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Gene Therapy Trial Report

Summary

Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy


NCTID NCT02161380 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Leber Hereditary Optic Neuropathy (LHON)
Disease Ontology Term DOID:705
Compound Name ScAAV2-P1ND4v2
Sponsor Byron Lam
Funder Type Other
Recruitment Status
Completed
Enrollment Count 28 (ACTUAL)
Results Posted View Results

Therapy Information


Target Gene/Variant ND4
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravitreal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV2
Dose 1 5.0E8 vg/eye (n=9)
Dose 2 2.4E9 vg/eye (n=3)
Dose 3 2.5E9 vg/eye (n=9)
Dose 4 1.0E10 vg/eye (n=7)

Study Record Dates


Current Stage Phase1
Submit Date 2014-06-06
Completion Date 2025-03-31
Last Update 2025-06-05

Participation Criteria


Eligible Age >=15 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. Age 15 or older; 2. 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; 3. Ability to perform tests of visual and retinal function; 4. Ability to comply with research procedures; 5. Able and willing to provide informed consent before undergoing any study-related procedures. 6. 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: 1. Unwilling or unable to give consent, 2. Unable or unlikely to return for scheduled protocol visits 3. Pregnant or nursing women or unwillingness for subject with childbearing potential to use contraception during the first year of the study. 4. Optic disc drusen on exam or in previous history. 5. Ocular diseases or visual dysfunction conditions other than refractive error (e.g. amblyopia, glaucoma, etc.) in the eye selected for the injection. 6. Previous eye surgery in the eye selected for injection. 7. 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 a) Any laboratory screening test that meets the abnormality criteria stated above can be repeated once between Baseline one to Baseline 2. 8. Type I diabetes or the presence of diabetic retinopathy 9. History of neurodegenerative conditions (e.g. multiple sclerosis, neuromyelitis optica, Parkinson's disease) 10. History of autoimmune conditions (e.g. systemic lupus erythematosus) 11. Systemic diseases having ocular manifestations likely to confound assessment of study results. History of cancer within five years other than localized basal or squamous cell carcinoma not near the orbital area. Patients with a prior history of cancer will need documentation from their cancer specialist that the cancer was cured at least 5 years before study entry. 12. Allergy to pupil dilating drops or narrow angles precluding safe dilation. 13. No Light Perception (NLP) vision in either eye.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Safe but low efficacy, researchers did not advance development

Resources/Links