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Clinical Trial Report

Gene Therapy Trial Report

Summary

Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial)


NCTID NCT02935517 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Achromatopsia
Disease Ontology Term DOID:0110007
Compound Name AGTC-402
Compound Description rAAV2tYF-PR1.7-hCNGA3
Sponsor Beacon Therapeutics
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 24
Results Posted Not Available

Therapy Information


Target Gene/Variant CNGA3
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 Cone cells
Delivery System Viral transduction
Vector Type AAV2tYF
Editor Type none
Dose 1 4.0E10 vg/mL
Dose 2 1.2E11 vg/mL
Dose 3 3.6E11 vg/mL
Dose 4 1.1E12 vg/mL
Dose 5 3.2E12 vg/mL

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2016-10-13
Completion Date 2026-08
Last Update 2022-07-22

Participation Criteria


Eligible Age >=4 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 6
Locations United States,Israel

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation
Recent Updates Development discontinued by Sponsor

Resources/Links