Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (IONIA)

Santhera

Status and phase: Completed; Phase 3

Conditions: Friedreich's Ataxia

Treatments: Drug: Placebo; Drug: Idebenone

Study type: Interventional

Funder types: Industry

Identifiers

NCT00537680

SNT-III-002

Details and patient eligibility

About

This study is meant to assess the effectiveness of idebenone on neurological outcome measures in patients with Friedreich's Ataxia over a 6 months period.

Full description

The study involves 6 clinic visits and upon completion the possibility to join a 12 months extension study where all patients will receive high dose Idebenone.

Enrollment: 70 patients

Sex: All

Ages: 8 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Documented diagnosis of Friedreich's Ataxia (FRDA) with confirmed FRDA mutations
• Patients 8 - 17 years of age at baseline
• Patients with a body weight greater than 25 kg/55 lbs at baseline
• Patients able to walk at least 10 meters without accompanying person (ICARS Walking Capacities score ≤6) at screening and baseline
• Patients who in the opinion of the investigator are able to comply with the requirements of the study, including swallowing the study medication
• Negative urine pregnancy test at screening and baseline (female patients of childbearing potential)

Exclusion criteria

• Treatment with idebenone, Coenzyme Q10 or vitamin E (if taken at a dose 5 times above the daily requirement) within the past month
• Patients with International Cooperative Ataxia Rating Scale (ICARS) score of greater than 54 or less than 10 at screening
• Pregnancy and/or breast-feeding
• Clinically significant abnormalities of clinical hematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of normal of Aspartate Aminotransferase (AST; also known as GOT), Alanine Aminotransferase (ALT; also known as GPT) or creatinine
• History of abuse of drugs or alcohol
• Participation in the previous (Phase II) study of idebenone at the National Institutes of Health (NIH)
• Participation in a trial of another investigational drug within the last 3 months

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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70 participants in 3 patient groups, including a placebo group

Arm 1 - 900 mg/day Idebenone

Experimental group

Description:

mid dose Idebenone

Treatment:

Drug: Idebenone

Drug: Idebenone

Arm 2 - up to 2250 mg/day Idebenone

Experimental group

Description:

high dose Idebenone

Treatment:

Drug: Idebenone

Drug: Idebenone

Arm 3 - Placebo

Placebo Comparator group

Description:

Placebo

Treatment:

Drug: Placebo