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CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)
NCT03566043 · View on ClinicalTrials.gov ↗
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 Studied
Interventions
- GENETIC RGX-121
Study Locations (5)
Pennsylvania
- Children's Hospital of Philadelphia — Philadelphia
- Children's Hospital of Pittsburgh - UPMC: Program for Neurodevelopment in Rare Disorders — Pittsburgh
California
- University of California San Francisco, Benioff Children's Hospital — Oakland
New Jersey
- St. Peter's University Hospital — New Brunswick
Rio Grande do Sul
- Hospital de Clinicas de Porto Alegre — Porto Alegre
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 48 participants |
| Start Date | 2018-09-27 |
| Est. Completion | 2025-08 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03566043
The ClinicalTrials.gov registry entry for NCT03566043 describes a study currently listed as active not recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 48 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is REGENXBIO, which has 32 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Mucopolysaccharidosis Type II (MPS II) appearing as the primary indexed condition, and to 1 intervention — of which RGX-121 is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT03566043 reports 5 study locations spanning 4 distinct geographic areas — top geographies include Pennsylvania, California, New Jersey. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT03566043 about?
NCT03566043 is a clinical study titled "CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)". 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.
What is the current status of trial NCT03566043?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 48 participants. The study started on 2018-09-27. Estimated completion is 2025-08.
What conditions does trial NCT03566043 study?
This clinical trial studies the following conditions: Mucopolysaccharidosis Type II (MPS II). These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT03566043?
The interventions under investigation include: RGX-121 (GENETIC). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT03566043?
This trial is sponsored by REGENXBIO, which has 32 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT03566043 being conducted?
This trial has 5 study locations across California, New Jersey, Pennsylvania, Rio Grande do Sul. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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