Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)

NCTID	NCT02781480 (View at clinicaltrials.gov)
Description	A clinical trial of AAV2/5 vector for patients with Defects in RPE65 (Show More)
Development Status	Active
Indication	Leber Congenital Amaurosis
Disease Ontology Term	DOID:0110016
Compound Name	Cevaretigene ritoparvovec
Compound Description	AAV2/5-RPE65P/OPTIRPRE65
Sponsor	MeiraGTx UK II Ltd
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	15 (ACTUAL)
Results Posted	View Results

Target Gene/Variant	RPE65
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Subretinal
Drug Product Type	Viral vector
Delivery System	Viral transduction
Vector Type	AAV2/5
Editor Type	none
Dose 1	1.0E11 vg/mL (n=9)
Dose 2	3.0E11 vg/mL (n=3)
Dose 3	1.0E12 vg/mL (n=3)

Eligible Age	>=3 Years
Standard Ages	Child, Adult, Older adult
Sexes Eligible for Study	ALL
Eligibility Criteria	Key Inclusion Criteria: * Aged 3 years or older * Early-onset severe retinal dystrophy consistent with RPE65 deficiency Key Exclusion Criteria: * Females who are pregnant or breastfeeding * Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months. View Inclusion and Exclusion Criteria at ClinicalTrials.gov

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

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