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

Clinical Trial

NCT04671433 Active, Not recruiting

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

Phase 3

Interventional

Disease

Disease type

Hereditary retinal disease

Retinitis Pigmentosa

Patient type

Adult

Children

Inclusion / Exclusion

Opening date

04/12/2020

Closing date

20/09/2024

Inclusion criteria :

Exclusion criteria :

Funding

private

Members involved

Others investigators

Pr Bart LEROY

Representative

Belgium

Genetic Diagnostics (TWG6), Low Vision Daily Life and Patients Groups (TWG5), National Integration (TWG9), Research (TWG8), Retinal Rare Eye Diseases (WG1)

Working Group Leader

Pr Michael LARSEN

Representative

Denmark

Neuro-Ophthalmology Rare Diseases (WG2), Research (TWG8), Retinal Rare Eye Diseases (WG1)

Working Group Leader

Pr Isabelle Audo

Representative

France

Genetic Diagnostics (TWG6), Low Vision Daily Life and Patients Groups (TWG5), National Integration (TWG9), Neuro-Ophthalmology Rare Diseases (WG2), Pediatric Ophthalmology Rare Diseases (WG3), Registries & Epidemiology (TWG7), Research (TWG8), Retinal Rare Eye Diseases (WG1)

Dr Giacomo Maria Bacci

Substitute

Italy

Anterior Segment Rare Eye Diseases (WG4), Genetic Diagnostics (TWG6), Low Vision Daily Life and Patients Groups (TWG5), Pediatric Ophthalmology Rare Diseases (WG3), Retinal Rare Eye Diseases (WG1)

Pr Francesca Simonelli

Representative

Italy

Genetic Diagnostics (TWG6), National Integration (TWG9), Registries & Epidemiology (TWG7), Retinal Rare Eye Diseases (WG1)

Pr Carlo Traverso

Representative

Italy

Pr Luca Rossetti

Representative

Italy

Pr Camiel BOON

Representative

Netherlands

Anterior Segment Rare Eye Diseases (WG4), Genetic Diagnostics (TWG6), National Integration (TWG9), Pediatric Ophthalmology Rare Diseases (WG3), Registries & Epidemiology (TWG7), Research (TWG8), Retinal Rare Eye Diseases (WG1)

Working Group Leader