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Clinical Trial Report

Gene Therapy Trial Report

Summary

Safety and Efficacy of rAAV.hCNGA3 Gene Therapy in Patients With CNGA3-linked Achromatopsia

NCTID NCT02610582 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Achromatopsia
Disease Ontology Term DOID:110007
Compound Name RAAV.hCNGA3
Compound Description AAV8-hArr3-hCNGA3-WPREm
Sponsor STZ eyetrial
Funder Type Other
Recruitment Status
Active not recruiting
Enrollment Count 13
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 AAV8
Editor Type none
Dose 1 1E11 vg/dose
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2015-09-18
Completion Date 2027-06
Last Update 2024-04-18

Participation Criteria

Eligible Age >=6 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 1
Locations Germany

Regulatory Information

Has US IND
FDA Designations
Recent Updates

Resources/Links

Clinical Publications
Preclinical Publications