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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Therapy Information
Study Record Dates
Participation Criteria
Locations
Regulatory Information
Resources/Links
Summary
Lentiviral Gene Therapy for Epilepsy
NCTID
NCT04601974
(View at clinicaltrials.gov)
Description
This is a phase I/IIa clinical trial investigating the safety of a lentiviral epilepsy gene therapy using an engineered potassium channel in patients with refractory epilepsy.
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Development Status
Active
Indication
Drug Resistant Epilepsy
Disease Ontology Term
DOID:1826
Compound Name
Lenti-CAMK2A-KCNA1
Sponsor
University College, London
Funder Type
Other
Recruitment Status
Not yet recruiting
Enrollment Count
10
Results Posted
Not Available
Therapy Information
Target Gene/Variant
KCNA1
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Overexpression of protective allele/gene
Route of Administration
Intracranial
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
LV
Editor Type
none
Dose 1
Undisclosed dose (single administration)
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2020-10-06
Completion Date
2032-09
Last Update
2023-05-15
Participation Criteria
Eligible Age
>=18 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
Locations
Regulatory Information
Has US IND
False
FDA Designations
Recent Updates
Resources/Links
Preclinical Publications
Epilepsy Gene Therapy Using an Engineered Potassium Channel
Optogenetic and potassium channel gene therapy in a rodent model of focal neocortical epilepsy