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Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients (IONIA-E)

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ClinicalTrials.gov Identifier: NCT00697073
Recruitment Status : Completed
First Posted : June 13, 2008
Results First Posted : August 4, 2011
Last Update Posted : June 4, 2013
Information provided by (Responsible Party):
Santhera Pharmaceuticals

Brief Summary:
This study is meant to assess the safety and tolerability of idebenone in patients with Friedreich's Ataxia over a 12 months period.

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

Detailed Description:
The study involves 6 clinic visits.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 68 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase III Open-Label, Single Group Extension Study of the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients
Study Start Date : July 2008
Primary Completion Date : May 2010
Study Completion Date : May 2010

Arm Intervention/treatment
Experimental: 1
high dose Idebenone
Drug: Idebenone
Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients > 45 kg/99 lbs: idebenone 2250 mg/day

Primary Outcome Measures :
  1. Change in ICARS [ Time Frame: baseline and 12 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 (Friedreich's Ataxia Rating Scale) [ Time Frame: baseline and 12 Months ]
  2. Nature and Frequency of Adverse Events [ Time Frame: 12 Months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion criteria:

  • Friedreich's ataxia patients completing core study SNT-III-002 (NCT00537680) and presenting at Week 24 (Visit 5) of that study
  • Body weight ≥ 25kg/55 lbs
  • Negative urine pregnancy test
  • Patients who in the opinion of the investigator are able to comply with the requirements of this study

Exclusion criteria:

  • Adverse events during the course of SNT-III-002(NCT00537680)which in the opinion of the investigator are attributable to idebenone and preclude further treatment with idebenone
  • Clinically significant abnormalities of clinical hematology or biochemistry including, but not limited to, elevations greater than 2 times the upper limit of normal of AST, ALT or creatinine
  • Treatment with coenzyme Q10, vitamin E (if taken at a dose 5 times above the daily requirement) or other sources of idebenone within the past month
  • Parallel participation in another clinical drug trial
  • Past or present history of abuse of drugs or alcohol
  • Pregnancy or breast-feeding

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): NCT00697073

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
Principal Investigator: Susan Perlman, MD University of California, Los Angeles
Principal Investigator: David Lynch, MD Children's Hospital of Philadelphia

Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00697073     History of Changes
Other Study ID Numbers: SNT-III-002-E
First Posted: June 13, 2008    Key Record Dates
Results First Posted: August 4, 2011
Last Update Posted: June 4, 2013
Last Verified: May 2013

Keywords provided by Santhera Pharmaceuticals:
Friedreich's Ataxia

Additional relevant MeSH terms:
Cerebellar Ataxia
Friedreich Ataxia
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
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
Growth Substances