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)
Indication	Mucopolysaccharidosis Type II (Hunter Syndrome)
Compound Name	RGX-121 (AAV9-IDS)
Sponsor	REGENXBIO Inc.
Funder Type	Industry
Status	Active not recruiting
Enrollment Count	48

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
Dose 1	1.3 x 10^10 GC/g brain mass
Dose 2	6.5 x 10^10 GC/g brain mass
Dose 3	2.9 x 10^11 GC/g brain mass
Dose 4
Dose 5

No.of Trial Sites	5
Locations	United States,Brazil

Has US IND	True
Recent Updates	BLA filing planned for Q3 2024