Contact SCGE
Your email
Message
Send
SCGE Consortium Home
About SCGE TCDC
Contact Us
License
Home
Gene Therapy Trial Browser
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
This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-402, administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGA3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy.
(Show More)
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
News and Press Releases
SEC Form 10-K: AGTC Annual Report for FY 2022
Orphan Drug Designation
Beacon Completed Clinical Trials
Preclinical Publications
Safety and Efficacy Evaluation of rAAV2tYF-PR1.7-hCNGA3 Vector Delivered by Subretinal Injection in CNGA3 Mutant Achromatopsia Sheep
Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia