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	Inactive
Indication	Achromatopsia
Disease Ontology Term	DOID:0110007
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 (ACTUAL)
Results Posted	View Results

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

Eligible Age	>=3 Years
Standard Ages	Child, Adult, Older adult
Sexes Eligible for Study	ALL
Eligibility Criteria	Inclusion Criteria: * Are aged years or over * Have achromatopsia confirmed by a retinal specialist investigator 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 investigator 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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