Trials / Completed
CompletedNCT04759131
Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A
A Phase 3 Open-label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Pediatric Patients <12 Years of Age With Severe Hemophilia A
- Status
- Completed
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 74 (actual)
- Sponsor
- Bioverativ, a Sanofi company · Industry
- Sex
- Male
- Age
- 11 Years
- Healthy volunteers
- Not accepted
Summary
Primary Objective: \- To evaluate the safety of BIVV001 in previously treated pediatric participants with hemophilia A. Secondary Objectives: * To evaluate the efficacy of BIVV001 as a prophylaxis treatment. * To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes. * To evaluate BIVV001 consumption for prevention and treatment of bleeding episodes. * To evaluate the effect of BIVV001 prophylaxis on joint health outcomes. * To evaluate the effect of BIVV001 prophylaxis on Quality of Life (QoL) outcomes. * To evaluate the efficacy of BIVV001 for perioperative management. * To evaluate the safety and tolerability of BIVV001 treatment. * To assess the pharmacokinetics (PK) of BIVV001.
Detailed description
Study duration per participants was approximately 60 weeks (maximum 8 weeks for screening and 52 weeks of treatment). All participants completing or remaining at the end of study were offered participation in the planned extension trial.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | efanesoctocog alfa (BIVV001) | Pharmaceutical form: solution for injection Route of administration: IV |
Timeline
- Start date
- 2021-02-19
- Primary completion
- 2023-01-18
- Completion
- 2023-01-18
- First posted
- 2021-02-18
- Last updated
- 2025-09-11
- Results posted
- 2024-02-13
Locations
40 sites across 15 countries: United States, Australia, Canada, France, Germany, Hungary, Ireland, Italy, Netherlands, Spain, Sweden, Switzerland, Taiwan, Turkey (Türkiye), United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04759131. Inclusion in this directory is not an endorsement.