MELAS Syndrome Clinical Trial
Official title:
Metabolic and Immune Responses to TIV in Patients With Mitochondrial Disease
This pilot clinical study, funded by the National Institutes of Health, will evaluate the safety and metabolic responses to a licensed inactivated seasonal influenza vaccine (TIV). This single arm study will consist of two cohorts: MELAS syndrome volunteers (a specific identified disorder of mitochondrial dysfunction: mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes) between 13-60 years for OR adult control volunteers between 18-65 years of age. Both cohorts will receive the same treatment: a single vaccination with an FDA-licensed intramuscular seasonal trivalent inactivated influenza vaccine (TIV).
Mitochondrial disease constitutes the most common neurometabolic disease of childhood with
an estimated minimal lifetime risk of developing this disorder of 1 in 5000 live births.
This pilot study will for the first time prospectively assess the safety of seasonal flu
immunization in a subgroup of patients with MELAS syndrome. Comparing their responses to
those of healthy young control volunteers will provide new information about the safety and
immunogenicity of flu vaccination in this important sub-population and help guide the design
of future studies that can be carried out in younger children.
All participants will receive a single administration of a licensed influenza vaccine. Prior
to vaccination, participants will provide information regarding health history and responses
to health questionnaires. Vital signs will be assessed. A blood sample and urine specimen
will be collected prior to vaccination, and at 6 hours, 5-7 days and 26-30 days
post-immunization. Volunteers will be asked to keep a diary of symptoms post-vaccination.
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Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Basic Science
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