Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)

NCTID	NCT01161576 (View at clinicaltrials.gov)
Description	This is a proposed follow up study on the investigators previous gene transfer human clinical trial entitled "Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Expressing the Human CLN2 cDNA to the Brain of Children with Late Infantile Neuronal Ceroid Lipofuscinosis" (Weill Cornell IRB# 0401007010). As in the previous study, the investigators propose to administer a biologic by direct gene transfer into the brain and assess its safety on children with a fatal genetic disease of the central nervous system (CNS). The disease is Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL, a form of Batten disease). This will be accomplished by using delivery of a gene (method called gene transfer) to administer to the brain an experimental drug called AAVRh.10CUhCLN2, a gene transfer vector. (Show More)
Development Status	Inactive
Indication	CLN2 Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis
Disease Ontology Term	DOID:0110726
Compound Name	LX1004
Compound Description	AAVrh.10CUhCLN2
Sponsor	Weill Medical College of Cornell University
Funder Type	Other
Recruitment Status	Completed
Enrollment Count	12
Results Posted	Not Available

Target Gene/Variant	TPP1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intracisternal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAVrh10
Editor Type	none
Dose 1	2.85E11 gc
Dose 2	9.0E11 gc
Dose 3	3E12 particle units (AAV2 vector)
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	Product was licensed to Lexeo Therapeutics, renamed LX1004, product development was discontinued