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

Study 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 and efficacy, dose ranging study to determine whether RGX-121 is safe, effective and well-tolerated by patients with MPS II.

Conditions

Mucopolysaccharidosis Type II (MPS II)

Interventions / Drugs

RGX-121

View on ClinicalTrials.gov ↗

Sponsor

Study Details

Enrollment48 participants

Primary Completion—

Lead SponsorREGENXBIO Inc.

Data Retrieved2026-04-11 04:25:55.708144+00:00

Not financial advice. BiotechSigns data is sourced from ClinicalTrials.gov, SEC EDGAR, and other public databases for research and informational purposes only. It does not constitute investment recommendations. Always do your own due diligence before making investment decisions.