Can a single injection save sight in a rare retinal disease?
NCT ID NCT06302608
First seen Aug 17, 2026 · Last updated Aug 17, 2026
Summary
This early-stage trial is testing whether a gene therapy called NGGT001, injected under the retina, is safe and tolerable for people with Bietti's crystalline dystrophy, a rare inherited condition that causes progressive vision loss. The study enrolls about 11 adults with a confirmed CYP4V2 gene mutation and reduced vision. Researchers will monitor for side effects and measure changes in visual acuity to see if the treatment might 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
- NGGT001, a gene therapy injected under the retina
- What this could lead to
- If successful, this could pave the way for a one-time treatment that slows or stops vision loss in people with Bietti's crystalline dystrophy.
- What could go wrong
- This is a very early, small safety trial, so it is unknown if the therapy will improve vision or how long any benefit might last. Gene therapy injections carry 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
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Xiuju Chen
Xiamen, Fujian, 361000, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.