Gene therapy targets second eye in rare blindness disorder
NCT ID NCT06646289
First seen Jun 27, 2026 · Last updated Jul 07, 2026 · Updated 2 times
Summary
This study tests a gene therapy called botaretigene sparoparvovec in the second eye of people with X-linked retinitis pigmentosa, a genetic condition that causes vision loss. The 24 participants previously received the therapy in one eye and are now being followed for safety and vision changes in the other eye. The goal is to see if treating both eyes is safe and may help preserve or improve sight.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- botaretigene sparoparvovec (gene therapy)
- What this could lead to
- If successful, this could improve vision in the second eye for people with X-linked retinitis pigmentosa, a genetic cause of blindness.
- What could go wrong
- This is a small, early-phase study (24 participants) focused on safety, so it may not lead to a proven treatment. Gene therapy carries risks like inflammation or vision changes.
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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Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, 02114, United States
-
Moorfields Eye Hospital
London, EC1V 2PD, United Kingdom
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University of Michigan Kellogg Eye Center
Ann Arbor, Michigan, 48105, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Gene Therapy's lasting effects on vision tracked in Long-Term study
- Gene therapy offers hope for rare Blindness-Causing eye disease
- Gene therapy offers hope for rare blindness
- Experimental gene therapy aims to halt vision loss in rare eye disease
- Gene therapy shows promise for rare blindness disease
- Gene therapy watch: 5-Year safety check for inherited blindness treatment