Gene therapy shows promise for rare eye disease causing blindness
NCT ID NCT02652780
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a one-time gene therapy injection, GS010, in 37 adults with Leber Hereditary Optic Neuropathy (LHON), a genetic condition that causes rapid vision loss. Participants had vision loss for 6 to 12 months before treatment. The goal was to see if the therapy could improve eyesight compared to a sham injection. Results measured changes in visual acuity over 48 and 96 weeks.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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
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Other studies related to the condition(s) this trial covers.