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Clinical Trial Report

Gene Therapy Trial Report

Summary

Trial of Subretinal Injection of (rAAV2-VMD2-hMERTK)

NCTID NCT01482195 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Retinal Disease, Retinitis Pigmentosa
Disease Ontology Term DOID:0110367
Compound Name AAV2-VMD2-hMERTK
Sponsor King Khaled Eye Specialist Hospital
Funder Type Other gov
Recruitment Status
Completed
Enrollment Count 6
Results Posted View Results

Therapy Information

Target Gene/Variant MERTK
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
Editor Type
Dose 1 5.96E10 vg/150ul (n=2)
Dose 2 1.788E11 vg/450ul (n=4)
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1
Submit Date 2011-09-28
Completion Date 2019-08
Last Update 2022-01-26

Participation Criteria

Eligible Age 14 Years - 70 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 1
Locations Saudi Arabia

Regulatory Information

Has US IND
FDA Designations
Recent Updates Therapy was well tolerated, 3/6 patients displayed improved visual acuity, but the effects persisted less than 2 years in 2 of the 3

Resources/Links

Clinical Publications
Preclinical Publications