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Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI) (PROTI)

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ClinicalTrials.gov Identifier: NCT01303406
Recruitment Status : Completed
First Posted : February 24, 2011
Results First Posted : March 9, 2016
Last Update Posted : March 9, 2016
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 29
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Placebo Idebenone
Hide Arm/Group Description

Following the body weight, patients will be allocated to one of the following regimen:

Placebo Patients < 45 kg - 3 tablets 3 times a day with meals

Placebo Patients > 45 kg - 5 tablets 3 times a day with meals

Idebenone: All PROTI patients randomised to idebenone treatment will receive high dose idebenone. This is defined according to body weight. In patients weighing 45 kg or less, it is 1350 mg/day (3 x 150 mg tablets three times per day with meals). In patients weighing more than 45 kg, it is 2250 mg/day (5 x 150 mg tablets three times per day with meals).

Following the body weight, patients will be allocated to one of the following regimen:

Idebenone Patients < 45 kg - 3 tablets 3 times a day with meals

Idebenone Patients > 45 kg - 5 tablets 3 times a day with meals

Idebenone: All PROTI patients randomised to idebenone treatment will receive high dose idebenone. This is defined according to body weight. In patients weighing 45 kg or less, it is 1350 mg/day (3 x 150 mg tablets three times per day with meals). In patients weighing more than 45 kg, it is 2250 mg/day (5 x 150 mg tablets three times per day with meals).

Period Title: Overall Study
Started 13 16
Completed 13 15
Not Completed 0 1
Arm/Group Title Placebo Idebenone Total
Hide Arm/Group Description

Following the body weight, patients will be allocated to one of the following regimen:

Placebo Patients < 45 kg - 3 tablets 3 times a day with meals

Placebo Patients > 45 kg - 5 tablets 3 times a day with meals

Idebenone: All PROTI patients randomised to idebenone treatment will receive high dose idebenone. This is defined according to body weight. In patients weighing 45 kg or less, it is 1350 mg/day (3 x 150 mg tablets three times per day with meals). In patients weighing more than 45 kg, it is 2250 mg/day (5 x 150 mg tablets three times per day with meals).

Following the body weight, patients will be allocated to one of the following regimen:

Idebenone Patients < 45 kg - 3 tablets 3 times a day with meals

Idebenone Patients > 45 kg - 5 tablets 3 times a day with meals

Idebenone: All PROTI patients randomised to idebenone treatment will receive high dose idebenone. This is defined according to body weight. In patients weighing 45 kg or less, it is 1350 mg/day (3 x 150 mg tablets three times per day with meals). In patients weighing more than 45 kg, it is 2250 mg/day (5 x 150 mg tablets three times per day with meals).

Total of all reporting groups
Overall Number of Baseline Participants 13 16 29
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 13 participants 16 participants 29 participants
38.7  (16.5) 35.8  (16.9) 37.1  (16.5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 13 participants 16 participants 29 participants
Female
4
  30.8%
7
  43.8%
11
  37.9%
Male
9
  69.2%
9
  56.3%
18
  62.1%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 13 participants 16 participants 29 participants
Germany 3 5 8
United Kingdom 7 7 14
Austria 2 2 4
Netherlands 1 2 3
1.Primary Outcome
Title Patient Assessment of Treatment Assignment: Comparison of the Proportions of Patients Randomised to Idebenone and Placebo Who Assessed That They Received Idebenone
Hide Description The primary efficacy endpoint was the comparison of the number of patients randomized to idebenone and placebo, who assessed that they received idebenone treatment.
Time Frame At 2 months after study start
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Idebenone
Hide Arm/Group Description:

Following the body weight, patients will be allocated to one of the following regimen:

Placebo Patients < 45 kg - 3 tablets 3 times a day with meals

Placebo Patients > 45 kg - 5 tablets 3 times a day with meals

Idebenone: All PROTI patients randomised to idebenone treatment will receive high dose idebenone. This is defined according to body weight. In patients weighing 45 kg or less, it is 1350 mg/day (3 x 150 mg tablets three times per day with meals). In patients weighing more than 45 kg, it is 2250 mg/day (5 x 150 mg tablets three times per day with meals).

Following the body weight, patients will be allocated to one of the following regimen:

Idebenone Patients < 45 kg - 3 tablets 3 times a day with meals

Idebenone Patients > 45 kg - 5 tablets 3 times a day with meals

Idebenone: All PROTI patients randomised to idebenone treatment will receive high dose idebenone. This is defined according to body weight. In patients weighing 45 kg or less, it is 1350 mg/day (3 x 150 mg tablets three times per day with meals). In patients weighing more than 45 kg, it is 2250 mg/day (5 x 150 mg tablets three times per day with meals).

Overall Number of Participants Analyzed 13 16
Measure Type: Number
Unit of Measure: participants
6 8
2.Secondary Outcome
Title Comparison of the Percentage of Participants Randomised to Idebenone and Placebo Who Withdrew Early Due to Recurrence or Worsening of FRDA Symptoms
Hide Description There was no Withdrawal due to recurrence or worsening of FRDA symptoms
Time Frame Within 2 months (i.e. Early withdrawal visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Idebenone
Hide Arm/Group Description:

Following the body weight, patients will be allocated to one of the following regimen:

Placebo Patients < 45 kg - 3 tablets 3 times a day with meals

Placebo Patients > 45 kg - 5 tablets 3 times a day with meals

Idebenone: All PROTI patients randomised to idebenone treatment will receive high dose idebenone. This is defined according to body weight. In patients weighing 45 kg or less, it is 1350 mg/day (3 x 150 mg tablets three times per day with meals). In patients weighing more than 45 kg, it is 2250 mg/day (5 x 150 mg tablets three times per day with meals).

Following the body weight, patients will be allocated to one of the following regimen:

Idebenone Patients < 45 kg - 3 tablets 3 times a day with meals

Idebenone Patients > 45 kg - 5 tablets 3 times a day with meals

Idebenone: All PROTI patients randomised to idebenone treatment will receive high dose idebenone. This is defined according to body weight. In patients weighing 45 kg or less, it is 1350 mg/day (3 x 150 mg tablets three times per day with meals). In patients weighing more than 45 kg, it is 2250 mg/day (5 x 150 mg tablets three times per day with meals).

Overall Number of Participants Analyzed 13 16
Measure Type: Number
Unit of Measure: percentage of patients
0 0
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo Idebenone
Hide Arm/Group Description

Following the body weight, patients will be allocated to one of the following regimen:

Placebo Patients < 45 kg - 3 tablets 3 times a day with meals

Placebo Patients > 45 kg - 5 tablets 3 times a day with meals

Idebenone: All PROTI patients randomised to idebenone treatment will receive high dose idebenone. This is defined according to body weight. In patients weighing 45 kg or less, it is 1350 mg/day (3 x 150 mg tablets three times per day with meals). In patients weighing more than 45 kg, it is 2250 mg/day (5 x 150 mg tablets three times per day with meals).

Following the body weight, patients will be allocated to one of the following regimen:

Idebenone Patients < 45 kg - 3 tablets 3 times a day with meals

Idebenone Patients > 45 kg - 5 tablets 3 times a day with meals

Idebenone: All PROTI patients randomised to idebenone treatment will receive high dose idebenone. This is defined according to body weight. In patients weighing 45 kg or less, it is 1350 mg/day (3 x 150 mg tablets three times per day with meals). In patients weighing more than 45 kg, it is 2250 mg/day (5 x 150 mg tablets three times per day with meals).

All-Cause Mortality
Placebo Idebenone
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Placebo Idebenone
Affected / at Risk (%) Affected / at Risk (%)
Total   0/13 (0.00%)   1/16 (6.25%) 
Injury, poisoning and procedural complications     
Fractured femur  0/13 (0.00%)  1/16 (6.25%) 
Musculoskeletal and connective tissue disorders     
Dislocated hip  0/13 (0.00%)  1/16 (6.25%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 2%
Placebo Idebenone
Affected / at Risk (%) Affected / at Risk (%)
Total   9/13 (69.23%)   12/16 (75.00%) 
Cardiac disorders     
Atrial fibrillation  0/13 (0.00%)  1/16 (6.25%) 
Endocrine disorders     
Parathyroid gland enlargement  0/13 (0.00%)  1/16 (6.25%) 
Gastrointestinal disorders     
Diarrhea  0/13 (0.00%)  2/16 (12.50%) 
Gastrointestinal disorder  0/13 (0.00%)  1/16 (6.25%) 
General disorders     
Fatigue  0/13 (0.00%)  3/16 (18.75%) 
Pain  1/13 (7.69%)  1/16 (6.25%) 
Injury, poisoning and procedural complications     
Fall  3/13 (23.08%)  3/16 (18.75%) 
Musculoskeletal and connective tissue disorders     
Muscle spasms  1/13 (7.69%)  1/16 (6.25%) 
Nervous system disorders     
Speech disorder  2/13 (15.38%)  2/16 (12.50%) 
Headache  1/13 (7.69%)  1/16 (6.25%) 
Tremor  1/13 (7.69%)  0/16 (0.00%) 
Psychiatric disorders     
Insomnia  1/13 (7.69%)  0/16 (0.00%) 
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
Layout table for Results Point of Contact information
Name/Title: Dr Paola Giunti
Organization: National Hospital for Neurology and Neurosurgery
Phone: 020 3448 3141
EMail: violet.le-fevre@uclh.nhs.uk
Layout table for additonal information
Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01303406     History of Changes
Other Study ID Numbers: SNT-III-004
First Submitted: February 22, 2011
First Posted: February 24, 2011
Results First Submitted: December 11, 2015
Results First Posted: March 9, 2016
Last Update Posted: March 9, 2016