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NCT03566043 · ClinicalTrials.gov registry record · Phase 2
CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)
A Phase 2 study of Mucopolysaccharidosis Type II (MPS II), sponsored by REGENXBIO.
- Active
- Registry status
- Phase 2
- Development phase
- 48
- Enrollment target
- 5
- Study locations
NCT03566043: Active Phase 2 study of Mucopolysaccharidosis Type II (MPS II), sponsored by REGENXBIO.
NCT03566043 is a Phase 2 study of Mucopolysaccharidosis Type II (MPS II) that is active but no longer recruiting, run by REGENXBIO. The registered enrollment target is 48 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (64% lower). The trial reports 5 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03566043, a Phase 2 study of Mucopolysaccharidosis Type II (MPS II), is active but no longer recruiting, sponsored by REGENXBIO.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 48 participants
- Enrollment target
- 5
- Study locations
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.
Primary Outcome
Number of participants with treatment-related adverse events and serious adverse events as assessed by CTCAE (Version 4.03).
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 |
What the registry record for NCT03566043 still lists
NCT03566043 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 48 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (64% lower).
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.
NCT03566043 lists 5 locations in 4 states (Pennsylvania, California, New Jersey).
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).
What interventions are being tested in trial NCT03566043?
The interventions under investigation include: RGX-121 (GENETIC).
Who is sponsoring clinical trial NCT03566043?
This trial is sponsored by REGENXBIO, which has 9 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03566043's enrollment target sits among peer trials
48 1265th of 2000 higher than 718 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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