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Clinical Trials in Denmark / NCT04794101
Active, not recruiting Phase 3

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

NCT04794101 · tracked via the Priya Life Science Denmark tracker
Sponsor
MeiraGTx Ocular UK Ltd
Phase
Phase 3
Started
2020-12-04
Last updated
2026-09-25

Condition(s) studied

X-Linked Retinitis Pigmentosa

Investigational drug(s) / intervention(s)

Genetic: AAV5-hRKp.RPGR Intermediate DoseGenetic: AAV5-hRKp.RPGR Low Dose

Genetic: AAV5-hRKp.RPGR Intermediate Dose: Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group

Genetic: AAV5-hRKp.RPGR Low Dose: Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group

Study summary

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

Eligibility

Sex
ALL
Min age
3 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria: * 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. Exclusion Criteria: * None

Primary outcome measure(s)

  • Number of Participants with Ocular and Non-ocular Adverse Events — Day 1 - Month 60
    Number of participants with ocular and non-ocular adverse events will be assessed.
  • Number of Participants With Abnormalities in Laboratory Assessments — Day 1 - Month 60
    Number of participants with abnormalities in laboratory assessments will be assessed.
  • Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment — From Baseline - Month 60
    Change from baseline in BCVA by ETDRS chart letter score in monocular assessment will be assessed.
  • Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Monocular Assessment — From Baseline- Month 60
    Change from baseline in low luminance visual acuity by ETDRS chart letter score in monocular assessment will be assessed.

Trial sites (28)

FacilityCityRegionStatus
Shiley Eye Institute Jacobs Retina Center La Jolla California
Childrens Hospital Los Angeles Los Angeles California
Stanford Health Care Palo Alto California
VitreoRetinal Associates, PA Gainesville Florida
Emory University Atlanta Georgia
Massachusetts Eye and Ear Infirmary Boston Massachusetts
Univ of Michigan Medical Center Ann Arbor Michigan
Duke Eye Center Durham North Carolina
University of Pittsburgh Medical Center (UPMC) Pittsburgh Pennsylvania
Retina Consultants of Houston Bellaire Texas
Ghent University Hospital Ghent Belgium
Hospital For Sick Children Toronto Ontario
Rigshospitalet Glostrup Glostrup Municipality Denmark
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts Paris France
Hadassah Medical Center Jerusalem Israel
Azienda Ospedaliero Universitaria Careggi Florence Italy
Ospedale San Paolo Milan Italy
Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli Naples Italy
IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS Rome Italy
VUMC Amsterdam Amsterdam Netherlands
Radboudumc Nijmegen Netherlands
Hosp Univ Fund Jimenez Diaz Madrid Spain
University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical Basel Switzerland
Universite de Lausanne, Hopital ophtalmique Jules-Gonin Lausanne Switzerland
NHS Lothian Edinburgh United Kingdom
Gartnavel General Hospital Glasgow United Kingdom
St James University Hospital Leeds United Kingdom
Moorfields Eye Hospital London United Kingdom
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04794101 on ClinicalTrials.gov ↗ ← All trials in Denmark