Gene Therapy's lasting effects on vision tracked in Long-Term study
NCT ID NCT03584165
First seen Jul 06, 2026 · Last updated Jul 07, 2026 · Updated 1 time
Summary
This study follows people with two inherited eye conditions—choroideremia and X-linked retinitis pigmentosa—who previously received gene therapy. Researchers monitor their safety and vision over several years to see if the treatment remains safe and helps preserve sight. Participants include both those who got the gene therapy and untreated controls from earlier studies.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- gene therapy (BIIB111 or BIIB112)
- What this could lead to
- If successful, this could show that a single gene therapy treatment provides lasting safety and helps preserve vision for years in people with these inherited eye diseases.
- What could go wrong
- This is a follow-up study, not a new treatment test. It cannot prove the therapy works for everyone, and long-term side effects or loss of vision are still possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
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Locations
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CHU Montpellier - Saint Eloi
Montpellier, 34295, France
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Centre Hospitalier National d Ophtalmologie (CHNO) des Quinze-Vingts
Paris, 75012, France
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Cincinnati Eye Institute - Blue Ash
Cincinnati, Ohio, 45242-5664, United States
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Columbia University Medical Center
New York, New York, 10032, United States
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Helsinki University Central Hospital (HUCH)
Helsinki, 00290 HUS, Finland
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Instituto Genetica Ocular
São Paulo, 04039, Brazil
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John Radcliffe Hospital
Oxford, OX3 9DU, United Kingdom
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Johns Hopkins Hospital
Baltimore, Maryland, 21287-0005, United States
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MEEI Massachusets Eye and Ear Infirmary
Boston, Massachusetts, 02114-3002, United States
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Manchester Royal Eye Hopsital
Manchester, M13 9WL, United Kingdom
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McGill University Health Centre
Montreal, Quebec, H4A 3JI, Canada
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Moorfields Eye Hospital
London, EC1V 2PD, United Kingdom
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OHSU - Casey Eye Institute
Portland, Oregon, 97239, United States
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Radboudumc
Nijmegen, 6525 GA, Netherlands
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Retina Foundation of the Southwest
Dallas, Texas, 75231-5080, United States
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Rigshospitalet-Glostrup, Oejenafdelingen
Glostrup Municipality, 2600, Denmark
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Southampton General Hospital
Southampton, SO16 6YD, United Kingdom
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The Northern Alberta Clinical Trials and Research Centre
Edmonton, Alberta, T5H 3V9, Canada
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The University of British Columbia - Eye Care Centre
Vancouver, British Columbia, V5Z 39N, Canada
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UCLA - Jules Stein Eye Institute
Los Angeles, California, 90095-7065, United States
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Universitats Klinikum Tubingen - Institute for Ophthalmic Research
Tübingen, 72076, Germany
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University of Miami
Miami, Florida, 33136, United States
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University of Wisconsin School of Medicine
Madison, Wisconsin, 53705-3644, United States
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Universitäts-Augenklinik Bonn
Bonn, 53127, Germany
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Vitreo Retinal Associates PA - The Millennium Center
Gainesville, Florida, 32607, United States
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