Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia
- Registration Number
- NCT00537680
- Lead Sponsor
- Santhera Pharmaceuticals
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 70
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Arm 2 - up to 2250 mg/day Idebenone Idebenone high dose Idebenone Arm 3 - Placebo Placebo Placebo Arm 1 - 900 mg/day Idebenone Idebenone mid dose Idebenone
- Primary Outcome Measures
Name Time Method Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24 baseline and 6 months International Cooperative Ataxia Rating Scale (ICARS):
ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability.
Minimum score: 0, maximum score 100, higher score indicates greater impairment
Calculation details: ICARS score at week 24 minus ICARS score baseline
- Secondary Outcome Measures
Name Time Method Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12 baseline and 12 weeks To compare the efficacy of 3 months' treatment with 2 different doses of idebenone with that of placebo on neurological impairment as assessed by the International Cooperative Ataxia Rating Scale (ICARS)
International Cooperative Ataxia Rating Scale (ICARS):
ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability.
Minimum score: 0, maximum score 100, higher score indicates greater impairment
Calculation details: ICARS score at week 12 minus ICARS score baselineAbsolute Change From Baseline to Week 24 in Left Ventricular Mass Index (LVMI) baseline and 6 months To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24 baseline and 6 months To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on neurological function as assessed by the Friedreich's Ataxia Rating Scale (FARS)
FARS consists of a 25 manoeuvre exam along with 4 quantitative performance measures. The neurological exam covers bulbar function, upper limb coordination, lower limb coordination, peripheral nervous system function, deep tendon reflexes, stability and gait. The use of FARS has been recently validated as a sensitive scale for this population.
The FARS exam scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability.
Calculation details FARS score at week 24 minus FARS score baselineActivities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24 baseline and 6 months To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on Activities of Daily Living (ADL) assessed by the ADL scale of the Friedreich's Ataxia Rating Scale (FARS)
score 0 to 36, higher score indicates greater impairmentPercent Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle baseline and 6 months To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Absolute Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle baseline and 6 months To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Absolute Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS) baseline and 6 months To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Percent Change From Baseline to Week 24 in Left Ventricular Mass Index baseline and 6 months To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Absolute Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle baseline and 6 months To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Percent Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle baseline and 6 months To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Percent Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS) baseline and 24 weeks To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Trial Locations
- Locations (2)
David Geffen School of Medicine, UCLA
🇺🇸Los Angeles, California, United States
The Children's Hopsital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States