Clinical trial NCT04770935: To Assess the Pharmacokinetics and Safety and Tolerability of Efanesoctocog Alfa (BIVV001)in Adults With Type 2N and 3 Von Willebrand Disease (VWD)
Official title: A Phase 1, Open-Label Study to Assess the Pharmacokinetics, and Safety and Tolerability of a Single Intravenous Injection of rFVIIIFc-VWF-XTEN (BIVV001) in Adults With Type 2N and 3 Von Willebrand Disease (VWD)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Von Willebrand's Disease (VWD)
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2021-02-25
Enrollment: 6
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

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 activated partial thromboplastin time (aPPT) clotting assay, as well as, BIVV001 capture chromogenic Coatest FVIII activity assay

Secondary Objective:

-To assess the safety and tolerability of a single IV dose of BIVV001 in adult patients with type 2N and 3 VWD

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 administration (this period includes PK sampling up to the first 10 days following administration).

Eligibility criteria: 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.

Primary outcomes:
measure: Pharmacokinetic Parameter: Maximum plasma concentration observed (Cmax)
timeFrame: Day 1 to Day 10
measure: Pharmacokinetic Parameter: Terminal half-life (t½z)
timeFrame: Day 1 to Day 10
measure: Pharmacokinetic Parameter: Total Clearance (CL)
timeFrame: Day 1 to Day 10
measure: Pharmacokinetic Parameter: Volume of distribution at steady state (Vss)
timeFrame: Day 1 to Day 10
measure: Pharmacokinetic Parameter: Area under the activity time curve extrapolated to infinity (AUC∞)
timeFrame: Day 1 to Day 10
measure: Pharmacokinetic Parameter: Mean residence time (MRT)
timeFrame: Day 1 to Day 10
measure: Pharmacokinetic Parameter: Incremental recovery (IR)
timeFrame: Day 1 to Day 10
Secondary outcomes:
measure: Number of participants with adverse events
timeFrame: Up to Day 29
Other outcomes:
Locations:
city: Iowa City
country: United States
facility: University of Iowa_Investigational Site Number :8400002
state: Iowa
city: Pittsburgh
country: United States
facility: Hemophilia Center of Western Pennsylvania_Investigational Site Number :8400001
state: Pennsylvania
city: Lille
country: France
facility: Investigational Site Number :2500001
city: Nantes
country: France
facility: Investigational Site Number :2500002
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: