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

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ClinicalTrials.gov Identifier: NCT00537680
Recruitment Status : Completed
First Posted : October 1, 2007
Results First Posted : December 29, 2011
Last Update Posted : February 3, 2012
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Friedreich's Ataxia
Interventions Drug: Idebenone
Drug: Placebo
Enrollment 70
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Mid Dose Idebenone High Dose Idebenone Placebo
Hide Arm/Group Description 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.) 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 was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets.
Period Title: Overall Study
Started 22 24 24
Completed 22 22 24
Not Completed 0 2 0
Arm/Group Title Mid Dose Idebenone High Dose Idebenone Placebo Total
Hide Arm/Group Description 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.) 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 was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets. Total of all reporting groups
Overall Number of Baseline Participants 22 24 24 70
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants 24 participants 24 participants 70 participants
<=18 years
22
 100.0%
24
 100.0%
24
 100.0%
70
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 22 participants 24 participants 24 participants 70 participants
13.93  (2.54) 13.44  (3.03) 13.69  (2.77) 13.68  (2.76)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants 24 participants 24 participants 70 participants
Female
7
  31.8%
14
  58.3%
16
  66.7%
37
  52.9%
Male
15
  68.2%
10
  41.7%
8
  33.3%
33
  47.1%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 22 participants 24 participants 24 participants 70 participants
22 24 24 70
1.Primary Outcome
Title ICARS
Hide Description

International Cooperative Ataxia Rating Scale (ICARS):

ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability.

Scores for each subscale quantify the extent of ataxia in each clinically important area. Subscale scores are summed to give a total score ranging from 0 (best) to 100 (worst).

Time Frame baseline and 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Mid Dose Idebenone High Dose Idebenone Placebo
Hide Arm/Group Description:
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.)
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 was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets.
Overall Number of Participants Analyzed 22 24 24
Mean (Standard Deviation)
Unit of Measure: ICARS points
-2.5  (6.3) -2.4  (4.8) -1.3  (4.4)
2.Secondary Outcome
Title FARS
Hide Description Friedreich’s Ataxia Rating Scale
Time Frame 6 Months
Outcome Measure Data Not Reported
3.Secondary Outcome
Title ADL of FARS
Hide Description ADL=Activities of Daily Living
Time Frame 6 Months
Outcome Measure Data Not Reported
4.Secondary Outcome
Title FACT
Hide Description Friedreich’s Ataxia Composite Test
Time Frame 6 Months
Outcome Measure Data Not Reported
Time Frame [Not Specified]
Adverse Event Reporting Description "0" means none.
 
Arm/Group Title Mid Dose Idebenone High Dose Idebenone Placebo
Hide Arm/Group Description 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.) 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 was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets.
All-Cause Mortality
Mid Dose Idebenone High Dose Idebenone Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Mid Dose Idebenone High Dose Idebenone Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/22 (0.00%)      2/24 (8.33%)      0/24 (0.00%)    
Blood and lymphatic system disorders       
idiopathic thrombocytopenic purpura *  0/22 (0.00%)  0 1/24 (4.17%)  1 0/24 (0.00%)  0
General disorders       
chest pain  0/22 (0.00%)  0 1/24 (4.17%)  2 0/24 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 1%
Mid Dose Idebenone High Dose Idebenone Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   18/22 (81.82%)      23/24 (95.83%)      21/24 (87.50%)    
Gastrointestinal disorders       
Gastrointestinal disorders  8/22 (36.36%)  15/24 (62.50%)  12/24 (50.00%) 
General disorders       
General disorders  10/22 (45.45%)  9/24 (37.50%)  9/24 (37.50%) 
Infections and infestations       
Infections *  11/22 (50.00%)  15/24 (62.50%)  16/24 (66.67%) 
Musculoskeletal and connective tissue disorders       
Musculoskeletal & connective tissue disorders  7/22 (31.82%)  10/24 (41.67%)  7/24 (29.17%) 
Nervous system disorders       
Nervous system disorders  10/22 (45.45%)  14/24 (58.33%)  12/24 (50.00%) 
*
Indicates events were collected by non-systematic assessment
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
Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00537680     History of Changes
Other Study ID Numbers: SNT-III-002
First Submitted: September 7, 2007
First Posted: October 1, 2007
Results First Submitted: June 7, 2011
Results First Posted: December 29, 2011
Last Update Posted: February 3, 2012