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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
NCTID
NCT04671433
(View at clinicaltrials.gov)
Description
A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
(Show More)
Indication
X-Linked Retinitis Pigmentosa
Compound Name
Botaretigene sparoparvovec (AAV2/5-RPGR)
Sponsor
Janssen Research & Development, LLC
Funder Type
Industry
Status
Completed
Enrollment Count
97
Therapy Information
Target Gene/Variant
RPGR
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Subretinal
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV2/5
Editor Type
none
Dose 1
2.0E10 vg/mL
Dose 2
2.0E11 vg/mL
Dose 3
2.0E12 vg/mL
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase3
Submit Date
2020-11-05
Completion Date
2024-09-30
Last Update
2025-01-09
Participation Criteria
Eligible Age
>=3 Years
Standard Ages
Child, Adult, Older adult
Eligible Sex
ALL
Locations
No.of Trial Sites
27
Locations
Canada,Netherlands,Belgium,United States,Denmark,Italy,United Kingdom,Israel,France,Switzerland,Spain
Regulatory Information
Has US IND
True
Recent Updates
Phase 3 dosing complete, potential BLA filing in 2025
Resources/Links
Clinical Publications
(Presentation) Ph1/2 AAV5-RPGR (Botaretigene Sparoparvovec) Gene Therapy Trial in RPGR-associated X-linked Retinitis Pigmentosa (XLRP) - AAO 2022
Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-Associated X-Linked Retinitis Pigmentosa
News and Press Releases
Corporate Presentation
Preclinical Publications
(Poster) AAV-RPGRGene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa (XLRP): Human retinal organoid vector efficacy data - ARVO 2022