The primary objective of the PROTI study is to establish whether patients can correctly determine which treatment assignment they received during the randomised phase of the trial.The key secondary objective is to compare the rate of withdrawal from…
ID
Source
Brief title
Condition
- Other condition
Synonym
Health condition
neuromuscular disorder, genetic, degenerative diseases affecting nerve and muscle tissue.
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Patient assessment of treatment assignment:
Comparison of the proportions of patients randomised to idebenone and placebo
who assessed that they received idebenone
Secondary outcome
The key secondary objective is to compare the rate of withdrawal from the PROTI
study of patients who have been withdrawn from treatment with high-dose
idebenone with the rate of withdrawal of patients continuing to receive high
dose idebenone.
Other secondary endpoints:
• Change in fatigue level as assessed by the MFIS
• Change in 9-HPT time
• Change in speech capability
• CGI-C
• Investigator assessment of treatment assignment
• Change in ICARS score
• PROs collected via Daily Patient Diary and Questionnaires
• Safety
Background summary
A Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study of Patient
Reported Outcomes in Friedreich*s Ataxia Patients after withdrawal from
Treatment with Idebenone will be achieved by randomising patients receiving
high dose idebenone in MICONOS-Extension study.
Therefore, patients participating in the MICONOS-Extension study will be
invited to suspend their participation in MES to participate in a randomised
withdrawal study in which they will be randomised either to placebo or to
continue on high dose idebenone treatment for a period of 2 months. During this
period, important additional information not otherwise available from the MES
will be collected, including Patient Reported Outcomes and performance
measures.
Study objective
The primary objective of the PROTI study is to establish whether patients can
correctly determine which treatment assignment they received during the
randomised phase of the trial.
The key secondary objective is to compare the rate of withdrawal from the PROTI
study of patients who have been withdrawn from treatment with high-dose
idebenone with the rate of withdrawal of patients continuing to receive high
dose idebenone.
Secondary objectives are to compare Patient Reported Outcomes (PROs) and
performance measures in FRDA patients who have been withdrawn from treatment
with high-dose idebenone and to compare these outcomes and performance measures
with those from patients continuing to receive high dose idebenone.
Study design
Double-blind, randomised, placebo-controlled, parallel-group multi-centre
withdrawal study
Intervention
Group A will receive Idebenone: Patients <=45 kg: 1350 mg/day - 3 tablets 3
times a day with food
Patients >45 kg: 2250
mg/day - 5 tablets 3 times a day with food
Group B will receive placebo: Placebo <=45 kg - 3 tablets 3 times a day with food
Placebo >45 kg - 5
tablets 3 times a day with food
Study burden and risks
Burden associated with participation as required by study procedures is:
- 2 study visits in period of 2 months per cycle. At each visit following test
will be performed:
Pregnancy test for women of childbearing potential
Routine ECG
Safety blood and urine sample
Performance measures and neurological assessments
- Patients will also complete a Patient Report Outcomes (PRO) Status
Questionnaire at first visit and Daily Patient Diary throughout the study.
- At second visit patient will complet PRO Change Questionnaire.
The risks of treatmant are considered to be acceptable.
Hammerstrasse 49
4410 Liestal
CH
Hammerstrasse 49
4410 Liestal
CH
Listed location countries
Age
Inclusion criteria
1. Completion of Visit 5 (Month 12), Visit 6 (Month 18), or Visit 7 (Month 24) in MICONOS-extension study
2. Body weight >= 25 kg
3. Patients who in the opinion of the investigator are able to comply with the requirements of the study
4. Negative urine pregnancy test (women of childbearing potential)
Exclusion criteria
1. Adverse event during the course of MES which in the opinion of the investigator is attributable to idebenone and precludes further treatment with idebenone
2. Clinically significant abnormalities of clinical haematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of normal of SGOT, SGPT, or creatinine
3. Parallel participation in another clinical drug trial
4. Pregnancy or breast-feeding
5. Abuse of drugs or alcohol
6. 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
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2010-023388-16-NL |
| ClinicalTrials.gov | NCT01303406 |
| CCMO | NL35243.042.11 |