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Gene Therapy Trial Browser
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
To assess the safety and tolerability of the investigational product (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
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.