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

PHASE3CompletedINTERVENTIONAL
Enrollment

97

Participants

Timeline

Start Date

December 4, 2020

Primary Completion Date

September 30, 2024

Study Completion Date

September 30, 2024

Conditions
X-Linked Retinitis Pigmentosa
Interventions
BIOLOGICAL

Genetic: AAV5-hRKp.RPGR

Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group

BIOLOGICAL

Genetic: AAV5-hRKp.RPGR

No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study)

Trial Locations (27)

1004

Universite de Lausanne, Hopital ophtalmique Jules-Gonin, Lausanne

2600

Rigshospitalet Glostrup, Glostrup Municipality

4031

University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical, Basel

9000

UZ Gent, Ghent

15213

University of Pittsburgh Medical Center (UPMC), Pittsburgh

20142

Ospedale San Paolo, Milan

27705

Duke Eye Center, Durham

28040

Hosp Univ Fund Jimenez Diaz, Madrid

30322

Emory University, Atlanta

32607

VitreoRetinal Associates, PA, Gainesville

48105

Univ of Michigan Medical Center, Ann Arbor

50134

Azienda Ospedaliero Universitaria Careggi, Florence

75012

Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts, Paris

80131

Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli, Napoli

90027

Childrens Hospital, Los Angeles

91120

Hadassah Medical Center, Jerusalem

94303

Stanford Health Care, Palo Alto

92093 0946

Shiley Eye Institute Jacobs Retina Center, La Jolla

02114

Massachusetts General Hospital - Center for Celiac Research and Treatment, Boston

M5G 1X8

Hospital For Sick Children, Toronto

00198

IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS, Roma

1105AZ

VUMC Amsterdam, Amsterdam

6525EX

Radboudumc, Nijmegen

EH3 9HA

NHS Lothian, Edinburgh

G12 0YN

Gartnavel General Hospital, Glasgow

LS9 7TF

St James University Hospital, Leeds

EC1V 2PD

Moorfields Eye Hospital, London

All Listed Sponsors
lead

Janssen Research & Development, LLC

INDUSTRY

NCT04671433 - Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene | Biotech Hunter | Biotech Hunter