Phase 2
N=27
Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes
MELAS Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT00887562 ↗Enrolled (actual)
27
Serious AEs
0.0%
Results posted
Aug 2016
Primary outcome: Primary: Mean Change in Cerebral Lactate Concentration (as Measured by Magnetic Resonance Spectroscopy) — -0.09; 0.16; -0.49 IU
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Idebenone (Drug); Placebo (Other)
- Age
- Pediatric, Adult, Older Adult · 8+ yrs
- Sex
- All
- Sponsor
- Michio Hirano
- Primary completion
- Jul 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Change in Cerebral Lactate Concentration (as Measured by Magnetic Resonance Spectroscopy) |
-0.09; 0.16; -0.49 | — |
| SECONDARY Mean Change in Venous Lactate Concentration |
-0.24; 0.7; -0.46 | — |
| SECONDARY Mean Change in Score on the Fatigue Severity Scale (FSS) |
-3.8; -1.3; 4.3 | — |
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 resonance spectroscopy.
Eligibility Criteria
Inclusion Criteria
- 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 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 Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT00887562). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.