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Completed

NCT Number: NCT00537680

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

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.

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Key information

Conditions

Friedreich's Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Friedreich Ataxia Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Spinal Cord Diseases Spinocerebellar Degenerations

Age range

8 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

David Geffen School of Medicine, UCLA, Los Angeles, California, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Idebenone

Drug

Patients ≤ 45 kg/99 lbs: idebenone 450 mg/day; Patients > 45 kg/99 lbs: idebenone 900 mg/day

Placebo

Drug

Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets.

Primary outcomes

  1. Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24

    Time frame: 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 outcomes

  1. Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24

    Time frame: 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 baseline

  2. Activities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24

    Time frame: 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 impairment

  3. Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12

    Time frame: 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 baseline

  4. Absolute Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle

    Time frame: 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

  5. Percent Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle

    Time frame: 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

  6. Absolute Change From Baseline to Week 24 in Left Ventricular Mass Index (LVMI)

    Time frame: 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

  7. Percent Change From Baseline to Week 24 in Left Ventricular Mass Index

    Time frame: 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

  8. Absolute Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle

    Time frame: 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

  9. Percent Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle

    Time frame: 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

  10. Absolute Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS)

    Time frame: 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

  11. Percent Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS)

    Time frame: 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

Sponsors and collaborators

Lead sponsor

Santhera Pharmaceuticals

Industry

Registry information

Official study 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

Acronym: IONIA

Important dates

Study start
2007
Primary completion
2009
Study completion
2009
First posted
Oct 1, 2007
Registry last updated
Jul 11, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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