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

Santhera Pharmaceuticals29 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Friedreich's Ataxia

Interventions

IdebenoneDRUG

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).

PlaceboDRUG

Eligibility

Sex: ALLMin age: 10 Years

Medical Language ↔ Plain English

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

Locations (6)

Unnamed facility

Innsbruck, Austria

Unnamed facility

Bonn, Germany

Unnamed facility

München, Germany

Unnamed facility

Tübingen, Germany

Unnamed facility

Groningen, Netherlands

Outcomes

Primary Outcomes

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.

Time frame: At 2 months after study start

Secondary Outcomes

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

Time frame: Within 2 months (i.e. Early withdrawal visit)

Data from ClinicalTrials.gov

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