drugset / Trial / NCT04030052
Emicizumab PUPs and Nuwiq ITI Study
Non-randomizedParallel-groupOpen-labelPrevention
Summary
This study prospectively investigates the safety, FVIII immunogenicity, and hemostatic efficacy of prophylactic HEMLIBRA® given with a concomitant low dose recombinant factor VIII (rFVIII) known as NUWIQ®, in HA infants and children \<3 years old who have had little to no previous exposure to FVIII. In addition, the study investigates the safety and efficacy of a novel FVIII ITI regimen in children \<21 with existing low and high titer inhibitors (LTI and HTI).
Timeline
- Start
- 2022-02-17
- Primary completion
- 2023-01-19
- Completion
- 2023-01-19
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Emicizumab | Monoclonal antibody | 1.5 mg/kg | Subcutaneous |
| Subject | Emicizumab | Monoclonal antibody | 3 mg/kg | Subcutaneous |
| Subject | Emicizumab | Monoclonal antibody | 6 mg/kg | Subcutaneous |
| Subject | simoctocog alfa | Protein / enzyme biologic | 25 iu/kg | Intravenous |
| Subject | simoctocog alfa | Protein / enzyme biologic | 100 iu/kg | Intravenous |