CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

NCTID	NCT03566043 (View at clinicaltrials.gov)
Description	RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study is a safety and efficacy, dose ranging study to determine whether RGX-121 is safe, effective and well-tolerated by patients with MPS II. (Show More)
Development Status	Active
Indication	Mucopolysaccharidosis Type II (Hunter Syndrome)
Disease Ontology Term	DOID:12799
Compound Name	RGX-121
Compound Alias	clemidosogene lanparvovec
Compound Description	AAV9-IDS
Sponsor	REGENXBIO Inc.
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	48
Results Posted	Not Available

Target Gene/Variant	IDS
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intracisterna magna
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type	none
Dose 1	1.3E10 GC/g brain mass
Dose 2	6.5E10 GC/g brain mass
Dose 3	2.9E11 GC/g brain mass (pivotal dose)
Dose 4
Dose 5

No.of Trial Sites	5
Locations	United States,Brazil

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates	Program is now being developed as part of a partnership with Nippon Shinyaku