Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension)

Santhera

Status and phase: Completed; Phase 3

Conditions: Freidreich's Ataxia

Treatments: Drug: idebenone

Study type: Interventional

Funder types: Industry

Identifiers

NCT00993967

SNT-III-001-E

Details and patient eligibility

About

This is an Extension study of the MICONOS main randomised placebo-controlled trial (NCT00905268), and open to those patients completing the main study. The scientific aim of this extension study is to monitor safety and tolerability of idebenone over two years in patients with Friedreich's Ataxia.

Enrollment: 200 patients

Sex: All

Ages: 9+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Completion of 52 weeks in study SNT-III-001
• Body weight ≥ 25 kg
• Negative urine pregnancy test
• Eligibility to participate in the present extension study as confirmed by investigator

Exclusion criteria

• Safety or tolerability issues arising during the course of SNT-III-001 which in the opinion of the investigator preclude further treatment with idebenone
• Clinically significant abnormalities of haematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of SGOT, SGPT or creatinine
• Parallel participation in another clinical drug trial
• Pregnancy or breast-feeding
• Abuse of drugs or alcohol

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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200 participants in 1 patient group

Idebenone

Experimental group

Description:

1350 mg/day or 2250 mg/day for patients weighing ≤45 kg or >45 kg, respectively.In case of poor tolerability, dose reduction to 450 mg/day or 900 mg/day, respectively, were allowed.

Treatment:

Drug: idebenone