CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)
Public ClinicalTrials.gov record NCT03566043. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 1/2/3 Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)
Study identification
- NCT ID
- NCT03566043
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 2, Phase 3
- Lead sponsor
- REGENXBIO Inc.
- Industry
- Enrollment
- 48 participants
Conditions and interventions
Conditions
Interventions
- RGX-121 Genetic
Genetic
Eligibility (public fields only)
- Age range
- 4 Months to 5 Years
- Sex
- Male
- Healthy volunteers
- Not listed
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 26, 2018
- Primary completion
- Nov 26, 2023
- Completion
- Jul 31, 2025
- Last update posted
- Jan 27, 2025
2018 – 2025
United States locations
- U.S. sites
- 4
- U.S. states
- 3
- U.S. cities
- 4
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California San Francisco, Benioff Children's Hospital | Oakland | California | 94609 | — |
| St. Peter's University Hospital | New Brunswick | New Jersey | 08901 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Children's Hospital of Pittsburgh - UPMC: Program for Neurodevelopment in Rare Disorders | Pittsburgh | Pennsylvania | 15224 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 1 non-U.S. site.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03566043, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jan 27, 2025 · Synced Apr 22, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03566043 live on ClinicalTrials.gov.