Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease

NCTID	NCT02725580 (View at clinicaltrials.gov)
Description	This is a phase 1/2, open-label, single dose study to evaluate the safety and efficacy of AT-GTX-501 delivered intrathecally into the lumbar spinal cord region of participants with mild to moderate variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene (vLINCL6 disease). (Show More)
Development Status	Inactive
Indication	Variant Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN6 Batten Disease)
Disease Ontology Term	DOID:0110729; DOID:0110730
Compound Name	AT-GTX-501
Compound Alias	Sesiclenegene cosaparvovec
Compound Description	scAAV9.CB.CLN6
Sponsor	Amicus Therapeutics
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	13
Results Posted	View Results

Target Gene/Variant	CLN6
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intrathecal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type	none
Dose 1	1.5E13 vg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations	Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Program was discontinued due to lack of sustained efficacy in LTFU study, Amicus returned development rights to Abigail Wexner Research Institute at Nationwide Children's Hospital in January 2024