Gene therapy offers hope for rare blindness
NCT ID NCT04671433
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This Phase 3 trial tests a gene therapy called botaretigene sparoparvovec for X-linked retinitis pigmentosa, a genetic condition that causes progressive vision loss. The study involves 105 participants who receive the therapy via injection under the retina. The main goal is to see if it improves their ability to navigate a maze after one year.
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 (AAV5-RPGR gene therapy)
- What this could lead to
- If successful, this gene therapy could slow or partially reverse vision loss in people with X-linked retinitis pigmentosa, potentially preserving independence.
- What could go wrong
- This is an early-stage Phase 3 trial with only 105 participants. The treatment involves eye surgery and may not work for everyone. Long-term safety and effectiveness are still unknown.
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 X-LINKED RETINITIS PIGMENTOSA 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
-
Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli
Naples, 80131, Italy
-
Azienda Ospedaliero Universitaria Careggi
Florence, 50134, Italy
-
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
Paris, 75012, France
-
Childrens Hospital
Los Angeles, California, 90027, United States
-
Duke Eye Center
Durham, North Carolina, 27705, United States
-
Emory University
Atlanta, Georgia, 30322, United States
-
Gartnavel General Hospital
Glasgow, G12 0YN, United Kingdom
-
Hadassah Medical Center
Jerusalem, 91120, Israel
-
Hosp Univ Fund Jimenez Diaz
Madrid, 28040, Spain
-
Hospital For Sick Children
Toronto, Ontario, M5G 1X8, Canada
-
IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS
Roma, 00198, Italy
-
Massachusetts General Hospital - Center for Celiac Research and Treatment
Boston, Massachusetts, 02114, United States
-
Moorfields Eye Hospital
London, EC1V 2PD, United Kingdom
-
NHS Lothian
Edinburgh, EH3 9HA, United Kingdom
-
Ospedale San Paolo
Milan, 20142, Italy
-
Radboudumc
Nijmegen, 6525EX, Netherlands
-
Retina Consultants of Houston
Bellaire, Texas, 77401, United States
-
Rigshospitalet Glostrup
Glostrup Municipality, 2600, Denmark
-
Shiley Eye Institute Jacobs Retina Center
La Jolla, California, 92093 0946, United States
-
St James University Hospital
Leeds, LS9 7TF, United Kingdom
-
Stanford Health Care
Palo Alto, California, 94303, United States
-
UZ Gent
Ghent, 9000, Belgium
-
Univ of Michigan Medical Center
Ann Arbor, Michigan, 48105, United States
-
Universite de Lausanne, Hopital ophtalmique Jules-Gonin
Lausanne, 1004, Switzerland
-
University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical
Basel, 4031, Switzerland
-
University of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, 15213, United States
-
VUMC Amsterdam
Amsterdam, 1105AZ, Netherlands
-
VitreoRetinal Associates, PA
Gainesville, Florida, 32607, 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 targets second eye in rare blindness disorder
- 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