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

Friedreich's Ataxia Clinical Trial

— IONIA  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00537680
• Other study ID #: SNT-III-002
• Status: Completed
• Phase: Phase 3
• Start date: December 2007
• Est. completion date: April 2009

Study information

• Verified date: June 2023
• Source: Santhera Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 70
• Est. completion date: April 2009
• Est. primary completion date: April 2009
• Accepts healthy volunteers: No
• Gender: All
• Age group: 8 Years to 17 Years

Eligibility

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

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Friedreich Ataxia
• Friedreich's Ataxia

Intervention

Drug:
• Idebenone
Patients = 45 kg/99 lbs: idebenone 450 mg/day; Patients > 45 kg/99 lbs: idebenone 900 mg/day
• Idebenone
Patients = 45 kg/99 lbs: idebenone 1350 mg/day; Patients > 45 kg/99 lbs: idebenone 2250 mg/day
• Placebo
Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets.

Locations

Country	Name	City	State
United States	David Geffen School of Medicine, UCLA	Los Angeles	California
United States	The Children's Hopsital of Philadelphia	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Santhera Pharmaceuticals

Country where clinical trial is conducted

United States, 

References & Publications (1)

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 Fe — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24	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	baseline and 6 months
Secondary	Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24	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 baseline	baseline and 6 months
Secondary	Activities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24	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 impairment	baseline and 6 months
Secondary	Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12	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 baseline	baseline and 12 weeks
Secondary	Absolute Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle	To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography	baseline and 6 months
Secondary	Percent Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle	To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography	baseline and 6 months
Secondary	Absolute Change From Baseline to Week 24 in Left Ventricular Mass Index (LVMI)	To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography	baseline and 6 months
Secondary	Percent Change From Baseline to Week 24 in Left Ventricular Mass Index	To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography	baseline and 6 months
Secondary	Absolute Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle	To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography	baseline and 6 months
Secondary	Percent Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle	To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography	baseline and 6 months
Secondary	Absolute Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS)	To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography	baseline and 6 months
Secondary	Percent Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS)	To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography	baseline and 24 weeks