September 7, 2007 |
April 15, 2009 |
December 2007 |
ICARS [ Time Frame: 6 months ] [ Designated as safety issue: No ] |
Same as current |
Complete list of historical versions of study NCT00537680 on ClinicalTrials.gov Archive Site |
- FARS [ Time Frame: 6 Months ] [ Designated as safety issue: No ]
- ADL of FARS [ Time Frame: 6 Months ] [ Designated as safety issue: No ]
- FACT [ Time Frame: 6 Months ] [ Designated as safety issue: No ]
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Same as current |
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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 were all patients will receive high dose Idebenone. |
Phase III |
Interventional |
Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Parallel Assignment, Safety/Efficacy Study |
Friedreich's Ataxia |
- Drug: Idebenone
- Drug: Placebo
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- Experimental: mid dose Idebenone
- Experimental: high dose Idebenone
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Active, not recruiting |
51 |
May 2009 |
May 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
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Both |
8 Years to 17 Years |
No |
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United States |
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NCT00537680 |
Thomas Meier, PhD / Chief Scientific Officer, Santhera Pharmaceuticals (Switzerland) Ltd. |
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Santhera Pharmaceuticals |
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Principal Investigator: |
Susan Perlman, MD |
University of California, Los Angeles |
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Principal Investigator: |
David Lynch, MD |
Children's Hospital of Philadelphia |
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Santhera Pharmaceuticals |
April 2009 |