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Clinical Trial Report

Gene Therapy Trial Report

Summary

A 6-Month Extension Study to Assess the Long-Term Safety of Engensis in Amyotrophic Lateral Sclerosis


NCTID NCT05176093 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Amyotrophic Lateral Sclerosis (ALS)
Disease Ontology Term DOID:332
Compound Name ENGENSIS
Compound Alias donaperminogene seltoplasmid, VM202
Compound Description pCK-HGF-X7 (HGF 728, HGF723 isoforms)
Sponsor Helixmith Co., Ltd.
Funder Type Industry
Recruitment Status
Completed
Enrollment Count 8 (ACTUAL)
Results Posted View Results

Therapy Information


Target Gene/Variant HGF
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intramuscular
Drug Product Type Plasmid
Target Tissue/Cell
Delivery System None (naked plasmid)
Vector Type
Editor Type
Dose 1 64mg/cycle, 3 cycles/patient, 2 days/cycle, 128 injections/cycle
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase2
Submit Date 2021-11-05
Completion Date 2024-08-23
Last Update 2025-10-06

Participation Criteria


Eligible Age 18 Years - 80 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 2
Locations South Korea,United States

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation
Recent Updates Same product as NL003

Resources/Links