One-Shot eye injection aims to halt rare genetic vision loss
NCT ID NCT07185256
Summary
This early-stage study is testing a one-time gene therapy injection for two rare, inherited eye diseases that cause progressive vision loss: Best vitelliform macular dystrophy (BVMD) and Autosomal-Recessive Bestrophinopathy (ARB). Researchers will give the experimental drug, OPGx-BEST1, to 10 adult participants to first check if it's safe and to find the best dose. The main goal is to see if a single injection into the eye can help control the disease over five years of follow-up.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Children's Hospital Los Angeles
RECRUITINGLos Angeles, California, 90027, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Cincinnati Eye Institute
RECRUITINGCincinnati, Ohio, 45242, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Retina Foundation of the Southwest
RECRUITINGDallas, Texas, 75231, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Vitreo Retinal Associates
NOT_YET_RECRUITINGGainesville, Florida, 34481, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
Explore the condition pages connected to this study.