Status:
COMPLETED
Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes
Lead Sponsor:
Michio Hirano
Collaborating Sponsors:
Santhera Pharmaceuticals
Conditions:
MELAS Syndrome
Eligibility:
All Genders
8-65 years
Phase:
PHASE2
Brief Summary
The purpose of this study is to compare the efficacy of two (2) different doses of idebenone with that of a placebo over a one month period on cerebral lactate concentration as measured by magnetic re...
Detailed Description
MELAS (Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-Like Episodes), a progressive and often devastating multisystem disorder, is most commonly associated with mitochondrial Deoxyribonu...
Eligibility Criteria
Inclusion
- Diagnosis of MELAS with confirmed A3243G mtDNA mutation, or evidence of central nervous system involvement (cognitive problems, migraines, memory loss)
- Cerebral lactate level equal to or greater than 5.0 i.u. at baseline
- Patients at least 8 and \< 65 years of age at baseline
- Patients with a body weight \> 37 kg/82 lbs at baseline
- Stable co-medication/vitamins/supplements within 1 month prior to 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
- Contraindication to MRS (e.g. metal implant, claustrophobia)
- Stroke like event within 2 months prior to baseline
- Treatment with idebenone at any dose, or coenzyme Q10 at doses above 100mg/d within 1 month prior to baseline
- Inadequate contraception use
- 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), alanine aminotransferase (ALT) or creatinine
- Current abuse of drugs or alcohol
- Participation in a trial of another investigational drug within the last month
- Other factor that, in the investigator's opinion, excludes the patient from entering the study
Key Trial Info
Start Date :
May 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2012
Estimated Enrollment :
27 Patients enrolled
Trial Details
Trial ID
NCT00887562
Start Date
May 1 2009
End Date
July 1 2012
Last Update
October 26 2016
Active Locations (1)
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1
Columbia University Medical Center
New York, New York, United States, 10032