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
Drug Product Name	AAV-CNGA3
Drug Product Alias	Aguracingene cadoparvovec
Drug Product 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
Regulatory Element	G1.7 (promoter)
Therapeutic Modality	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
Gene Delivery System Type	Viral transduction
Vector Type 🔄	Adeno-associated virus
Viral Vector Subtype	AAV2/8
Dose 1 🔄	Undisclosed dose escalation, 3 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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