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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
Indication Achromatopsia
Compound Name RAAV.hCNGA3 (AAV8-hArr3-hCNGA3-WPREm)
Sponsor STZ eyetrial
Funder Type Other
Status
Active not recruiting
Enrollment Count 13

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
Dose 1  1x10^11 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
Eligible Sex ALL

Locations

No.of Trial Sites 1
Locations Germany

Regulatory Information

Has US IND
Recent Updates

Resources/Links

Patents
  • US20180353619
  • WO2017144080A1
  • WO2016146669A1
  • WO2018010965A1
Clinical Publications
Preclinical Publications