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Trial record 44 of 219 for:    Ataxia

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

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ClinicalTrials.gov Identifier: NCT00697073
Recruitment Status : Completed
First Posted : June 13, 2008
Results First Posted : August 4, 2011
Last Update Posted : June 4, 2013
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Friedreich's Ataxia
Intervention Drug: Idebenone
Enrollment 68
Recruitment Details  
Pre-assignment Details  
Arm/Group Title High Dose Idebenone
Hide Arm/Group Description

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.)

Period Title: Overall Study
Started 68
Completed 59
Not Completed 9
Reason Not Completed
Withdrawal by Subject             2
Pregnancy             1
non compliance and other             6
Arm/Group Title High Dose Idebenone
Hide Arm/Group Description

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.)

Overall Number of Baseline Participants 68
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 68 participants
<=18 years
68
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 68 participants
14.0  (2.72)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 68 participants
Female
36
  52.9%
Male
32
  47.1%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 68 participants
68
1.Primary Outcome
Title Change in 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 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title High Dose Idebenone
Hide Arm/Group Description:

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.)

Overall Number of Participants Analyzed 68
Mean (Standard Deviation)
Unit of Measure: ICARS points
1.1  (5.64)
2.Secondary Outcome
Title FARS (Friedreich’s Ataxia Rating Scale)
Hide Description [Not Specified]
Time Frame baseline and 12 Months
Outcome Measure Data Not Reported
3.Secondary Outcome
Title Nature and Frequency of Adverse Events
Hide Description [Not Specified]
Time Frame 12 Months
Outcome Measure Data Not Reported
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title High Dose Idebenone
Hide Arm/Group Description

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.)

All-Cause Mortality
High Dose Idebenone
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
High Dose Idebenone
Affected / at Risk (%) # Events
Total   4/68 (5.88%)    
Cardiac disorders   
cardiac failure  1/68 (1.47%)  1
tachycardia  1/68 (1.47%)  1
Endocrine disorders   
Hyperthyroidism  1/68 (1.47%)  1
General disorders   
Hypotension  1/68 (1.47%)  1
Deep vein thrombosis  1/68 (1.47%)  1
Dehydratation  1/68 (1.47%)  1
Infections and infestations   
Pharyngitis streptococcal  1/68 (1.47%)  1
Pneumonia  1/68 (1.47%)  1
Pregnancy, puerperium and perinatal conditions   
Pregnancy  1/68 (1.47%)  1
intra uterine death  1/68 (1.47%)  1
abortion spontaneous  1/68 (1.47%)  1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 1%
High Dose Idebenone
Affected / at Risk (%) # Events
Total   66/68 (97.06%)    
Gastrointestinal disorders   
nausea  15/68 (22.06%) 
General disorders   
headache  21/68 (30.88%) 
pyrexia  14/68 (20.59%) 
Infections and infestations   
upper respiratory tract infection  18/68 (26.47%) 
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
Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00697073     History of Changes
Other Study ID Numbers: SNT-III-002-E
First Submitted: June 11, 2008
First Posted: June 13, 2008
Results First Submitted: June 7, 2011
Results First Posted: August 4, 2011
Last Update Posted: June 4, 2013