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Clinical Trial Report

Gene Therapy Trial Report

Summary

The Safety and Tolerability of Engensis (VM202) in Patients With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A)


NCTID NCT05361031 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Charcot-Marie-Tooth Disease 1A
Disease Ontology Term DOID:0110148
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 12
Results Posted Not Available

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 0.25mg/0.5ml (56 injections on days 0,14,90,104)
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2022-03-31
Completion Date 2021-09-09
Last Update 2022-05-04

Participation Criteria


Eligible Age 19 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations Korea, Republic of

Regulatory Information


Has US IND False
FDA Designations
Recent Updates Same product as NL003

Resources/Links


Resources/Links

No External Links Available.