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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
History of Changes

September 7, 2007
February 2, 2012
December 2007
April 2009   (final data collection date for primary outcome measure)
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).
ICARS [ Time Frame: 6 months ]
Complete list of historical versions of study NCT00537680 on ClinicalTrials.gov Archive Site
  • 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
  • FARS [ Time Frame: 6 Months ]
  • ADL of FARS [ Time Frame: 6 Months ]
  • FACT [ Time Frame: 6 Months ]
Not Provided
Not Provided
 
Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia
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

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.

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.
Interventional
Phase 3
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Friedreich's Ataxia
  • Drug: Idebenone
    Patients ≤ 45 kg/99 lbs: idebenone 450 mg/day; Patients > 45 kg/99 lbs: idebenone 900 mg/day
  • Drug: Idebenone
    Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients > 45 kg/99 lbs: idebenone 2250 mg/day
  • Drug: Placebo
    Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets.
  • Experimental: 1
    mid dose Idebenone
    Intervention: Drug: Idebenone
  • Experimental: 2
    high dose Idebenone
    Intervention: Drug: Idebenone
  • Placebo Comparator: 3
    Intervention: Drug: Placebo

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
70
April 2009
April 2009   (final data collection date for primary outcome measure)

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
Both
8 Years to 17 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00537680
SNT-III-002
Yes
Santhera Pharmaceuticals
Santhera Pharmaceuticals
Not Provided
Principal Investigator: Susan Perlman, MD University of California, Los Angeles
Principal Investigator: David Lynch, MD Children's Hospital of Philadelphia
Santhera Pharmaceuticals
February 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP