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

Study title: 
Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene
Long title: 
Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene
Date receipt dossier: 
28 jan 2022
EU record number: 
B/BE/21/BVW8
EudraCT number: 
2020-002873-88
Company / Sponsor: 
Meira GTx UK II Limited
Phase: 
III
Treated organism: 
Humans
Indication category: 
Genetic disorders
Disease: 
X-linked Retinitis Pigmentosa associated with Variants in the RPGR gene
Therapeutic approach: 
Gene therapy
Genetic modification: 
Non-replicating recombinant vector derived from adeno-associated virus AAV 5 carrying the human RPGR gene
Method of transfer of nucleic acid of interest: 
Non-replicating recombinant vector derived from adeno-associated virus AAV 5
Administered biological material: 
Genetically modified virus
Route of administration: 
Subretinal
Locations in Belgium: 
UZ Gent
Nr of subjects: 
Up to 60 patients overall and 10 patients in Belgium
Foreseen duration: 
Start April 2022 with long-term safety follow-up of patients
Type of procedure: 
Contained use and Deliberate release
Current status: 
Authorized

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