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

Santhera

Status and phase: Completed; Phase 3

Conditions: Friedreich's Ataxia

Treatments: Drug: Idebenone; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT01303406

SNT-III-004

Details and patient eligibility

About

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.

Enrollment: 29 patients

Sex: All

Ages: 10+ years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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29 participants in 2 patient groups, including a placebo group

Placebo

Placebo Comparator group

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

Treatment:

Drug: Placebo

idebenone

Experimental group

Description:

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

Treatment:

Drug: Idebenone