CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)
Running, not enrolling · Phase 2/Phase 3
Conditions studied: Mucopolysaccharidosis Type II (MPS II)
In brief
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.
Key facts
- Study ID
- NCT03566043
- Run by
- REGENXBIO Inc.
- People needed
- 48
- Starts
- 2018-09-27
- Expected to finish
- 2025-08-01
- Last updated by the study team
- 2025-01-28
Who can join
Age: 0 and older, up to 5. Sex: male. Healthy volunteers: not accepted.
Where it is running
- University of California San Francisco, Benioff Children's Hospital — Oakland, California, United States
- St. Peter's University Hospital — New Brunswick, New Jersey, United States
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- Children's Hospital of Pittsburgh - UPMC: Program for Neurodevelopment in Rare Disorders — Pittsburgh, Pennsylvania, United States
- Hospital de Clinicas de Porto Alegre — Porto Alegre, Rio Grande do Sul, Brazil
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.