Phase
Condition
Macular Degeneration
Retina
Myopic Macular Degeneration
Treatment
AAV8.hLCA5
Clinical Study ID
Ages > 13 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Are willing and able to provide written informed consent (ICF) and, whereappropriate, willing to sign an assent prior to any study procedures.
Are willing to adhere to the clinical protocol and able to perform testingprocedures.
Participants must be at least 13 years of age at consent.
Carry disease-causing biallelic LCA5 gene mutations determined by a ClinicalLaboratory Improvement Amendments (CLIA) certified laboratory.
Visual acuity: BCVA < 20/80 on the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity chart (modified for low vision participants) in the eye to betreated
Show evidence of detectable photoreceptors by Spectral Domain Optical CoherenceTomography (SD-OCT)
Participant is a good candidate for surgery per investigator judgement
Participant agrees to follow direction of investigator regarding restrictionspost-surgery.
Exclusion
Exclusion Criteria:
Individuals of childbearing potential (male and female) who are pregnant orunwilling to use effective contraception for the duration of the study, includingbarrier methods for the first year after investigational product (IP)administration.
Pre-existing eye conditions or complicating systemic diseases that would precludethe planned surgery. This includes individuals who are immunocompromised.
History of intraocular surgery for either eye within 6 months prior to planned IPadministration.
Have previously received gene therapy.
Have used any investigational drug or device within 90 days or 5 estimatedhalf-lives of treatment, whichever is longer or plan to participate in another studyof drug or device during the study period.
History of disease which may preclude the participant from participation, or whichmay interfere with outcome measure testing or test results.
Incapable of performing visual function testing (e.g., FST testing) for reasonsother than poor vision.
Any absolute contraindication to a course of oral steroids.
Any other condition that would not allow the potential participant to completefollow-up examinations during the study and, in the opinion of the Investigator,makes the potential participant unsuitable for the study.
Study Design
Study Description
Connect with a study center
University of Pennsylvania Perelman School of Medicine
Philadelphia, Pennsylvania 19104
United StatesActive - Recruiting
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