NCT00887562

Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes

A Phase IIa Double-Blind, Randomized, Placebo-Controlled, Dose-Finding Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis and Stroke-like Episodes

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 27 (actual)
• Sponsor: Michio Hirano · Academic / Other
• Sex: All
• Age: 8 Years – 65 Years
• Healthy volunteers: Not accepted

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.

Detailed description

MELAS (Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-Like Episodes), a progressive and often devastating multisystem disorder, is most commonly associated with mitochondrial Deoxyribonucleic acid (mtDNA) point mutation at nucleotide 3243. Seizures, cognitive deterioration, and neurobehavioral abnormalities are frequent features of this disease which typically shortens life expectancy. Idebenone, an ATP production modulator and antioxidant, improves neurological function in Friedreich's ataxia, a disease also associated with mitochondrial dysfunction. Given that there is no effective treatment for MELAS, the investigators propose a Phase II proof of concept trial of idebenone to study its preliminary efficacy in patients with MELAS and the A3243G mtDNA mutation, and to study its safety and tolerability in this patient group. The investigators propose to evaluate 21 patients with the A3243G mitochondrial DNA mutation and MELAS (defined by a history of either seizures or stroke). Patients will receive idebenone (900 mg/day or 2250 mg/day) or matching placebo for one month. The primary outcome measure is cerebral lactate levels measured by Magnetic Resonance Spectroscopy (MRS), a biomarker associated with disease worsening. This study will help the investigators to determine if there is sufficient signal to proceed to efficacy studies. Also it will provide additional information on the safety and tolerability of two different doses of idebenone in MELAS.

Conditions

• MELAS Syndrome

Interventions

Type	Name	Description
DRUG	Idebenone	900 mg/day for 1 month
DRUG	Idebenone	2250 mg/day for 1 month
OTHER	Placebo	Placebo - No idebenone

Timeline

• Start date: 2009-05-01
• Primary completion: 2012-07-01
• Completion: 2012-07-01
• First posted: 2009-04-24
• Last updated: 2016-10-26
• Results posted: 2016-08-10

Locations

1 site across 1 country: United States