Status:
COMPLETED
To Assess the Pharmacokinetics and Safety and Tolerability of Efanesoctocog Alfa (BIVV001)in Adults With Type 2N and 3 Von Willebrand Disease (VWD)
Lead Sponsor:
Bioverativ, a Sanofi company
Conditions:
Von Willebrand's Disease (VWD)
Eligibility:
All Genders
18-65 years
Phase:
PHASE1
Brief Summary
Primary Objective: -To characterize the pharmacokinetics (PK) of BIVV001 after a single intravenous (IV) administration, as assessed by factor VIII (FVIII) activity determined by the one-stage activa...
Detailed Description
Duration of each part of the study for one participant: Total study duration: Up to 57 days. * Screening: up to 28 days. * Up to 29 days of safety observation following the IV BIVV001 dose administr...
Eligibility Criteria
Inclusion
- Inclusion criteria :
- \-- Male and/or female participant, between 18 and 65 years of age, inclusive at the time of informed consent.
- The participant has been diagnosed with hereditary type 3 VWD or type 2N VWD as documented in historical medical records OR a documented genotype known to produce VWD type 3 or 2N VWD.
- Type 3 VWD participants are included if they have a medical history of at least 25 exposure days to VWF and factor VIII-containing coagulation factor concentrates
- Type 2N VWD participants are included if the use of DDAVP is deemed insufficient or contraindicated, as assessed by the Investigator, or if they have required prior use of VWF- and FVIII- containing coagulation factor concentrates.
- Exclusion criteria:
- Hereditary or acquired coagulation disorder other than VWD (including qualitative and quantitative platelet disorders, and thrombocytopenia \< 100,000 cells/uL at Screening)
- The participant has a FVIII activity levels \>20 IU/dL, at Screening
- History or presence of a VWF inhibitor or clinical suspicion of a VWF inhibitor
- History of a positive FVIII inhibitor test, defined as ≥0.6 BU/mL (by Nijmegen modified Bethesda assay) or a clinical suspicion of a FVIII inhibitor
- Positive FVIII inhibitor test, defined as ≥0.6 BU/mL, at Screening
- History of hypersensitivity or anaphylaxis associated with any FVIII- or VWF- containing product
- The participant has received or anticipates receiving systemic immunosuppressive or immunomodulatory treatment within 12 weeks prior to Baseline.
- The participant requires the use of acetylsalicylic acid, non-NSAID anti-platelets, and NSAIDs above the maximum dose product
- Patients currently on a prophylaxis regimen for the treatment of VWD that, in the Investigator's opinion, would preclude participation in the study due to the possible increased risk of bleeding associated with the requirement to withhold prophylaxis during the study.
- The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Exclusion
Key Trial Info
Start Date :
May 3 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 20 2022
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT04770935
Start Date
May 3 2021
End Date
December 20 2022
Last Update
September 25 2025
Active Locations (4)
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1
University of Iowa_Investigational Site Number :8400002
Iowa City, Iowa, United States, 52242
2
Hemophilia Center of Western Pennsylvania_Investigational Site Number :8400001
Pittsburgh, Pennsylvania, United States, 15213
3
Investigational Site Number :2500001
Lille, France, 59037
4
Investigational Site Number :2500002
Nantes, France, 44093