Exploratory Clinical Trial of EPI-743 in Japanese MELAS Syndrome Patients

Not Applicable

Recruiting

• Conditions: MELAS: mitochondrial myopathy, encephalopathy, lactic acidosis and stroke-like episodes

• Registration Number: JPRN-UMIN000010783
• Lead Sponsor: ational Center of Neurology and Psychiatry, Japan

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2013-05-23
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

Not provided

Exclusion Criteria

1) Patients for whom the MELAS syndrome genetic mutation has not been confirmed 2) Patients who cannot undergo magnetic resonance spectroscopy (MRS) 3) Patients allergic to EPI-743 or to sesame oil 4) Patients with a bleeding tendency, or with abnormal PT/PTT values (INR 1.5 or higher) 5) Patients with AST or ALT values of more than double the institutional limit 6) Patients with renal failure and who require dialysis 7) Patients with fat malabsorption syndrome that impedes drug absorption 8) Patients with pyruvate dehydrogenase (PDH) deficiency or other inborn error of metabolism 9) Patients who are pregnant or planning to become pregnant 10) Patients with an alcohol dependency or who are illegal drug users 11) Patients with severe heart failure accompanied by lactate acidosis 12) Patients who have received another trial drug or test drug within three months of the start of dosing of the test drug in this trial 13) Patients with a history of severe drug hypersensitivity 14) Other patients deemed by the researchers to be unsuitable for this trial

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Tabulate EPI-743 blood concentrations and calculate fundamental pharmacokinetic parameters

Secondary Outcome Measures

Name	Time	Method
1) Lactate levels at MRS testing 2) blood and CSF concentrations of lactate and pyruvate 3) QOL values (SF-36, PedsQL) 4) Mitochondrial disease evaluation scale scores (NPMDS or NMDAS) 5) Incidence of abnormal brain waves 6) Incidence of epileptiform seisures 7) Fatigue level 8) Safety evaluation