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Clinical Trial Report

Gene Therapy Trial Report

Summary

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

NCTID NCT02781480 (View at clinicaltrials.gov)
Description
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
Results Posted View Results

Therapy Information

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
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV2/5
Editor Type none
Dose 1 1.0E11 vg/mL
Dose 2 3.0E11 vg/mL
Dose 3 1.0E12 vg/mL
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2016-04-28
Completion Date 2018-12
Last Update 2021-07-12

Participation Criteria

Eligible Age >=3 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL

Locations

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

Regulatory Information

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

Resources/Links

Preclinical Publications
Protocol