A study to test whether patients can tell if they feel better when they are treated with idebenone

A Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study of Patient Reported Outcomes in Friedreich’s Ataxia Patients after withdrawal from Treatment with Idebenone (PROTI Study) - PROTI

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2010-023388-16-AT

Enrollment

Registered

2011-03-30

Start date

2011-10-20

Completion date

Unknown

Last updated

2012-09-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Friedreich's Ataxia MedDRA version: 13.1 Level: PT Classification code 10017374 Term: Friedreich's ataxia System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: Idebenone Product Code: SNT-MC17 Pharmaceutical Form: Tablet INN or Proposed INN: Idebenone CAS Number: 58186279 Current Sponsor code: SNT-MC17 Concentration unit: mg milligram(s) Concen

Eligibility

Sex/Gender

All

Inclusion criteria

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) Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 6 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

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 outcomes

Primary

Measure	Time frame
Main 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.;Secondary Objective: 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. ;Primary end point(s): • Patient assessment of treatment assignment: Comparison of the proportions of patients randomised to idebenone and placebo who assessed that they received idebenone ;Timepoint(s) of evaluation of this end point: At 2 months after study start	—

Measure

Time frame

Main 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.;Secondary Objective: 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. ;Primary end point(s): • Patient assessment of treatment assignment: Comparison of the proportions of patients randomised to idebenone and placebo who assessed that they received idebenone ;Timepoint(s) of evaluation of this end point: At 2 months after study start

—

Secondary

Measure	Time frame
Secondary end point(s): • Comparison of the proportions of patients randomised to idebenone and placebo who withdrew early due to recurrence or worsening of FRDA symptoms ;Timepoint(s) of evaluation of this end point: Within 2 months (i.e. Early withdrawal visit)	—

Countries

Austria, Germany, Netherlands, United Kingdom

Contacts

Public ContactSylvia Boesch

Medizinische Universitätsklinik Innsbruck

Sylvia.Boesch@i-med.ac.at+43 NA512 504 - 0NA

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026