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

Phase 3

Completed

• Conditions: Friedreich's Ataxia
• Interventions: Drug: Placebo; Drug: Idebenone

• Registration Number: NCT01303406
• Lead Sponsor: Santhera Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2011-02-22
• Study First Submitted QC: 2011-02-23
• Study First Posted: 2011-02-24
• Study Start: 2011-04
• Primary Completion: 2012-07
• Study Completion: 2012-07
• Results First Submitted: 2015-12-11
• Status Verified: 2016-02
• Results First Submitted QC: 2016-02-10
• Last Update Submitted: 2016-02-10
• Results First Posted: 2016-03-09
• Last Update Posted: 2016-03-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 29

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	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	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

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

The National Hospital, University College London; 🇬🇧 London, United Kingdom