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

NCT ID: NCT01303406

Last Updated: 2016-03-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 29 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-04-30
• Study Completion Date: 2012-07-31

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.

Conditions

Friedreich's Ataxia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

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

Group Type: EXPERIMENTAL

Idebenone

Intervention Type: DRUG

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).

Interventions

Idebenone

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

Placebo

Intervention Type: DRUG

Other Intervention Names

Catena (approved name in Canada)

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

• Minimum Eligible Age: 10 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Santhera Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Paola Giunti, M.D

Role: PRINCIPAL_INVESTIGATOR

Institute of Neurology, The National Hospital, University College London

Locations

Innsbruck, , Austria

Bonn, , Germany

München, , Germany

Tübingen, , Germany

Groningen, , Netherlands

The National Hospital, University College London

London, , United Kingdom

Countries

Austria; Germany; Netherlands; United Kingdom

Other Identifiers

SNT-III-004

Identifier Type: -

Identifier Source: org_study_id