Von Willebrand's Disease (VWD)Phase 1NCT04770935
To Assess the Pharmacokinetics and Safety and Tolerability of Efanesoctocog Alfa (BIVV001)in Adults With Type 2N and 3 Von Willebrand Disease (VWD)
A Phase 1 study testing efanesoctocog alfa (BIVV001) in people with Von Willebrand's Disease (VWD), run by Bioverativ, a Sanofi company at 4 sites in 2 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Von Willebrand's Disease (VWD)
- What is being tested?
- efanesoctocog alfa (BIVV001)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 3 May 2021to 20 Dec 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- -- 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.
Who cannot join
- 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
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- 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.
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
United States2 sites
University of Iowa_Investigational Site Number :8400002
Iowa City, Iowa, 52242
Hemophilia Center of Western Pennsylvania_Investigational Site Number :8400001
Pittsburgh, Pennsylvania, 15213
France2 sites
Investigational Site Number :2500001
Lille, 59037
Investigational Site Number :2500002
Nantes, 44093
About this study
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)
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
Read the 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).
- Conditions
- Von Willebrand's Disease (VWD)
- Being tested
- efanesoctocog alfa (BIVV001)
- Medical subject headings
- von Willebrand Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
efanesoctocog alfa (BIVV001)Drug
Pharmaceutical form:solution for injection Route of administration: intravenous injection
| Study group | Role | What they receive |
|---|---|---|
| efanesoctocog alfa (BIVV001) | Experimental | A single IV dose of BIVV001 will be administered to each patient Receives: Drug: efanesoctocog alfa (BIVV001) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetic Parameter: Maximum plasma concentration observed (Cmax)
Measured: Day 1 to Day 10
Pharmacokinetic Parameter: Terminal half-life (t½z)
Measured: Day 1 to Day 10
Pharmacokinetic Parameter: Total Clearance (CL)
Measured: Day 1 to Day 10
Pharmacokinetic Parameter: Volume of distribution at steady state (Vss)
Measured: Day 1 to Day 10
Pharmacokinetic Parameter: Area under the activity time curve extrapolated to infinity (AUC∞)
Measured: Day 1 to Day 10
Pharmacokinetic Parameter: Mean residence time (MRT)
Measured: Day 1 to Day 10
Pharmacokinetic Parameter: Incremental recovery (IR)
Measured: Day 1 to Day 10
Secondary outcomes (1)
Number of participants with adverse events
Measured: Up to Day 29
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2021
- Study start3 May 2021
- Primary completion1 Dec 2022
- Study completion20 Dec 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Willebrand's Disease (VWD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bioverativ, a Sanofi company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04770935
- Sponsor's protocol ID
- PKM16978
- Other IDs
- 2020-004947-10, U1111-1255-4463
- Status last verified
- Sep 2025
- First submitted
- 22 Feb 2021
- Last updated
- 25 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT04770935
- Acronym / sponsor ID
- PKM16978
- Conditions
- Von Willebrand's Disease (VWD)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Bioverativ, a Sanofi companyIndustry (pharma, biotech, devices)
- Study start
- 3 May 2021 (actual)
- Primary completion
- 1 Dec 2022 (actual)
- Study completion
- 20 Dec 2022 (actual)
- First posted
- 25 Feb 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| efanesoctocog alfa (BIVV001) | Experimental | Drug: efanesoctocog alfa (BIVV001)A single IV dose of BIVV001 will be administered to each patient |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- -- 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.
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| efanesoctocog alfa (BIVV001)Pharmaceutical form:solution for injection Route of administration: intravenous injection | Drug | efanesoctocog alfa (BIVV001) |
Primary outcome
Pharmacokinetic Parameter: Maximum plasma concentration observed (Cmax)
Time frame: Day 1 to Day 10
Pharmacokinetic Parameter: Terminal half-life (t½z)
Time frame: Day 1 to Day 10
Pharmacokinetic Parameter: Total Clearance (CL)
Time frame: Day 1 to Day 10
Pharmacokinetic Parameter: Volume of distribution at steady state (Vss)
Time frame: Day 1 to Day 10
Pharmacokinetic Parameter: Area under the activity time curve extrapolated to infinity (AUC∞)
Time frame: Day 1 to Day 10
Pharmacokinetic Parameter: Mean residence time (MRT)
Time frame: Day 1 to Day 10
Pharmacokinetic Parameter: Incremental recovery (IR)
Time frame: Day 1 to Day 10
1 secondary outcome
Number of participants with adverse events
Time frame: Up to Day 29
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2025.
- PKM16978 Plain Language Results SummaryLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations study director
Sanofi
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing efanesoctocog alfa (BIVV001) in people with Von Willebrand's Disease (VWD), run by Bioverativ, a Sanofi company at 4 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Pharmacokinetic Parameter: Maximum plasma concentration observed (Cmax) (time frame: Day 1 to Day 10). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Sep 2025.