Clinical trial NCT00537680: Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia
Official title: A Phase III Double-Blind, Randomized, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Friedreich's Ataxia
Keywords: Friedreich's Ataxia
Idebenone
ICARS
Lead sponsor: Santhera Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2007-10-01
Enrollment: 70
Sex: ALL
Minimum age (years): 8
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24
timeFrame: baseline and 6 months
Secondary outcomes:
measure: Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24
timeFrame: baseline and 6 months
measure: Activities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24
timeFrame: baseline and 6 months
measure: Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12
timeFrame: baseline and 12 weeks
measure: Absolute Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle
timeFrame: baseline and 6 months
measure: Percent Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle
timeFrame: baseline and 6 months
measure: Absolute Change From Baseline to Week 24 in Left Ventricular Mass Index (LVMI)
timeFrame: baseline and 6 months
measure: Percent Change From Baseline to Week 24 in Left Ventricular Mass Index
timeFrame: baseline and 6 months
measure: Absolute Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle
timeFrame: baseline and 6 months
measure: Percent Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle
timeFrame: baseline and 6 months
measure: Absolute Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS)
timeFrame: baseline and 6 months
measure: Percent Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS)
timeFrame: baseline and 24 weeks
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: David Geffen School of Medicine, UCLA
state: California
city: Philadelphia
country: United States
facility: The Children's Hopsital of Philadelphia
state: Pennsylvania
Study officials:
affiliation: University of California, Los Angeles
name: Susan Perlman, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital of Philadelphia
name: David Lynch, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Meier T, Perlman SL, Rummey C, Coppard NJ, Lynch DR. Assessment of neurological efficacy of idebenone in pediatric patients with Friedreich's ataxia: data from a 6-month controlled study followed by a 12-month open-label extension study. J Neurol. 2012 Feb;259(2):284-91. doi: 10.1007/s00415-011-6174-y. Epub 2011 Jul 22.
citation: Lagedrost SJ, Sutton MS, Cohen MS, Satou GM, Kaufman BD, Perlman SL, Rummey C, Meier T, Lynch DR. Idebenone in Friedreich ataxia cardiomyopathy-results from a 6-month phase III study (IONIA). Am Heart J. 2011 Mar;161(3):639-645.e1. doi: 10.1016/j.ahj.2010.10.038. Epub 2011 Jan 31.
citation: Lynch DR, Perlman SL, Meier T. A phase 3, double-blind, placebo-controlled trial of idebenone in friedreich ataxia. Arch Neurol. 2010 Aug;67(8):941-7. doi: 10.1001/archneurol.2010.168.