Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (IONIA)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00537680
First received: September 7, 2007
Last updated: February 2, 2012
Last verified: February 2012
Results First Received: June 7, 2011  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, 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
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Reporting Groups
  Description
Mid Dose Idebenone 25 kg/55 lbs to ≤45 kg/99 lbs: idebenone 450 mg/day (1 x 150 mg tablet, t.i.d.) >45 kg/99 lbs: idebenone 900 mg/day (2 x 150 mg tablet, t.i.d.)
High Dose Idebenone 25 kg/55 lbs to ≤45 kg/99 lbs: idebenone 1350 mg/day (2 x 150 mg tablet, t.i.d.) >45 kg/99 lbs: idebenone 2250 mg/day (2 x 150 mg tablet, t.i.d.)
Placebo Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets.

Participant Flow:   Overall Study
    Mid Dose Idebenone     High Dose Idebenone     Placebo  
STARTED     22     24     24  
COMPLETED     22     22     24  
NOT COMPLETED     0     2     0  



  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
Mid Dose Idebenone 25 kg/55 lbs to ≤45 kg/99 lbs: idebenone 450 mg/day (1 x 150 mg tablet, t.i.d.) >45 kg/99 lbs: idebenone 900 mg/day (2 x 150 mg tablet, t.i.d.)
High Dose Idebenone 25 kg/55 lbs to ≤45 kg/99 lbs: idebenone 1350 mg/day (2 x 150 mg tablet, t.i.d.) >45 kg/99 lbs: idebenone 2250 mg/day (2 x 150 mg tablet, t.i.d.)
Placebo Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets.
Total Total of all reporting groups

Baseline Measures
    Mid Dose Idebenone     High Dose Idebenone     Placebo     Total  
Number of Participants  
[units: participants]
  22     24     24     70  
Age  
[units: participants]
       
<=18 years     22     24     24     70  
Between 18 and 65 years     0     0     0     0  
>=65 years     0     0     0     0  
Age  
[units: years]
Mean ± Standard Deviation
  13.93  ± 2.54     13.44  ± 3.03     13.69  ± 2.77     13.68  ± 2.76  
Gender  
[units: participants]
       
Female     7     14     16     37  
Male     15     10     8     33  
Region of Enrollment  
[units: participants]
       
United States     22     24     24     70  



  Outcome Measures

1.  Primary:   ICARS   [ Time Frame: baseline and 6 months ]

2.  Secondary:   FARS   [ Time Frame: 6 Months ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

3.  Secondary:   ADL of FARS   [ Time Frame: 6 Months ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

4.  Secondary:   FACT   [ Time Frame: 6 Months ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events


  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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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: Dr Thomas Meier
Organization: Santhera Pharmaceuticals
phone: +41619068964
e-mail: thomas.meier@santhera.com


Publications of Results:
Publications automatically indexed to this study:

Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00537680     History of Changes
Other Study ID Numbers: SNT-III-002
Study First Received: September 7, 2007
Results First Received: June 7, 2011
Last Updated: February 2, 2012
Health Authority: United States: Food and Drug Administration