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

Study Overview
=================
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.

Official Title
-----------------
A Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study of Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone

Conditions
-----------------
Friedreich's Ataxia

Intervention / Treatment
-----------------
* Drug: Idebenone
* Drug: Placebo


Participation Criteria
=================
Eligibility Criteria
-----------------
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

Ages Eligible for Study
-----------------
Minimum Age: 10 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Placebo<br>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 | Drug: Placebo<br> <br> |
| Experimental: idebenone<br>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 | Drug: Idebenone<br>* 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).<br>* Other names: Catena (approved name in Canada);|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | At 2 months after study start | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | Within 2 months (i.e. Early withdrawal visit) | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
randomized withdrawal, idebenone, friedreich's ataxia, Miconos, Patient reported outcome, ICARS