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)
Development Status	Active
Indication	Achromatopsia
Disease Ontology Term	DOID:0110008
Compound Name	AAV-CNGB3
Compound Alias	Entacingene turiparvovec
Compound Description	AAV8-hCARp.hCNGB3
Sponsor	MeiraGTx UK II Ltd
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	23 (ACTUAL)
Results Posted	View Results

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 (n=3)
Dose 2	0.3E12 vg/mL; 0.5mL (n=12)
Dose 3	0.6E12 vg/mL; 0.5mL (n=3)
Dose 4	1.0E12 vg/mL; 0.5mL (n=5)

Eligible Age	>=3 Years
Standard Ages	Child, Adult, Older adult
Sexes Eligible for Study	ALL
Eligibility Criteria	Inclusion Criteria: * Are aged 3 years or older * Have achromatopsia confirmed by a retinal specialist (CI or PI) Exclusion Criteria: * Are females who are pregnant or breastfeeding * Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months * Have any other condition that the CI/PI considers makes them inappropriate for entry into the trial View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites	2
Locations	United States,United Kingdom

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
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