New eye injection aims to slow blindness in rare genetic disease
NCT ID NCT06627179
First seen Jun 24, 2026 · Last updated Jul 10, 2026 · Updated 4 times
Summary
This study tests an experimental drug called ultevursen for people with retinitis pigmentosa caused by a specific gene mutation (USH2A). The drug is injected into the eye and may help slow vision loss. The trial involves 81 participants, some of whom will receive a sham (fake) injection for comparison. The study lasts two years and measures changes in the retina and vision.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Ultevursen (given as an eye injection)
- What this could lead to
- If it works, this could slow vision loss in people with a specific genetic form of retinitis pigmentosa.
- What could go wrong
- This is an early Phase 2 trial with only 81 people. The drug is injected into the eye, which carries risks like infection or inflammation. It may not improve vision or could cause side effects.
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
Locations
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ASST Santi Paolo e Carlo Hospital, University of Milan
Milan, 20142, Italy
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Amsterdam University Medical Center - Locatie AMC
Amsterdam, 1105 AZ, Netherlands
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Bascom Palmer Eye Institute/University of Miami
Miami, Florida, 33136, United States
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Baylor College of Medicine
Houston, Texas, 77030, United States
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Casey Eye Institute, Oregon Health & Science University
Portland, Oregon, 97239, United States
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Centre de maladies rares CHNO des Quinze Vingt
Paris, 75012, France
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Emory University
Atlanta, Georgia, 30322, United States
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Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP)
São Paulo, 04021-001, Brazil
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Ghent University Hospital
Ghent, B-9000, Belgium
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Het Oogziekenhuis Rotterdam
Rotterdam, 3011 BH, Netherlands
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Hospital for Sick Children
Toronto, Ontario, M5G1E8, Canada
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Hôpital Gui de Chauliac - CHRU de Montpellier - Maladies Sensorielles Génétique
Montpellier, 34295, France
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INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte
Belo Horizonte, 30150270, Brazil
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Massachusetts Eye and Ear
Boston, Massachusetts, 02114, United States
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McGill University Health Centre for Innovative Medicine
Montreal, Quebec, H4A3J1, Canada
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Moorfields Eye Hosptial
London, EC1V 2PD, United Kingdom
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Oxford Eye Hospital
Headington, Oxford, OX3 9DU, United Kingdom
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Radboud Universitair Medisch Centrum
Nijmegen, 6525 GA, Netherlands
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Retina Foundation of the Southwest
Dallas, Texas, 75231, United States
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Rigshospitalet and University of Copenhagen
Glostrup Municipality, 2600, Denmark
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The University of California, San Francisco
San Francisco, California, 94143, United States
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University of Edinburgh / NHS Lothian
Edinburgh, EH39HA, United Kingdom
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University of Michigan- Kellogg Eye Center
Ann Arbor, Michigan, 48105, United States
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University of Pennsylvania, Scheie Eye Institute
Philadelphia, Pennsylvania, 19104, United States
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University of Wisconsin- Madison
Madison, Wisconsin, 53705, United States
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Universitätsklinikum Tübingen
Tübingen, 72076, Germany
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