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

NCTID	NCT05361031 (View at clinicaltrials.gov)
Description	To assess the safety and tolerability of the investigational product Engensis (VM202) injected in the weakened lower limb muscles of CMT1A patients (Show More)
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 (ACTUAL)
Results Posted	View Results

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

No.of Trial Sites	1
Locations	South Korea