Friedreich's Ataxia
Conditions
Keywords
randomized withdrawal, idebenone, friedreich's ataxia, Miconos, Patient reported outcome, ICARS
Brief summary
This is a Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study. The aim is to further investigate the effects of idebenone in patients with Friedreich's ataxia. The objective of the PROTI study is to establish whether patients can correctly determine which treatment assignment (placebo or idebenone) they received during the randomised phase of the trial, and identify any potential changes on symptoms or activities.
Interventions
DRUGIdebenone
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).
DRUGPlacebo
Sponsors
Santhera Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
10 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Completion of V5 (Month 12), V6 (Month 18), or V7 (Month 24) in the MICONOS extension study * Patients who in the opinion of the investigator are able to comply with the requirements of the study * Body weight ≥ 25kg * Negative urine pregnancy test
Exclusion criteria
* AE during the course of the MICONOS extension study which in the opinion of the investigator is attributable to idebenone and precludes further treatment with idebenone * Clinically significant abnormalities of clinical haematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of normal SGOT, SGPT or creatinine * Parallel participation in another clinical drug trial * Pregnancy or breast-feeding * Abuse of drugs or alcohol * Any change of concomitant medication within the last 30 days that in the opinion of the investigator the intake could negatively impact the study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Patient Assessment of Treatment Assignment: Comparison of the Proportions of Patients Randomised to Idebenone and Placebo Who Assessed That They Received Idebenone | At 2 months after study start | The primary efficacy endpoint was the comparison of the number of patients randomized to idebenone and placebo, who assessed that they received idebenone treatment. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Comparison of the Percentage of Participants Randomised to Idebenone and Placebo Who Withdrew Early Due to Recurrence or Worsening of FRDA Symptoms | Within 2 months (i.e. Early withdrawal visit) | There was no Withdrawal due to recurrence or worsening of FRDA symptoms |
Countries
Austria, Germany, Netherlands, United Kingdom
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 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). | 13 |
| 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). | 16 |
| Total | 29 |
Baseline characteristics
| Characteristic | Placebo | Idebenone | Total |
|---|---|---|---|
| Age, Continuous | 38.7 years STANDARD_DEVIATION 16.5 | 35.8 years STANDARD_DEVIATION 16.9 | 37.1 years STANDARD_DEVIATION 16.5 |
| Region of Enrollment Austria | 2 participants | 2 participants | 4 participants |
| Region of Enrollment Germany | 3 participants | 5 participants | 8 participants |
| Region of Enrollment Netherlands | 1 participants | 2 participants | 3 participants |
| Region of Enrollment United Kingdom | 7 participants | 7 participants | 14 participants |
| Sex: Female, Male Female | 4 Participants | 7 Participants | 11 Participants |
| Sex: Female, Male Male | 9 Participants | 9 Participants | 18 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 9 / 13 | 12 / 16 |
| serious Total, serious adverse events | 0 / 13 | 1 / 16 |
Outcome results
Primary
Patient Assessment of Treatment Assignment: Comparison of the Proportions of Patients Randomised to Idebenone and Placebo Who Assessed That They Received Idebenone
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
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Placebo | Patient Assessment of Treatment Assignment: Comparison of the Proportions of Patients Randomised to Idebenone and Placebo Who Assessed That They Received Idebenone | 6 participants |
| Idebenone | Patient Assessment of Treatment Assignment: Comparison of the Proportions of Patients Randomised to Idebenone and Placebo Who Assessed That They Received Idebenone | 8 participants |
Secondary
Comparison of the Percentage of Participants Randomised to Idebenone and Placebo Who Withdrew Early Due to Recurrence or Worsening of FRDA Symptoms
There was no Withdrawal due to recurrence or worsening of FRDA symptoms
Time frame: Within 2 months (i.e. Early withdrawal visit)
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Placebo | 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 |
| Idebenone | 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 |