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Clinical Trial Report

Gene Therapy Trial Report

Summary

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations


NCTID NCT05820152 (View at clinicaltrials.gov)
Description
Indication Leber Hereditary Optic Neuropathy (LHON)
Compound Name NFS-02
Sponsor Neurophth Therapeutics Inc
Funder Type Other
Status
Terminated
Enrollment Count 11

Therapy Information


Target Gene/Variant ND1G3460A
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravitreal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV2
Editor Type none
Dose 1 5.0E7 vg, 0.05mL/eye
Dose 2 Starting dose: 1.5E8 vg, 0.05mL/eye
Dose 3 5.0E8 vg, 0.05mL/eye
Dose 4 1.5E9 vg, 0.05mL/eye
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2023-04-06
Completion Date 2024-06-24
Last Update 2024-09-20

Participation Criteria


Eligible Age 18 Years - 75 Years
Standard Ages Adult, Older adult
Eligible Sex ALL

Locations


No.of Trial Sites 4
Locations United States,China

Regulatory Information


Has US IND True
Recent Updates Study terminated due to sponsor circumstances and external reasons

Resources/Links