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

Primary Purpose

Friedreich's Ataxia

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Idebenone

Placebo

About this trial

This is an interventional treatment trial for Friedreich's Ataxia focused on measuring randomized withdrawal, idebenone, friedreich's ataxia, Miconos, Patient reported outcome, ICARS

Eligibility Criteria

10 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

The National Hospital, University College London

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Experimental

Arm Label

Placebo

idebenone

Arm Description

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

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

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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

Full Information

NCT ID

NCT01303406

First Posted

February 22, 2011

Last Updated

February 10, 2016

Sponsor

Santhera Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT01303406

Brief Title

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

Acronym

PROTI

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

Study Type

Interventional

2. Study Status

Record Verification Date

February 2016

Overall Recruitment Status

Completed

Study Start Date

April 2011 (undefined)

Primary Completion Date

July 2012 (Actual)

Study Completion Date

July 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Santhera Pharmaceuticals

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Friedreich's Ataxia

Keywords

randomized withdrawal, idebenone, friedreich's ataxia, Miconos, Patient reported outcome, ICARS

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

29 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Arm Title

idebenone

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Idebenone

Other Intervention Name(s)

Catena (approved name in Canada)

Intervention Description

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).

Intervention Type

Drug

Intervention Name(s)

Placebo

Primary Outcome Measure Information:

Title

Patient Assessment of Treatment Assignment: Comparison of the Proportions of Patients Randomised to Idebenone and Placebo Who Assessed That They Received Idebenone

Description

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

Secondary Outcome Measure Information:

Title

Comparison of the Percentage of Participants Randomised to Idebenone and Placebo Who Withdrew Early Due to Recurrence or Worsening of FRDA Symptoms

Description

There was no Withdrawal due to recurrence or worsening of FRDA symptoms

Time Frame

Within 2 months (i.e. Early withdrawal visit)

10. Eligibility

Sex

All

Minimum Age & Unit of Time

10 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Paola Giunti, M.D

Organizational Affiliation

Institute of Neurology, The National Hospital, University College London

Official's Role

Principal Investigator

Facility Information:

City

Innsbruck

Country

Austria

City

Bonn

Country

Germany

City

München

Country

Germany

City

Tübingen

Country

Germany

City

Groningen

Country

Netherlands

Facility Name

The National Hospital, University College London

City

London

ZIP/Postal Code

WC 1N 3BG

Country

United Kingdom

12. IPD Sharing Statement

Learn more about this trial

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

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