Can a single injection restore sight in a rare genetic eye disease?
NCT ID NCT06706427
First seen Aug 12, 2026 · Last updated Aug 13, 2026 · Updated 1 time
Summary
This trial tests an experimental gene therapy called NGGT001 for people with Bietti Crystalline Dystrophy, a rare inherited condition that causes progressive vision loss. The therapy is delivered as a single injection under the retina, aiming to replace a faulty gene with a working copy. The study will evaluate the safety and tolerability of the treatment and look for early signs that it can improve or stabilize vision.
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 using a modified virus to deliver a working copy of the CYP4V2 gene into the retina via subretinal injection.
- What this could lead to
- If successful, this could lead to a one-time treatment that slows or stops vision loss in people with Bietti Crystalline Dystrophy, potentially preserving sight.
- What could go wrong
- This is an early-stage trial with only 12 participants, so results may not apply broadly. Risks include potential side effects from the injection or the viral vector, and the therapy may not improve vision.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for BIETTI CRYSTALLINE CORNEORETINAL DYSTROPHY are added.
By submitting, you agree to our Terms of use
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
-
Southwest Hospital/Southwest Eye Hospital, Third Military Medical University (Army Medical University)
Chongqing, Chongqing Municipality, 400000, China
-
Xiamen Eye Center of Xiamen University
Xiamen, Fujian, 361000, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.