Can a One-Time gene therapy preserve sight for years? a 15-Year study aims to find out
NCT ID NCT03602820
First seen Jul 24, 2026 · Last updated Aug 20, 2026 · Updated 2 times
Summary
This study follows people who previously received a one-time gene therapy injection for a rare inherited eye disease caused by RPE65 mutations. Researchers will test vision and light sensitivity over up to 15 years to see how well the treatment holds up. The goal is to understand the long-term safety and durability of this approach.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- a gene therapy called voretigene neparvovec-rzyl (AAV2-hRPE65v2) given as a one-time injection under the retina
- What this could lead to
- If vision remains stable over many years, this could show that a single gene therapy can provide lasting benefit for people with this rare inherited eye disease.
- What could go wrong
- This is a long-term follow-up study, not a new treatment trial. It cannot prove the therapy works better than other options, and vision may still decline over time.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
-
41 people
The number who actually took part.
- Started
-
Jun 2015
- Expected to finish
-
Jan 2030
An estimate. End dates often move.
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
Individuals who received the subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl) in the Phase 1 or Phase 3 clinical trials
- Ages
-
Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1\. Subjects who participated in prior subretinal AAV2-hRPE65v2 gene therapy clinical studies Exclusion Criteria: 1. Subjects who will not consent for study. 2. Subjects who the investigators believe are not capable of performing study assessments
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Inherited retinal dystrophy due to RPE65 mutations 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.