Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients (IONIA-E)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00697073
First received: June 11, 2008
Last updated: May 24, 2013
Last verified: May 2013
Results First Received: June 7, 2011  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Friedreich's Ataxia
Intervention: Drug: Idebenone

  Participant Flow
  Hide 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
High Dose Idebenone

Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day (3 x 150 mg tablets, t.i.d.)

Patients > 45 kg/99 lbs: idebenone 2250 mg/day (5 x 150 mg tablets, t.i.d.)


Participant Flow:   Overall Study
    High Dose Idebenone  
STARTED     68  
COMPLETED     59  
NOT COMPLETED     9  
Withdrawal by Subject                 2  
Pregnancy                 1  
non compliance and other                 6  



  Baseline Characteristics
  Hide 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
High Dose Idebenone

Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day (3 x 150 mg tablets, t.i.d.)

Patients > 45 kg/99 lbs: idebenone 2250 mg/day (5 x 150 mg tablets, t.i.d.)


Baseline Measures
    High Dose Idebenone  
Number of Participants  
[units: participants]
  68  
Age  
[units: participants]
 
<=18 years     68  
Between 18 and 65 years     0  
>=65 years     0  
Age  
[units: years]
Mean ± Standard Deviation
  14.0  ± 2.72  
Gender  
[units: participants]
 
Female     36  
Male     32  
Region of Enrollment  
[units: participants]
 
United States     68  



  Outcome Measures

1.  Primary:   Change in ICARS   [ Time Frame: baseline and 12 months ]

2.  Secondary:   FARS (Friedreich’s Ataxia Rating Scale)   [ Time Frame: baseline and 12 Months ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

3.  Secondary:   Nature and Frequency of Adverse Events   [ Time Frame: 12 Months ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   Yes


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide 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
  Hide 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: Dr Thomas Meier
Organization: Santhera
phone: +41 61 906 8964
e-mail: thomas.meier@santhera.com


Publications of Results:

Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00697073     History of Changes
Other Study ID Numbers: SNT-III-002-E
Study First Received: June 11, 2008
Results First Received: June 7, 2011
Last Updated: May 24, 2013
Health Authority: United States: Food and Drug Administration