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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 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. The trial reports 5 study locations across 4 states.
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.
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 |
Interested in This Trial?
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, categorized as Phase 2. 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 9 total studies on file at ClinicalTrials.gov.
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 reports 5 study locations spanning 4 distinct geographic areas - top geographies include 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.
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