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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

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Freidreich's Ataxia
Intervention: Drug: idebenone

  Participant Flow

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
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.

idebenone: Idebenone 1350 mg/d, patients < or equal 45 kg Idebenone 2250 mg/d, patients > 45 kg


Participant Flow:   Overall Study
    Idebenone
STARTED   200 
COMPLETED   139 
NOT COMPLETED   61 
Adverse Event                8 
Death                1 
Lost to Follow-up                14 
Withdrawal by Subject                36 
Protocol Violation                2 



  Baseline Characteristics

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
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.

idebenone: Idebenone 1350 mg/d, patients < or equal 45 kg Idebenone 2250 mg/d, patients > 45 kg


Baseline Measures
   Idebenone 
Overall Participants Analyzed 
[Units: Participants]
 200 
Age 
[Units: Years]
Mean (Standard Deviation)
 32  (13.68) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      93  46.5% 
Male      107  53.5% 
Region of Enrollment 
[Units: Participants]
 
Netherlands   8 
Austria   6 
Belgium   10 
France   13 
Germany   143 
United Kingdom   20 


  Outcome Measures

1.  Primary:   Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)   [ Time Frame: overall study, up to 24 months ]

2.  Primary:   Absolute Change in The International Cooperative Ataxia Rating Scale (ICARS)   [ Time Frame: Baseline, Month 12 and month 24 ]

3.  Other Pre-specified:   Measures of Safety and Tolerability: Physical Examinations and Vital Signs   [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

4.  Other Pre-specified:   Measures of Safety and Tolerability: Electrocardiograms (ECGs)   [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

5.  Other Pre-specified:   Measures of Safety and Tolerability: Haematological and Biochemical Laboratory Parameters   [ Time Frame: Month 1, 3, 6, 12, 18 and 24 ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

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
No text entered.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Thomas Meier, PhD
Organization: Santhera Pharmaceuticals
phone: +41 61 906 89 50
e-mail: thomas.meier@santhera.com <Thomas.Meier@santhera.com>



Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00993967     History of Changes
Other Study ID Numbers: SNT-III-001-E
First Submitted: October 13, 2009
First Posted: October 14, 2009
Results First Submitted: October 14, 2016
Results First Posted: March 5, 2018
Last Update Posted: March 5, 2018