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

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ClinicalTrials.gov Identifier: NCT00537680
Recruitment Status : Completed
First Posted : October 1, 2007
Results First Posted : December 29, 2011
Last Update Posted : February 3, 2012
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Friedreich's Ataxia Drug: Idebenone Drug: Placebo Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 70 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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 Start Date : December 2007
Actual Primary Completion Date : April 2009
Actual Study Completion Date : April 2009

Arm Intervention/treatment
Experimental: 1
mid dose Idebenone
Drug: Idebenone
Patients ≤ 45 kg/99 lbs: idebenone 450 mg/day; Patients > 45 kg/99 lbs: idebenone 900 mg/day

Experimental: 2
high dose Idebenone
Drug: Idebenone
Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients > 45 kg/99 lbs: idebenone 2250 mg/day

Placebo Comparator: 3 Drug: Placebo
Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets.

Primary Outcome Measures :
  1. ICARS [ 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.

    Scores for each subscale quantify the extent of ataxia in each clinically important area. Subscale scores are summed to give a total score ranging from 0 (best) to 100 (worst).

Secondary Outcome Measures :
  1. FARS [ Time Frame: 6 Months ]
    Friedreich's Ataxia Rating Scale

  2. ADL of FARS [ Time Frame: 6 Months ]
    ADL=Activities of Daily Living

  3. FACT [ Time Frame: 6 Months ]
    Friedreich's Ataxia Composite Test

Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00537680

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United States, California
David Geffen School of Medicine, UCLA
Los Angeles, California, United States, 90095-1769
United States, Pennsylvania
The Children's Hopsital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Santhera Pharmaceuticals
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Principal Investigator: Susan Perlman, MD University of California, Los Angeles
Principal Investigator: David Lynch, MD Children's Hospital of Philadelphia
Publications of Results:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00537680    
Other Study ID Numbers: SNT-III-002
First Posted: October 1, 2007    Key Record Dates
Results First Posted: December 29, 2011
Last Update Posted: February 3, 2012
Last Verified: February 2012
Keywords provided by Santhera Pharmaceuticals:
Friedreich's Ataxia
Additional relevant MeSH terms:
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Cerebellar Ataxia
Friedreich Ataxia
Neurologic Manifestations
Nervous System Diseases
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs