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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00537680
First received: September 7, 2007
Last updated: February 2, 2012
Last verified: February 2012
  Purpose

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 Intervention Phase
Friedreich's Ataxia
Drug: Idebenone
Drug: Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:


Further study details as provided by Santhera Pharmaceuticals:

Primary Outcome Measures:
  • ICARS [ Time Frame: baseline and 6 months ] [ Designated as safety issue: No ]

    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:
  • FARS [ Time Frame: 6 Months ] [ Designated as safety issue: No ]
    Friedreich's Ataxia Rating Scale

  • ADL of FARS [ Time Frame: 6 Months ] [ Designated as safety issue: No ]
    ADL=Activities of Daily Living

  • FACT [ Time Frame: 6 Months ] [ Designated as safety issue: No ]
    Friedreich's Ataxia Composite Test


Enrollment: 70
Study Start Date: December 2007
Study Completion Date: April 2009
Primary Completion Date: April 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

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.

  Eligibility

Ages Eligible for Study:   8 Years to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00537680

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

Publications:
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00537680     History of Changes
Other Study ID Numbers: SNT-III-002
Study First Received: September 7, 2007
Results First Received: June 7, 2011
Last Updated: February 2, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Santhera Pharmaceuticals:
Friedreich's Ataxia
Idebenone
ICARS

Additional relevant MeSH terms:
Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinocerebellar Degenerations
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Idebenone
Antioxidants
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Protective Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on September 18, 2014