RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)

Summary

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, efficacy, and pharmacodynamic dose ranging study to determine whether RGX-121 is safe, effective and well-tolerated by patients with MPS II (Hunter Syndrome)

Timeline

Start

2025-11-25

Primary completion

2028-04-30

Completion

2031-05-30

Drugs

Indications