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Gene Therapy Trial Browser
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
The purpose of this study is to proof the safety and efficacy of a single bilateral subretinal injection of rAAV.hCNGA3 in adult and minor patients with CNGA3-linked achromatopsia.
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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
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
Eligible Sex
ALL
Locations
No.of Trial Sites
1
Locations
Germany
Regulatory Information
Has US IND
Recent Updates
Resources/Links
Clinical Publications
Three-year results of phase I retinal gene therapy trial for CNGA3-mutated achromatopsia: results of a non randomised controlled trial
Safety and Vision Outcomes of Subretinal Gene Therapy Targeting Cone Photoreceptors in Achromatopsia: A Nonrandomized Controlled Trial
Development of Methodology and Study Protocol: Safety and Efficacy of a Single Subretinal Injection of rAAV.hCNGA3 in Patients with CNGA3-Linked Achromatopsia Investigated in an Exploratory Dose-Escalation Trial
Preclinical Publications
Restoration of cone vision in the CNGA3-/- mouse model of congenital complete lack of cone photoreceptor function