Gene therapy injection tested to halt blindness from rare genetic disease
NCT ID NCT02652780
Summary
This Phase 3 trial tested whether a single gene therapy injection (GS010) into the eye could improve vision in people with Leber Hereditary Optic Neuropathy (LHON), a rare genetic condition that causes rapid vision loss. The study involved 37 participants who had experienced vision loss for 6 to 12 months due to a specific genetic mutation (G11778A). Researchers compared the treated eye to an eye that received a sham (placebo) injection to see if the gene therapy helped restore vision over 96 weeks.
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Contacts and locations
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Locations
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Centre National Hospitalier d'Ophtalmologie des Quinze-Vingt
Paris, Paris, France
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Department of Neurology, University of Munich, Friedrich-Baur-Institute
Munich, 80336, Germany
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Department of Ophthalmology, Emory University School of Medicine
Atlanta, Georgia, 30322, United States
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Doheny Eye Center, University of California, Los Angeles
Los Angeles, California, United States
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IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, Ospedale Bellaria
Bologna, Bologna, Italy
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Moorfields Eye Hospital NHS Foundation Trust
London, EC1V 2PD, United Kingdom
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Neuro Ophthalmologic Associates, Wills Eye Hospital, Thomas Jefferson University
Philadelphia, Pennsylvania, 19107, United States
Conditions
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