A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children with SCN1A-Positive Dravet Syndrome

NCTID	NCT05419492 (View at clinicaltrials.gov)
Description	ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to \<36 months (Part 1) and aged ≥6 to \<48 months (Part 2). Part 1 follows an open-label, dose-escalation design, and Part 2 is a randomized, double-blind, sham delayed-treatment control, dose-selection study. (Show More)
Development Status	Active
Indication	Dravet Syndrome
Disease Ontology Term	DOID:0080422
Compound Name	ETX101
Compound Description	rAAV9-reGABA-eTFSCN1A
Sponsor	Encoded Therapeutics
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	22
Results Posted	Not Available

Target Gene/Variant	SCN1A
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intracerebroventricular
Drug Product Type	Viral vector
Target Tissue/Cell	GABAergic inhibitory interneurons
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type	none
Dose 1	Undisclosed low dose
Dose 2	Undisclosed high dose
Dose 3
Dose 4
Dose 5

No.of Trial Sites	3
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Dosing and preliminary safety and efficacy data expected in 2H25