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A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients (MICONOS)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00905268
First received: May 19, 2009
Last updated: May 19, 2016
Last verified: May 2016
History of Changes
Results First Received: October 20, 2015  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Friedreich's Ataxia
Interventions: Drug: idebenone
Drug: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Group A: Idebenone

Patients under/equal 45 kg: idebenone 180 mg/day Patients over 45 kg: idebenone 360 mg/day

idebenone: 12 months of 1 of 3 treatments arms of oral idebenone or placebo.Treatment taken 3 times daily with meals.
Group B: Idebenone

Patients under/equal 45 kg: idebenone 450 mg/day Patients over 45 kg: idebenone 900 mg/day

idebenone: 12 months of 1 of 3 treatments arms of oral idebenone or placebo.Treatment taken 3 times daily with meals.
C: Idebenone

Patients under/equal 45 kg: idebenone 1350 mg/day Patients over 45 kg: idebenone 2250 mg/day

idebenone: 12 months of 1 of 3 treatments arms of oral idebenone or placebo.Treatment taken 3 times daily with meals.
D: Placebo

placebo

Placebo: 12 months of 1 of 3 treatments arms of oral idebenone or placebo.Treatment taken 3 times daily with meals.

Participant Flow:   Overall Study
    Group A: Idebenone   Group B: Idebenone   C: Idebenone   D: Placebo
STARTED   57   57   59   59 
COMPLETED   53   56   59   57 
NOT COMPLETED   4   1   0   2 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Group A: Idebenone

Patients under/equal 45 kg: idebenone 180 mg/day Patients over 45 kg: idebenone 360 mg/day

idebenone: 12 months of 1 of 3 treatments arms of oral idebenone or placebo.Treatment taken 3 times daily with meals.
Group B: Idebenone

Patients under/equal 45 kg: idebenone 450 mg/day Patients over 45 kg: idebenone 900 mg/day

idebenone: 12 months of 1 of 3 treatments arms of oral idebenone or placebo.Treatment taken 3 times daily with meals.
C: Idebenone

Patients under/equal 45 kg: idebenone 1350 mg/day Patients over 45 kg: idebenone 2250 mg/day

idebenone: 12 months of 1 of 3 treatments arms of oral idebenone or placebo.Treatment taken 3 times daily with meals.
D: Placebo

placebo

Placebo: 12 months of 1 of 3 treatments arms of oral idebenone or placebo.Treatment taken 3 times daily with meals.
Total Total of all reporting groups

Baseline Measures
   Group A: Idebenone   Group B: Idebenone   C: Idebenone   D: Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 		 57   57   59   59   232 
Age 
[Units: Years]
Mean (Standard Deviation) 		 30.9  (13.7)   31.6  (13.1)   30.9  (13.6)   30.4  (13.3)   30.9  (13.3) 
Gender 
[Units: Participants]
 		         
Female   23   25   27   32   107 
Male   34   32   32   27   125 
Region of Enrollment [1] 
[Units: Participants]
 		         
Germany   40   41   39   36   156 
United Kingdom   4   6   6   5   21 
Austria   2   4   2   5   13 
Belgium   3   1   5   4   13 
France   5   2   4   6   17 
Netherlands   3   3   3   3   12 
[1] The Intent-To-Treat (ITT) population included all randomized subjects who received at least one dose of the study medication and had a confirmed diagnosis of FRDA, did not take idebenone within one month pre-Screening or between Screening and Visit 1, and were not under idebenone treatment at Baseline according to available PK results at Visit 1. The subjects were analyzed as randomized regardless of protocol deviations


  Outcome Measures
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1.  Primary:   Absolute Change in International Cooperative Ataxia Rating Scale (ICARS) Scores From Baseline Assessment to Week 52   [ Time Frame: Baseline and week 52 ]
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2.  Secondary:   Absolute Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline Assessment to Week 52   [ Time Frame: Baseline and week 52 ]
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3.  Secondary:   Proportion of Patients Improving (Responding) on ICARS by a Clinically Relevant Margin   [ Time Frame: week 52 ]
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4.  Secondary:   Proportion of Patients Improving on Left Ventricular Peak Systolic Strain Rate or Showing a Reduction in Left Ventricular Mass Index (LVMI) With no Worsening in Strain Rate   [ Time Frame: 1 year ]
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5.  Secondary:   Change in Peak Systolic Strain Rate From Baseline to Week 52   [ Time Frame: 1 year ]
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6.  Secondary:   Change in Peak Workload From Baseline to Week 52   [ Time Frame: 1 year ]
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  Serious Adverse Events
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  Other Adverse Events
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  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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