Clinical Trial

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

Active, Not Recruiting Phase 3
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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)
Protocol Amendment History 3 amendments
This ClinicalTrials.gov record has been amended 3 times since 2025-11-14; most recent amendment 2026-06-02.
Status change: Suspended → Active, Not Recruiting 2026-06-02
Status change: Recruiting → Suspended 2026-02-25
Status change: Not Yet Recruiting → Recruiting 2025-11-24
Trial Details
NCT Number NCT07236606
Lead Sponsor REGENXBIO Inc.
Conditions MPS II, Hunter Syndrome (MPS II)
Enrollment 2 participants
Start Date 2025-11-25
Primary Completion 2028-04-30 (estimated)
Study Completion 2031-05-30 (estimated)
Updated on ClinicalTrials.gov 2026-06-04