Trial Outcomes & Findings for Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI) (NCT NCT01303406)
NCT ID: NCT01303406
Last Updated: 2016-03-09
Results Overview
The primary efficacy endpoint was the comparison of the number of patients randomized to idebenone and placebo, who assessed that they received idebenone treatment.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
29 participants
Primary outcome timeframe
At 2 months after study start
Results posted on
2016-03-09
Participant Flow
Participant milestones
| Measure |
Placebo
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).
|
Idebenone
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 Study
STARTED
|
13
|
16
|
|
Overall Study
COMPLETED
|
13
|
15
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI)
Baseline characteristics by cohort
| Measure |
Placebo
n=13 Participants
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).
|
Idebenone
n=16 Participants
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
n=29 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
38.7 years
STANDARD_DEVIATION 16.5 • n=5 Participants
|
35.8 years
STANDARD_DEVIATION 16.9 • n=7 Participants
|
37.1 years
STANDARD_DEVIATION 16.5 • n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
3 participants
n=5 Participants
|
5 participants
n=7 Participants
|
8 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
7 participants
n=5 Participants
|
7 participants
n=7 Participants
|
14 participants
n=5 Participants
|
|
Region of Enrollment
Austria
|
2 participants
n=5 Participants
|
2 participants
n=7 Participants
|
4 participants
n=5 Participants
|
|
Region of Enrollment
Netherlands
|
1 participants
n=5 Participants
|
2 participants
n=7 Participants
|
3 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: At 2 months after study startThe primary efficacy endpoint was the comparison of the number of patients randomized to idebenone and placebo, who assessed that they received idebenone treatment.
Outcome measures
| Measure |
Placebo
n=13 Participants
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).
|
Idebenone
n=16 Participants
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).
|
|---|---|---|
|
Patient Assessment of Treatment Assignment: Comparison of the Proportions of Patients Randomised to Idebenone and Placebo Who Assessed That They Received Idebenone
|
6 participants
|
8 participants
|
SECONDARY outcome
Timeframe: Within 2 months (i.e. Early withdrawal visit)There was no Withdrawal due to recurrence or worsening of FRDA symptoms
Outcome measures
| Measure |
Placebo
n=13 Participants
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).
|
Idebenone
n=16 Participants
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).
|
|---|---|---|
|
Comparison of the Percentage of Participants Randomised to Idebenone and Placebo Who Withdrew Early Due to Recurrence or Worsening of FRDA Symptoms
|
0 percentage of patients
|
0 percentage of patients
|
Adverse Events
Placebo
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Idebenone
Serious events: 1 serious events
Other events: 12 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=13 participants at risk
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).
|
Idebenone
n=16 participants at risk
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).
|
|---|---|---|
|
Injury, poisoning and procedural complications
Fractured femur
|
0.00%
0/13
|
6.2%
1/16
|
|
Musculoskeletal and connective tissue disorders
Dislocated hip
|
0.00%
0/13
|
6.2%
1/16
|
Other adverse events
| Measure |
Placebo
n=13 participants at risk
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).
|
Idebenone
n=16 participants at risk
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).
|
|---|---|---|
|
Injury, poisoning and procedural complications
Fall
|
23.1%
3/13
|
18.8%
3/16
|
|
General disorders
Fatigue
|
0.00%
0/13
|
18.8%
3/16
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/13
|
12.5%
2/16
|
|
Nervous system disorders
Speech disorder
|
15.4%
2/13
|
12.5%
2/16
|
|
General disorders
Pain
|
7.7%
1/13
|
6.2%
1/16
|
|
Nervous system disorders
Headache
|
7.7%
1/13
|
6.2%
1/16
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
7.7%
1/13
|
6.2%
1/16
|
|
Psychiatric disorders
Insomnia
|
7.7%
1/13
|
0.00%
0/16
|
|
Nervous system disorders
Tremor
|
7.7%
1/13
|
0.00%
0/16
|
|
Cardiac disorders
Atrial fibrillation
|
0.00%
0/13
|
6.2%
1/16
|
|
Endocrine disorders
Parathyroid gland enlargement
|
0.00%
0/13
|
6.2%
1/16
|
|
Gastrointestinal disorders
Gastrointestinal disorder
|
0.00%
0/13
|
6.2%
1/16
|
Additional Information
Dr Paola Giunti
National Hospital for Neurology and Neurosurgery
Phone: 020 3448 3141
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place