Conditions: Friedreich's Ataxia
Keywords: randomized withdrawal idebenone friedreich's ataxia Miconos Patient reported outcome ICARS
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 29
Healthy volunteers: no

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:

Arms:
arm 1: 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 arm 2: 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

Interventions: intervention 1: Idebenone intervention 2: Placebo
Intervention descriptions: intervention 1: 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 2: None

Protocol document: