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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Gene Therapy for Achromatopsia (CNGA3)
NCTID
NCT03758404
(View at clinicaltrials.gov)
Description
A clinical trial of adeno-associated virus vector (AAV) CNGA3 retinal gene therapy for patients with achromatopsia
(Show More)
Development Status
Active
Indication
Achromatopsia
Disease Ontology Term
DOID:110007
Compound Name
AAV-CNGA3
Compound Alias
Aguracingene cadoparvovec
Compound Description
AAV8-hG1.7p.co.CNGA3
Sponsor
MeiraGTx UK II Ltd
Funder Type
Industry
Recruitment Status
Completed
Enrollment Count
11
Results Posted
View Results
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
AAV2/8
Editor Type
none
Dose 1
3 undisclosed dose levels
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2018-11-27
Completion Date
2021-06-10
Last Update
2022-12-01
Participation Criteria
Eligible Age
>=3 Years
Standard Ages
Child, Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
2
Locations
United States,United Kingdom
Regulatory Information
Has US IND
True
FDA Designations
Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates
Resources/Links
Clinical Publications
A demonstration of cone function plasticity after gene therapy in achromatopsia
News and Press Releases
Pipeline - MeiraGTx
Preclinical Publications
(Poster Presentation) Development and efficacy assessment of AAV2/8-hG1.7p.coCNGA3, a CNGA3gene therapy vector - ARVO 2019
Protocol
Statistical Analysis Plan
Clinical Trial Protocol
Related NCTID
Long Term Follow-Up: NCT03278873