drugset / Trial / NCT03566043
CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)
Non-randomizedParallel-groupOpen-labelTreatment
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.
Timeline
- Start
- 2018-09-27
- Primary completion
- 2023-11-27
- Completion
- 2025-08
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | CLEMIDSOGENE LANPARVOVEC | Gene therapy (AAV / viral vector) | 1.3e+10 unknown | — |
| Subject | CLEMIDSOGENE LANPARVOVEC | Gene therapy (AAV / viral vector) | 6.5e+10 unknown | — |
| Subject | CLEMIDSOGENE LANPARVOVEC | Gene therapy (AAV / viral vector) | 2e+11 unknown | — |
| Subject | CLEMIDSOGENE LANPARVOVEC | Gene therapy (AAV / viral vector) | 2.9e+11 unknown | — |