Experimental gene therapy aims to halt vision loss in rare eye disease
NCT ID NCT05926583
First seen Jun 26, 2026 · Last updated Jul 07, 2026 · Updated 3 times
Summary
This Phase 3 study tests a gene therapy called AAV5-hRKp.RPGR in 4 Japanese people with X-linked retinitis pigmentosa, a genetic condition that causes progressive vision loss. The therapy is injected into the eye to deliver a working copy of the RPGR gene. The main goal is to check safety, but researchers will also measure changes in vision over a year.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- AAV5-hRKp.RPGR (a gene therapy injected into the eye)
- What this could lead to
- If successful, this could lead to a treatment that slows or stops vision loss in people with X-linked retinitis pigmentosa.
- What could go wrong
- This is a very small early-phase study with only 4 participants, so results may not apply to everyone. Gene therapy carries risks like inflammation or retinal damage.
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
-
National Hospital Organization Tokyo Medical Center
Meguro-ku, 1528902, Japan
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- A sharper look at the retina: new imaging technology may spot eye disease earlier
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- Gene Therapy's lasting effects on vision tracked in Long-Term study
- Could stem cells restore sight in damaged eyes?
- Gene therapy injection aims to restore sight in rare blindness