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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Gene Therapy for Achromatopsia (CNGB3)
NCTID
NCT03001310
(View at clinicaltrials.gov)
Description
A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia
(Show More)
Indication
Achromatopsia
Compound Name
Entacingene turiparvovec (AAV8-hCARp.hCNGB3)
Sponsor
MeiraGTx UK II Ltd
Funder Type
Industry
Status
Completed
Enrollment Count
23
Therapy Information
Target Gene/Variant
CNGB3
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
0.1E12 vg/mL; 0.5 mL
Dose 2
0.3E12 vg/mL; 0.5mL
Dose 3
0.6E12 vg/mL; 0.5mL
Dose 4
1.0E12 vg/mL; 0.5mL
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2016-11-22
Completion Date
2019-10-25
Last Update
2023-03-08
Participation Criteria
Eligible Age
>=3 Years
Standard Ages
Child, Adult, Older adult
Eligible Sex
ALL
Locations
No.of Trial Sites
2
Locations
United States,United Kingdom
Regulatory Information
Has US IND
True
Recent Updates
Resources/Links
Clinical Publications
First-in-Human Gene Therapy Trial of AAV8-hCARp.hCNGB3 in Adults and Children With CNGB3-associated Achromatopsia
A demonstration of cone function plasticity after gene therapy in achromatopsia
News and Press Releases
https://meiragtx.com/research-development/pipeline/
Preclinical Publications
Gene therapy rescues cone function in congenital achromatopsia
Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy
(Poster) Efficacy assessment and pre-clinical toxicology of AAV2/8-hCARp.hCNGB3, a CNGB3 gene therapy vector - ESGCT 2017
Protocol
Statistical Analysis Plan
Clinical Trial Protocol