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Clinical Trial Report

Gene Therapy Trial Report

Summary

PDE6A Gene Therapy for Retinitis Pigmentosa


NCTID NCT04611503 (View at clinicaltrials.gov)
Description
Indication Retinitis Pigmentosa
Compound Name RAAV.hPDE6A
Sponsor STZ eyetrial
Funder Type Other
Status
Active not recruiting
Enrollment Count 9

Therapy Information


Target Gene/Variant PDE6A
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 AAV8
Editor Type
Dose 1 1 x 10^9 vg
Dose 2 5 x 10^9 vg
Dose 3 1 x 10^10 vg
Dose 4 5 x 10^10 vg
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2020-05-20
Completion Date 2027-07
Last Update 2024-04-18

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Eligible Sex ALL

Locations


No.of Trial Sites 1
Locations Germany

Regulatory Information


Has US IND False
Recent Updates

Resources/Links