharmaceuticals:
FRDA
idebenone
FRDA disease

Additional relevant MeSH terms:
Ataxia
Cerebellar Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Idebenone
Ubiquinone
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Micronutrients
Growth Substances