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Gene Therapy Trial Report

Summary

PDE6A Gene Therapy for Retinitis Pigmentosa


NCTID NCT04611503 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Retinitis Pigmentosa
Disease Ontology Term DOID:0110379
Compound Name RAAV.hPDE6A
Sponsor STZ eyetrial
Funder Type Other
Recruitment Status
Active not recruiting
Enrollment Count 9 (ACTUAL)
Results Posted Not Available

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 none
Dose 1 1E9 vg
Dose 2 5E9 vg
Dose 3 1E10 vg
Dose 4 5E10 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
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations Germany

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links