Clinical Trial
NCT04794101 Active, Not recruiting
Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
Disease
Disease type
Hereditary retinal disease
Retinitis Pigmentosa
Orphan drug recognition
Yes
Patient type
Adult
Children
Inclusion / Exclusion
Funding
Industry
Members involved
Main investigators
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)
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 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
Pr Carel HOYNG
Representative
Netherlands
Genetic Diagnostics (TWG6), Registries & Epidemiology (TWG7), Research (TWG8), Retinal Rare Eye Diseases (WG1)
ERN EYE member investigating site
HCP : Principal investigators
Ghent University Hospital, Ghent, Belgium
Adress
Corneel Heymanslaan 10
9000 Ghent
Belgium
Rigshospitalet, Copenhagen, Denmark
Adress
Inge Lehmanns Vej 7 Entrance 2, 6. floor Section 2061
2100 Copenhagen
Denmark
RÉFÉRET National Hospital of Ophthalmology of Quinze-Vingts, Paris, France
Adress
28 Rue de Charenton
75012 Paris
France
AOU Careggi – AOU Meyer Consortium, Firenze, Italy
Adress
Largo Brambilla 3
50134 Florence
Italy
ASST Santi Paolo e Carlo, Milan, Italy
Adress
Via Antonio di Rudinì, n. 8
20142 Milano MI
Italy
AOU Università degli Studi della Campania Luigi Vanvitelli, Napoli, Italy
Adress
Via Santa Maria di Costantinopoli
104, 80138 Napoli NA
Italy
Clinica Oculistica – Ospedale Policlinico San Martino IRCCS, Genova, Italy
Adress
Viale Benedetto XV, 7
16132 Genova GE
Italy
Leiden University Medical Center/Academic Medical Center Amsterdam, Netherlands
Adress
Albinusdreef 2
2333 ZA Leiden
Netherlands
Radboud university medical center, Nijmegen, Netherlands
Adress
Geert Grooteplein Zuid 10
6525 GA Nijmegen
Netherlands