Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neu… (NCT02161380) | Clinical Trial Compass
CompletedPhase 1
Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy
United States28 participantsStarted 2014-07-14
Plain-language summary
The study is a dose-escalation study, phase 1. The objective of this proposed clinical trial is to evaluate the safety of mitochondrially targeted ND4 gene therapy with the adeno-associated viral vector in appropriate LHON patients.
Who can participate
Age range15 Years
SexALL
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Inclusion criteria
✓. Age 15 or older;
✓. Patients with LHON and the G11778A mitochondrial DNA mutation. A previous CLIA-certified genetic lab result showing the LHON G11778A mutation will be accepted for inclusion;
✓. Ability to perform tests of visual and retinal function;
✓. Ability to comply with research procedures;
✓. Able and willing to provide informed consent before undergoing any study-related procedures.
✓. Good general health as based on the investigator's assessment of the history, physical examination, and laboratory testing performed at the baseline examination.
Exclusion criteria
✕. Unwilling or unable to give consent,
✕. Unable or unlikely to return for scheduled protocol visits
✕. Pregnant or nursing women or unwillingness for subject with childbearing potential to use contraception during the first year of the study.
✕. Optic disc drusen on exam or in previous history.
✕. Ocular diseases or visual dysfunction conditions other than refractive error (e.g. amblyopia, glaucoma, etc.) in the eye selected for the injection.
✕. Previous eye surgery in the eye selected for injection.
. Aspartate transaminase (AST)/alanine transaminase (ALT) \>5.0 x upper limit of normal (ULN); Total bilirubin \>3 x ULN; Hemoglobin \< 8 g/dL; neutrophil count \<1.0 x 109/L; or platelet count \< 50 x 109/L
✕. Type I diabetes or the presence of diabetic retinopathy