A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients (MICONOS)

Santhera

Status and phase: Completed; Phase 3

Conditions: Friedreich's Ataxia

Treatments: Drug: idebenone; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT00905268

SNT-III-001

Details and patient eligibility

About

The purpose of this trial is to study the efficacy, safety and tolerability of idebenone in 12 months of treatment in children and adults with Friedreich's Ataxia. This is a randomised placebo-controlled double-blind trial conducted in Europe. Efficacy outcomes include measures of neurological impairment and function, and measures of the heart.

Full description

Idebenone, a short-chain analogue of Co-enzyme Q10 (CoQ10), has the potential to moderate underlying causes of Friedreich's Ataxia through its antioxidant activity and its role as an electron carrier in the respiratory chain promoting mitochondrial ATP production.

The current 12-month placebo-controlled treatment study in 232 patients aims to confirm the positive effect of idebenone on neurological function, as for instance observed in the recent 48-patient, 6-month NICOSIA study in children, using the International Cooperative Ataxia Rating Scale (ICARS) and the newly developed Friedreich's Ataxia Rating Scale (FARS).

In addition, the study aims to confirm the positive effect on cardiomyopathy associated with FRDA observed in several small studies. Cardiac anatomy and function will be assessed using echocardiography, tissue Doppler imaging and cardiac MRI methods in patients with FRDA cardiomyopathy. In addition exercise capacity, measured as peak workload, will be assessed in patients able to comply with a modified exercise protocol.

Enrollment: 232 patients

Sex: All

Ages: 8+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Documented diagnosis of FRDA with confirmed FRDA mutations
• Patients 8 years of age or older at baseline
• Patients with body weight ≥ 25kg
• Patients who in the opinion of the investigator are able to comply with the requirements of the study, including swallowing the medication
• Negative urine pregnancy test at screening and at baseline (women of childbearing potential)

Exclusion criteria

• Treatment with idebenone or Coenzyme Q10 within the past 1 month
• Pregnancy and/or breast-feeding
• Clinically significant abnormalities of clinical haematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of normal of SGOT, SGPT, or creatinine
• Past or present history of abuse of drugs or alcohol

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

232 participants in 4 patient groups, including a placebo group

Group A: Idebenone

Experimental group

Description:

Patients under/equal 45 kg: idebenone 180 mg/day Patients over 45 kg: idebenone 360 mg/day

Treatment:

Drug: idebenone

Group B: Idebenone

Experimental group

Description:

Patients under/equal 45 kg: idebenone 450 mg/day Patients over 45 kg: idebenone 900 mg/day

Treatment:

Drug: idebenone

C: Idebenone

Experimental group

Description:

Patients under/equal 45 kg: idebenone 1350 mg/day Patients over 45 kg: idebenone 2250 mg/day

Treatment:

Drug: idebenone

D: Placebo

Placebo Comparator group

Description:

placebo

Treatment:

Drug: Placebo