MPS II Treatment with RGX-121 – Phase I/II

Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects with MPS II (Hunter Syndrome)

Protocol Description

The purpose of this multi-center study is to assess the safety and effects of a gene transfer called RGX-121 to help patients with mucopolysaccharidosis type II (MPS II) also called Hunter syndrome. In this inherited genetic condition, a defective iduronate-2-sulfatase gene (IDS) prevents production of the enzyme iduronate-2-sulfatase (I2S). The lack of circulating I2S enzyme can lead to progressive damage to the brain, heart, and other organs. RGX-121 contains a healthy IDS gene that is thought to help cells to make the needed enzyme. This study is for boys whose disease progression has affected their neurocognitive abilities.

Study Level: Phase I/II

Eligibility Criteria

Subject to exclusion criteria, this study is enrolling male babies, toddlers, and very young boys who have been diagnosed with MPS II with neurocognitive involvement.
Boys: Ages 4 months to 5 years

Requirements

Participants will receive a single infusion of RGX-121 delivered to the central nervous system. After that, participants will return to UPMC Children’s Hospital of Pittsburgh for examinations, testing, imaging, and blood draws in accordance with the protocol schedule over a 2-year period. By invitation, participants may be able to participate in a long-term follow-up study.
Duration: 104 weeks (about 2 years) after receiving RGX-121

Status: Open to Enrollment

Source(s) of Support

Regenxbio Inc.

Primary Investigator

Maria L. Escolar, MD, MS

Contact Information

For more information about the study or enrollment, please contact:
Jodi Martin, Clinical Research Coordinator
412-692-6351