Gene Therapy for Children With CLN3 Batten Disease

NCTID	NCT03770572 (View at clinicaltrials.gov)
Description	This is a phase 1/2, open-label, single dose, dose-escalation clinical trial to evaluate the safety and efficacy of AT-GTX-502 (previous NCH Code: scAAV9.P546.CLN3) delivered intrathecally into the lumbar spinal cord region of subjects with CLN3 Batten disease. (Show More)
Development Status	Inactive
Indication	CLN3 Batten Disease, Juvenile Neuronal Ceroid Lipofuscinosis
Disease Ontology Term	DOID:0110731
Compound Name	AT-GTX-502
Compound Description	scAAV9.Mecp2.CLN3
Sponsor	Amicus Therapeutics
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	7
Results Posted	Not Available

Target Gene/Variant	CLN3
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	6E13 vg (no more than 5mL)
Dose 2	1.2E14 vg (no more than 10mL)
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Amicus returned development rights to Abigail Wexner Research Institute at Nationwide Children's Hospital in January 2024