Clinical Trial

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

Active, Not Recruiting Phase 2/3
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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 and efficacy, dose ranging study to determine whether RGX-121 is safe, effective and well-tolerated by patients with MPS II.
Protocol Amendment History 21 amendments
This ClinicalTrials.gov record has been amended 21 times since 2018-06-11; most recent amendment 2025-01-24.
Status change: Recruiting → Active, Not Recruiting 2023-11-17
Status change: Not Yet Recruiting → Recruiting 2018-07-11
Trial Details
NCT Number NCT03566043
Lead Sponsor REGENXBIO Inc.
Conditions Mucopolysaccharidosis Type II (MPS II)
Enrollment 48 participants
Start Date 2018-09-27
Primary Completion 2023-11-27 (estimated)
Study Completion 2025-08 (estimated)
Updated on ClinicalTrials.gov 2025-01-28