Who is sponsoring NCT04571970?

NCT04571970 is sponsored by REGENXBIO Inc..

What condition does NCT04571970 study?

NCT04571970 studies Mucopolysaccharidosis Type II (MPS II).

What is the status of NCT04571970?

NCT04571970 is currently COMPLETED.

Last reviewed 2025-01-28 · How we verify

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

NCT04571970 PHASE1, PHASE2 COMPLETED

RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a phase I/II study to determine whether RGX-121 is safe, well tolerated, and potentially effective in children five years of age and over who have severe MPS II.

Details

Lead sponsor	REGENXBIO Inc.
Phase	PHASE1, PHASE2
Status	COMPLETED
Enrolment	6
Start date	Thu Mar 11 2021 00:00:00 GMT+0000 (Coordinated Universal Time)
Completion	Thu May 23 2024 00:00:00 GMT+0000 (Coordinated Universal Time)

Conditions

Mucopolysaccharidosis Type II (MPS II)

Interventions

RGX-121

Countries

Canada, United States