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NCT04671433CompletedPhase 3

Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Source: ClinicalTrials.gov · Last updated 24 Sept 2026

Brief summary (registry, in English)

A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)

Status (registry)
Completed
Phase (registry)
Phase 3
Study type
Interventional
Enrollment
105 (actual)
First submitted
5 Nov 2020
Start date
4 Dec 2020
Primary completion date
30 Sept 2024
Lead sponsor
MeiraGTx Ocular UK Ltd
NCT number
NCT04671433
EudraCT number
2020-002873-88

What you commit to

  • Study type: Interventional · Phase 3
  • Participants enrolled: 105
  • From 4 Dec 2020 to the primary completion date, 30 Sept 2024
  • 28 sites in 11 countries
  • Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Prior treatment

  • Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the study intervention administration
  • Has undergone prior retinal surgery involving the macula, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery

Other

  • Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule

Who the study is looking for

  • Male or female
  • 3 years of age or older
  • Has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory

Age in the registry: 3 years to - · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (28)

Sites in registry order. Save your city in your profile to sort them by distance.

SiteCityCountrySite status
UZ Gent
Coordinator of this site? Confirm its status
GhentBelgiumUnknown status
Hospital for Sick Children
Coordinator of this site? Confirm its status
TorontoCanadaUnknown status
University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical
Coordinator of this site? Confirm its status
BaselSwitzerlandUnknown status
Universite de Lausanne, Hopital ophtalmique Jules-Gonin
Coordinator of this site? Confirm its status
LausanneSwitzerlandUnknown status
Rigshospitalet Glostrup
Coordinator of this site? Confirm its status
Glostrup MunicipalityDenmarkUnknown status
Hosp Univ Fund Jimenez Diaz
Coordinator of this site? Confirm its status
MadridSpainUnknown status
Centre Hospitalier National D'ophtalmologie Des Quinze Vingts
Coordinator of this site? Confirm its status
ParisFranceUnknown status
NHS Lothian
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EdinburghUnited KingdomUnknown status
Gartnavel General Hospital
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GlasgowUnited KingdomUnknown status
St James University Hospital
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LeedsUnited KingdomUnknown status
Moorfields Eye Hospital
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LondonUnited KingdomUnknown status
Hadassah medical Center
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JerusalemIsraelUnknown status
Azienda Ospedaliero-Universitaria Careggi
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FlorenceItalyUnknown status
Ospedale San Paolo
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MilanItalyUnknown status
Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli
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NaplesItalyUnknown status
IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS
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RomaItalyUnknown status
VUMC Amsterdam
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AmsterdamNetherlandsUnknown status
Radboudumc
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NijmegenNetherlandsUnknown status
Univ of Michigan Medical Center
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Ann ArborUnited StatesUnknown status
Emory University
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AtlantaUnited StatesUnknown status
Retina Consultants of Houston
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BellaireUnited StatesUnknown status
Massachusetts General Hospital - Center for Celiac Research and Treatment
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BostonUnited StatesUnknown status
Duke Eye Center
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DurhamUnited StatesUnknown status
VitreoRetinal Associates, PA
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GainesvilleUnited StatesUnknown status
Shiley Eye Institute Jacobs Retina Center
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La JollaUnited StatesUnknown status
Childrens Hospital
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Los AngelesUnited StatesUnknown status
Stanford Health Care
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Palo AltoUnited StatesUnknown status
University of Pittsburgh Medical Center (UPMC)
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PittsburghUnited StatesUnknown status

This page is information, not medical advice. Discuss any trial with your doctor.