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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

October 13, 2009
October 14, 2009
October 14, 2016
March 5, 2018
March 5, 2018
June 2007
June 2012   (Final data collection date for primary outcome measure)
  • 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: Nature and Frequency of Adverse Events (AEs) [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ]
  • Measures of safety and tolerability: haematological and biochemical laboratory parameters [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ]
  • Measures of safety and tolerability: physical examinations and vital signs [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ]
  • Measures of safety and tolerability: electrocardiograms (ECGs) [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ]
Complete list of historical versions of study NCT00993967 on ClinicalTrials.gov Archive Site
Not Provided
Absolute change in The International Cooperative Ataxia Rating Scale (ICARS) and Friedreich Ataxia Rating Scale (FARS) scores [ Time Frame: Baseline, Month 12 and month 24 ]
  • 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.
Not Provided
 
Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension)
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.
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.
Not Provided
Interventional
Phase 3
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Freidreich's Ataxia
Drug: idebenone
Idebenone 1350 mg/d, patients < or equal 45 kg Idebenone 2250 mg/d, patients > 45 kg
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.
Intervention: Drug: idebenone
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
200
204
June 2012
June 2012   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
9 Years and older   (Child, Adult, Older Adult)
No
Contact information is only displayed when the study is recruiting subjects
Austria,   Belgium,   France,   Germany,   Netherlands
 
 
NCT00993967
SNT-III-001-E
Yes
Not Provided
Not Provided
Santhera Pharmaceuticals
Santhera Pharmaceuticals
Not Provided
Principal Investigator: Nick Wood, Professor Dept of Molecular Neuroscience, Institute of Neurology. The National Hospital, University College London
Santhera Pharmaceuticals
August 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP