A Phase 1 interventional study of efanesoctocog alfa (BIVV001) in Von Willebrand's Disease (VWD), sponsored by Bioverativ, a Sanofi company. Completed at 4 sites in 2 countries. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2025-09-25.
Sponsored by Bioverativ, a Sanofi company (part of Sanofi) · Phase 1, Interventional, and Other
Among 38 studies in von Willebrand Diseases with a site in United States — 11 open to participants now, 12 under way, 2 of them phase 3. von Willebrand Diseases · United States.
Among the 38 studies in von Willebrand Diseases with a site in United States, this one is the 21st largest by enrollment of the 22 interventional ones that give a count, is one of 4 that started in 2021, is one of 2 in phase 1, and was the 26th registered.
96 studies on the registry are indexed under von Willebrand Diseases; 25 are open to participants now.
Browse von Willebrand Diseases studies →11 of the 38 studies in von Willebrand Diseases with a site in United States are open now. Recruiting first, the newest first:
Alnylam Pharmaceuticals · Sites in United States, Netherlands, Sweden and 2 more countries
Ages 16–45 · Women only
Vega Therapeutics, Inc · Sites in United States, Australia, Austria and 7 more countries
Ages 12–75
Oregon Health and Science University · Sites in United States
Ages 10–24 · Women only
Hemab ApS · Sites in United States, Australia, United Kingdom
Ages 16–69
Takeda · Sites in United States, France, Ireland and 3 more countries
Ages up to 17
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 23 von Willebrand Diseases studies recruiting now, 15 (65%) set no upper age limit and 8 (35%) stop at 75 or younger; 14 take children under 18; 3 take healthy volunteers.
This study: 18 Years to 65 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
38 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 4 · Phase 2 7 · Phase 3 11 · Phase 4 1
First in 2001; this study’s year is marked. 4 started in 2021.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 6 is below the median of 25 across 41 interventional studies indexed under von Willebrand Diseases.
Median enrollment 30 across the 22 interventional studies, 127 across the 16 observational. Of the 25 that completed or were terminated, 13 (52%) have results posted.
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18 of 38 have an industry lead sponsor. The 10 most frequent:
This study is registered under Bioverativ, a Sanofi company, one of the names counted as Sanofi. Under that name alone:
Bioverativ, a Sanofi company is the lead sponsor of 16 studies on the registry; none are open to participants now.
Of its 12 completed or terminated interventional studies of FDA-regulated products, 7 (58%) have results posted.
Sanofi has 1 study filed under von Willebrand Diseases — fewer than ten, so there is no comparison to draw.
4 of the studies in von Willebrand Diseases with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06610201Hemab ApSA Study of Bleeding and Treatment in Participants With Von Willebrand Disease | 2026-12 |
| NCT04146376University of WashingtonVon Willebrand Factor in Pregnancy (VIP) Study | 2026-12 |
| NCT07575308Alnylam PharmaceuticalsHMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB | 2027-06-30 |
| NCT06754852Hemab ApSA Study Assessing HMB-002 in Participants With Von Willebrand Disease | 2027-07 |
Add all 4 to your calendar (.ics) → With an account.
Among the 38 studies in von Willebrand Diseases with a site in United States, from their registry updates:
Nothing recorded among them since 2026-09-25.
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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
Duration of each part of the study for one participant:
Total study duration: Up to 57 days.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
-- Male and/or female participant, between 18 and 65 years of age, inclusive at the time of .
Exclusion criteria:
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A single IV dose of BIVV001 will be administered to each patient
Drug: efanesoctocog alfa (BIVV001)
Pharmaceutical form:solution for injection Route of administration: intravenous injection
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
Number of participants with adverse events
Time frame: Up to Day 29
Plan to share: 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
Ran from May 2021 to primary completion in Dec 2022, 18 months.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Sep 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Efanesoctocog alfa
Bioverativ, a Sanofi company →