Von Willebrand Disease, Type 3Phase 1BCDI-XIINCT05500807
Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A
A Phase 1 study testing Emicizumab in people with Von Willebrand Disease, Type 3 and Concomitant VWD and Hemophilia, run by Bleeding and Clotting Disorders Institute Peoria, Illinois at 12 sites in United States.
Trial summary
Von Willebrand Disease (VWD) is the most common inherited bleeding disorder affecting up to 0.1% of the population, is usually characterized by mucocutaneous bleeding, HMB, surgical bleeding or other hemostatic challenges. Severe bleeding events require VWF concentrates administered solely through intravenous access. Read more about this study
- Who is it for?
- Ages 0 to 90 years
- Conditions the study is for
- Von Willebrand Disease, Type 3, Concomitant VWD and Hemophilia
- What is being tested?
- Emicizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 12 sitesUnited States
- Study dates
- 1 Nov 2022to Feb 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 0 to 90 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Signed informed consent
- Age 0 and older (infants weighing ≥3 kg)
- ability to comply with protocol in investigators judgement
- diagnosis of: severe VWD type 3, or VWD with VWF antigen, activity or collagen binding </= 20 U/dl or variant VWD confirmed by genetic mutation and VWF ag, activity or CB < 50 U/dl based on historical medical records of study site.
- diagnosis of VWD/hemophilia A defined as VWF:ag, activity or CB <50 U/dl, and mild moderate or severe hemophilia A(defined by ISTH criteria) based on historical medical records of the study site.
- plan to be adherent to emicizumab prophylaxis during the study
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- Patient's bleeding phenotype necessitating prophylaxis per treating provider recommendations.
- Patient on current prophylaxis for VWD or VWD/hemophilia A may enroll if they are currently on a non-emicizumab agent, and if it has been > 18 months since last off-label dose of emicizumab, and are willing to discontinue current prophylaxis.
- For menstruating individuals: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods that result in a failure rate of < 1% per year during the study period. A menstruating individual is considered to be of childbearing potential if they are post-menarchal, have not reached a postmenopausal state (12 continuous months of amenorrhea with no identified cause other than menopause), and have not undergone surgical sterilization (removal of ovaries and/or uterus).
- Examples of highly effective contraceptive methods with a failure rate of < 1% per year include proper use of combined oral or injected hormonal contraceptive, bilateral tubal ligation, male sterilization, hormone-releasing intrauterine devices, and copper intrauterine devices. The reliability of sexual abstinence should be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, or post-ovulation methods) and withdrawal are not acceptable methods of contraception.
Who cannot join
- Patients and/or infants weighing < 3 kg.
- Patients with low VWF or non-severe VWD (ie.not meeting the above criteria)
- Other concomitant bleeding disorders including coagulopathy from liver cirrhosis.
- Current treatment with emicizumab or emicizumab therapy in the previous 18 months.
- Previous (in the past 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or current signs of thromboembolic disease
- Other conditions (e.g., certain autoimmune diseases, including, but not limited to diseases such as systemic lupus erythematosus, inflammatory bowel disease, and antiphospholipid syndrome) that may increase the risk of bleeding or thrombosis
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- Patients who are at high risk for thrombotic microangiopathy (TMA; e.g., have a previous medical or family history of TMA), in the investigator's judgment
- Would refuse treatment with blood or blood products, if necessary.
- Any serious medical condition or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the patient's safe participation in and completion of the study
- Treatment with any of the following:
- An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration before Study Day 1 A non-hemophilia-related investigational drug within the last 30 days or 5 halflives- before Study Day 1, whichever is longer An investigational drug concurrently
- History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
- Pregnant or lactating, or intending to become pregnant during the study
- Women of childbearing potential must have a negative serum pregnancy test result within 7 days before Study Day 1
- Illicit drug or alcohol abuse within 12 months prior to screening, in the investigator's judgment
- Serious infection requiring oral or IV antibiotics within 30 days prior to screening
Trial locations and contacts
12 sites in 1 country. Contact the study team or a site near you to ask about taking part.
12 sites in 1 country
United States12 sites, 12 recruiting
- Recruiting
University of Michigan Medical School
Ann Arbor, Michigan, 48109
Jordan Kindshoven, jkindsh@med.umich.edu
Adam Comstock, acomstoc@med.umich.edu
- Recruiting
University of Miami - Miller School of Medicine
Coral Gables, Florida, 33146
Jorge Perez, jxp943@med.miami.edu
Leandro Pisani, lfp34@miami.edu
- Recruiting
Penn State College of Medicine
Hershey, Pennsylvania, 17033
Erica Miller, emiller25@pennstatehealth.psu.edu
Brennen Hartig, bharding@pennstatehealth.psu.edu
- Recruiting
Innovative Hematology, Inc. (IHI)
Indianapolis, Indiana, 46260
Shannon Mears, smears@IHTC.org
Young Chong, ychong@IHTC.org
- Recruiting
Children's Mercy Hospital
Kansas City, Missouri, 64108
Anna Wisemann, amwiseman@cmh.edu
Shannon L Carpenter, MD
- Recruiting
UW Health Comprehensive Program for Bleeding Disorders
Madison, Wisconsin, 53792
Hannah Ranous, jmranous@medicine.wisc.edu
Alec J Anderson, ajanderson@medicine.wisc.edu
- Recruiting
Central Michigan University: Children's Hospital of Michigan
Mount Pleasant, Michigan, 48859
Negin Salehi, saleh1n@cmich.edu
Meera Chitlur, MD
- Recruiting
The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders (CIBD)
Orange, California, 92868
Alyssa Barron, abarron@cibd-ca.org
Nicole Crook, ncrook@cibd-ca.org
- Recruiting
Bleeding and Clotting Disorders Institute (BCDI)
Peoria, Illinois, 61614
Dayna Lenski, BS, 309-692-5337 ext. 166, dayna@ilbcdi.org
Sara Malik, MPH, 309-692-5337 ext. 108, sarawm@ilbcdi.org
- Recruiting
Stanford University: Stanford Children's Health
Redwood City, California, 94063
Stephanie Gam, sgam@stanford.edu
Riko Lee, rikolee@stanford.edu
- Recruiting
Washington Center for Bleeding Disorders
Seattle, Washington, 98101
Sophia Beyer, sophia.beyer@wacbd.org
Laura Masserman, laura.masserman@wacbd.org
- Recruiting
St. Joseph's Children's Hospital - Center for Bleeding and Clotting Disorders
Tampa, Florida, 33607
Cindy Manis, Cindy.Manis@baycare.org
Denise Cardwell, Denise.Cardwell@baycare.org
About this study
Official title: Emicizumab for Severe VON Willebrand Disease (VWD) and VWD/Hemophilia A
Von Willebrand Disease (VWD) is the most common inherited bleeding disorder affecting up to 0.1% of the population, is usually characterized by mucocutaneous bleeding, HMB, surgical bleeding or other hemostatic challenges. Severe bleeding events require VWF concentrates administered solely through intravenous access. Emicizumab (Hemlibra) is a monoclonal bispecific antibody developed to bind activated FIX and FX and mimic FVIII cofactor functionality. Hemlibra is administered via subcutaneous injection rather than intravenous infusion. The hypothesis of this study is that Emicizumab is safe and efficacious for prophylaxis in severe VWD and concomitant VWD/hemophilia patients.
Read the detailed description
Von Willebrand Disease (VWD) is the most common inherited bleeding disorder affecting up to 0.1% of the population, is usually characterized by mucocutaneous bleeding, HMB, surgical bleeding or other hemostatic challenges. VWD currently has few therapies generally useful in management of bleeding events including antifibrinolytics, desmopressin (DDAVP), and VWF concentrates, which may be plasma-derived (VWF with and without FVIII) or recombinant. Minor bleeding may be successfully treated with antifibrinolytics and DDAVP; however, more severe bleeding requires VWF concentrates that are administered solely through intravenous access.
Similarly, it can be challenging to treat concomitant bleeding disorders with the existing therapeutic options available, and patients with concurrent VWD and hemophilia A primarily have VWF/FVIII concentrate or desmopressin (DDAVP) available for treatment. It has been well-recognized that patients, caregivers, and medical providers desire additional, simplified therapeutic options that are not intravenous to treat severe bleeding disorders. Therefore, a simplified, subcutaneous therapeutic that prevents bleeding would be strongly desired. Though its use in the hemophilia A population is growing, additional potential emicizumab applications for hemostatic control in other hemostatic disorders remain unknown. A recent case report highlighted the hemostatic efficacy of emicizumab off-label use in type 3 von Willebrand Disease (VWD), another severe bleeding disorder. This pediatric patient had type 3 VWD with alloantibodies and a bleeding phenotype similar to hemophilia A with inhibitor patients, requiring suboptimal bleeding management with rFVIIa and activated prothrombin complex concentrates (aPCC). Emicizumab prophylaxis was initiated and the patient no longer required aPCC prophylaxis and rare use of rFVIIa for acute bleeding events (only 1 trauma-induced soft tissue hematoma at the time of publication). The authors concluded their report suggested the bleeding phenotype in type 3 VWD is expressed mainly due to factor VIII deficiency. This study suggests a potential additional application for emicizumab in severe VWD.
A pilot multicenter, prospective open-label study of emicizumab prophylaxis in severe VWD and concomitant VWD/hemophilia A. Patients will have a one-year retrospective chart review of annualized bleed rate and hemostatic therapies collected at the time of enrollment. Patients will then be treated with emicizumab with 3mg/kg weekly for 4 weeks loading dose, followed by once weekly prophylaxis of 1.5mg/kg for 1 year. Per emicizumab FDA-approved prescribing information for hemophilia A, dose up-titration to 3 mg/kg once weekly will be allowed after 24 weeks on HEMLIBRA prophylaxis in case of suboptimal efficacy (i.e., ≥ 2 spontaneous and clinically significant bleeds) Treatment records will be maintained along with bleeding event logs. Breakthrough bleeding events may be treated with the patients usual on-demand therapy with antifibrinolytics or VWF/FVIII concentrates per clinician discretion. Patient reported outcome assessments will be collected throughout the clinical study to collect impact of the treatment on the individual patients, assessing quality of life, physical, emotional, social and general symptoms.
- Being tested
- Emicizumab
- Medical subject headings
- von Willebrand Disease, Type 3, Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EmicizumabDrug
Also called Hemlibra
Subcutaneous injection of emicizumab for prophylaxis
| Study group | Role | What they receive |
|---|---|---|
| Open Label Emicizumab | Other | Emicizumab prophylaxis Receives: Drug: Emicizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Emicizumab is efficacious for prophylaxis in severe VWD & concomitant VWD/hemophilia A
Establish bleed occurrence during treatment evaluated through descriptive statistical analysis to determine proof of principle
Measured: 18 months
Emicizumab is safe for prophylaxis in severe VWD & concomitant VWD/Hemophilia A
Collection of AE's, hypersensitivity reactions, thrombotic events during treatment
Measured: 18 months
Secondary outcomes (3)
Reduced treatment burden vs VWF/FVIII prophylaxis
# of infusions and methods of infusions collected during study
Measured: 18 months
Decreased bleed occurrence
Evaluate historical ABR with on demand or prophy prior to study treatment
Measured: 12 months
Diminish bleed severity
Collection of bleed data prior to study entry and throughout study treatment
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Aug 2022
- Study start1 Nov 2022
- Primary completionJun 2027 (estimated)
- Study completionFeb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (19)Papers the sponsor has linked to this study.
- Other trials for Von Willebrand Disease, Type 3Studies 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- 40 patients who will receive emicizumab prophylactically
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Bleeding and Clotting Disorders Institute Peoria, Illinois
- Sponsor type
- University, hospital or other
- Collaborators
- Genentech, Inc.
- Responsible party
- Principal investigator, Jonathan Roberts, Bleeding and Clotting Disorders Institute Peoria, Illinois
- Investigators
- Jonathan C Roberts, MD, Principal investigator, Bleeding and Clotting Disorders Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05500807
- Sponsor's protocol ID
- BCDI-XII
- Acronym
- BCDI-XII
- Status last verified
- Mar 2026
- First submitted
- 12 Aug 2022
- Last updated
- 3 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
James PD, Connell NT, Ameer B, Di Paola J, Eikenboom J, Giraud N, Haberichter S, Jacobs-Pratt V, Konkle B, McLintock C, McRae S, R Montgomery R, O'Donnell JS, Scappe N, Sidonio R, Flood VH, Husainat N, Kalot MA, Mustafa RA. ASH ISTH NHF WFH 2021 guidelines on the diagnosis of von Willebrand disease. Blood Adv. 2021 Jan 12;5(1):280-300. doi: 10.1182/bloodadvances.2020003265.
PubMed 33570651Connell NT, Flood VH, Brignardello-Petersen R, Abdul-Kadir R, Arapshian A, Couper S, Grow JM, Kouides P, Laffan M, Lavin M, Leebeek FWG, O'Brien SH, Ozelo MC, Tosetto A, Weyand AC, James PD, Kalot MA, Husainat N, Mustafa RA. ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease. Blood Adv. 2021 Jan 12;5(1):301-325. doi: 10.1182/bloodadvances.2020003264.
PubMed 33570647Rodeghiero F, Castaman G, Dini E. Epidemiological investigation of the prevalence of von Willebrand's disease. Blood. 1987 Feb;69(2):454-9.
PubMed 3492222Werner EJ, Broxson EH, Tucker EL, Giroux DS, Shults J, Abshire TC. Prevalence of von Willebrand disease in children: a multiethnic study. J Pediatr. 1993 Dec;123(6):893-8. doi: 10.1016/s0022-3476(05)80384-1.
PubMed 8229521Weyand AC, Flood VH, Shavit JA, Pipe SW. Efficacy of emicizumab in a pediatric patient with type 3 von Willebrand disease and alloantibodies. Blood Adv. 2019 Sep 24;3(18):2748-2750. doi: 10.1182/bloodadvances.2019000656.
PubMed 31540901Roberts JC, Morateck PA, Christopherson PA, Yan K, Hoffmann RG, Gill JC, Montgomery RR; Zimmerman Program Investigators. Rapid discrimination of the phenotypic variants of von Willebrand disease. Blood. 2016 May 19;127(20):2472-80. doi: 10.1182/blood-2015-11-664680. Epub 2016 Feb 25.
PubMed 26917779Miller CH, Hilgartner MW, Harris MB, Bussel JB, Aledort LM. Concurrence of von Willebrand's disease and hemophilia A: implications for carrier detection and prevalence. Am J Med Genet. 1986 May;24(1):83-94. doi: 10.1002/ajmg.1320240110.
PubMed 3085499Casonato A, Pontara E, Boscaro M, Dannhauser D, Sartori MT, Girolami A. Combined haemophilia A and type I von Willebrand's disease: a family study including an evaluation of the effects of DDAVP infusion. Haematologia (Budap). 1993;25(1):57-67.
PubMed 8339998Kitazawa T, Igawa T, Sampei Z, Muto A, Kojima T, Soeda T, Yoshihashi K, Okuyama-Nishida Y, Saito H, Tsunoda H, Suzuki T, Adachi H, Miyazaki T, Ishii S, Kamata-Sakurai M, Iida T, Harada A, Esaki K, Funaki M, Moriyama C, Tanaka E, Kikuchi Y, Wakabayashi T, Wada M, Goto M, Toyoda T, Ueyama A, Suzuki S, Haraya K, Tachibana T, Kawabe Y, Shima M, Yoshioka A, Hattori K. A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model. Nat Med. 2012 Oct;18(10):1570-4. doi: 10.1038/nm.2942. Epub 2012 Sep 30.
PubMed 23023498Shima M, Hanabusa H, Taki M, Matsushita T, Sato T, Fukutake K, Fukazawa N, Yoneyama K, Yoshida H, Nogami K. Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A. N Engl J Med. 2016 May 26;374(21):2044-53. doi: 10.1056/NEJMoa1511769.
PubMed 27223146Uchida N, Sambe T, Yoneyama K, Fukazawa N, Kawanishi T, Kobayashi S, Shima M. A first-in-human phase 1 study of ACE910, a novel factor VIII-mimetic bispecific antibody, in healthy subjects. Blood. 2016 Mar 31;127(13):1633-41. doi: 10.1182/blood-2015-06-650226. Epub 2015 Dec 1.
PubMed 26626991Oldenburg J, Mahlangu JN, Kim B, Schmitt C, Callaghan MU, Young G, Santagostino E, Kruse-Jarres R, Negrier C, Kessler C, Valente N, Asikanius E, Levy GG, Windyga J, Shima M. Emicizumab Prophylaxis in Hemophilia A with Inhibitors. N Engl J Med. 2017 Aug 31;377(9):809-818. doi: 10.1056/NEJMoa1703068. Epub 2017 Jul 10.
PubMed 28691557Mahlangu J, Oldenburg J, Paz-Priel I, Negrier C, Niggli M, Mancuso ME, Schmitt C, Jimenez-Yuste V, Kempton C, Dhalluin C, Callaghan MU, Bujan W, Shima M, Adamkewicz JI, Asikanius E, Levy GG, Kruse-Jarres R. Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors. N Engl J Med. 2018 Aug 30;379(9):811-822. doi: 10.1056/NEJMoa1803550.
PubMed 30157389Levy GG, Asikanius E, Kuebler P, Benchikh El Fegoun S, Esbjerg S, Seremetis S. Safety analysis of rFVIIa with emicizumab dosing in congenital hemophilia A with inhibitors: Experience from the HAVEN clinical program. J Thromb Haemost. 2019 Sep;17(9):1470-1477. doi: 10.1111/jth.14491. Epub 2019 Jun 17.
PubMed 31124272Petrini P. Identifying and overcoming barriers to prophylaxis in the management of haemophilia. Haemophilia. 2007 Sep;13 Suppl 2:16-22. doi: 10.1111/j.1365-2516.2007.01501.x.
PubMed 17685919
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Emicizumab for Severe VON Willebrand Disease (VWD) and VWD/Hemophilia A
- ClinicalTrials.gov ID
- NCT05500807
- Acronym / sponsor ID
- BCDI-XII / BCDI-XII
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Bleeding and Clotting Disorders Institute Peoria, IllinoisUniversity, hospital or other
- Collaborators
- Genentech, Inc.
- Study start
- 1 Nov 2022 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Feb 2028 (estimated)
- First posted
- 15 Aug 2022 (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.
Von Willebrand Disease (VWD) is the most common inherited bleeding disorder affecting up to 0.1% of the population, is usually characterized by mucocutaneous bleeding, HMB, surgical bleeding or other hemostatic challenges. Severe bleeding events require VWF concentrates administered solely through intravenous access. Emicizumab (Hemlibra) is a monoclonal bispecific antibody developed to bind activated FIX and FX and mimic FVIII cofactor functionality. Hemlibra is administered via subcutaneous injection rather than intravenous infusion. The hypothesis of this study is that Emicizumab is safe and efficacious for prophylaxis in severe VWD and concomitant VWD/hemophilia patients.
Detailed description
Von Willebrand Disease (VWD) is the most common inherited bleeding disorder affecting up to 0.1% of the population, is usually characterized by mucocutaneous bleeding, HMB, surgical bleeding or other hemostatic challenges. VWD currently has few therapies generally useful in management of bleeding events including antifibrinolytics, desmopressin (DDAVP), and VWF concentrates, which may be plasma-derived (VWF with and without FVIII) or recombinant. Minor bleeding may be successfully treated with antifibrinolytics and DDAVP; however, more severe bleeding requires VWF concentrates that are administered solely through intravenous access.
Similarly, it can be challenging to treat concomitant bleeding disorders with the existing therapeutic options available, and patients with concurrent VWD and hemophilia A primarily have VWF/FVIII concentrate or desmopressin (DDAVP) available for treatment. It has been well-recognized that patients, caregivers, and medical providers desire additional, simplified therapeutic options that are not intravenous to treat severe bleeding disorders. Therefore, a simplified, subcutaneous therapeutic that prevents bleeding would be strongly desired. Though its use in the hemophilia A population is growing, additional potential emicizumab applications for hemostatic control in other hemostatic disorders remain unknown. A recent case report highlighted the hemostatic efficacy of emicizumab off-label use in type 3 von Willebrand Disease (VWD), another severe bleeding disorder. This pediatric patient had type 3 VWD with alloantibodies and a bleeding phenotype similar to hemophilia A with inhibitor patients, requiring suboptimal bleeding management with rFVIIa and activated prothrombin complex concentrates (aPCC). Emicizumab prophylaxis was initiated and the patient no longer required aPCC prophylaxis and rare use of rFVIIa for acute bleeding events (only 1 trauma-induced soft tissue hematoma at the time of publication). The authors concluded their report suggested the bleeding phenotype in type 3 VWD is expressed mainly due to factor VIII deficiency. This study suggests a potential additional application for emicizumab in severe VWD.
A pilot multicenter, prospective open-label study of emicizumab prophylaxis in severe VWD and concomitant VWD/hemophilia A. Patients will have a one-year retrospective chart review of annualized bleed rate and hemostatic therapies collected at the time of enrollment. Patients will then be treated with emicizumab with 3mg/kg weekly for 4 weeks loading dose, followed by once weekly prophylaxis of 1.5mg/kg for 1 year. Per emicizumab FDA-approved prescribing information for hemophilia A, dose up-titration to 3 mg/kg once weekly will be allowed after 24 weeks on HEMLIBRA prophylaxis in case of suboptimal efficacy (i.e., ≥ 2 spontaneous and clinically significant bleeds) Treatment records will be maintained along with bleeding event logs. Breakthrough bleeding events may be treated with the patients usual on-demand therapy with antifibrinolytics or VWF/FVIII concentrates per clinician discretion. Patient reported outcome assessments will be collected throughout the clinical study to collect impact of the treatment on the individual patients, assessing quality of life, physical, emotional, social and general symptoms.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- 40 patients who will receive emicizumab prophylactically
| Arm | Type | Receives |
|---|---|---|
| Open Label Emicizumab | Other | Drug: EmicizumabEmicizumab prophylaxis |
Who may qualify
- Ages
- 0 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Signed informed consent
- Age 0 and older (infants weighing ≥3 kg)
- ability to comply with protocol in investigators judgement
- diagnosis of: severe VWD type 3, or VWD with VWF antigen, activity or collagen binding </= 20 U/dl or variant VWD confirmed by genetic mutation and VWF ag, activity or CB < 50 U/dl based on historical medical records of study site.
- diagnosis of VWD/hemophilia A defined as VWF:ag, activity or CB <50 U/dl, and mild moderate or severe hemophilia A(defined by ISTH criteria) based on historical medical records of the study site.
Key exclusion criteria (16 listed)
- Patients and/or infants weighing < 3 kg.
- Patients with low VWF or non-severe VWD (ie.not meeting the above criteria)
- Other concomitant bleeding disorders including coagulopathy from liver cirrhosis.
- Current treatment with emicizumab or emicizumab therapy in the previous 18 months.
- Previous (in the past 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or current signs of thromboembolic disease
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 |
|---|---|---|
| EmicizumabAlso: HemlibraSubcutaneous injection of emicizumab for prophylaxis | Drug | Open Label Emicizumab |
Primary outcome
Emicizumab is efficacious for prophylaxis in severe VWD & concomitant VWD/hemophilia A
Time frame: 18 months
Establish bleed occurrence during treatment evaluated through descriptive statistical analysis to determine proof of principle
Emicizumab is safe for prophylaxis in severe VWD & concomitant VWD/Hemophilia A
Time frame: 18 months
Collection of AE's, hypersensitivity reactions, thrombotic events during treatment
3 secondary outcomes
Reduced treatment burden vs VWF/FVIII prophylaxis
Time frame: 18 months
Decreased bleed occurrence
Time frame: 12 months
Diminish bleed severity
Time frame: 18 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Apr 2026.
- Background: PubMed 33570651James PD, Connell NT, Ameer B, Di Paola J, Eikenboom J, Giraud N, Haberichter S, Jacobs-Pratt V, Konkle B, McLintock C, McRae S, R Montgomery R, O'Donnell JS, Scappe N, Sidonio R, Flood VH, Husainat N, Kalot MA, Mustafa RA. ASH ISTH NHF WFH 2021 guidelines on the diagnosis of von Willebrand disease. Blood Adv. 2021 Jan 12;5(1):280-300. doi: 10.1182/bloodadvances.2020003265.
- Background: PubMed 33570647Connell NT, Flood VH, Brignardello-Petersen R, Abdul-Kadir R, Arapshian A, Couper S, Grow JM, Kouides P, Laffan M, Lavin M, Leebeek FWG, O'Brien SH, Ozelo MC, Tosetto A, Weyand AC, James PD, Kalot MA, Husainat N, Mustafa RA. ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease. Blood Adv. 2021 Jan 12;5(1):301-325. doi: 10.1182/bloodadvances.2020003264.
- Background: PubMed 3492222Rodeghiero F, Castaman G, Dini E. Epidemiological investigation of the prevalence of von Willebrand's disease. Blood. 1987 Feb;69(2):454-9.
- Background: PubMed 8229521Werner EJ, Broxson EH, Tucker EL, Giroux DS, Shults J, Abshire TC. Prevalence of von Willebrand disease in children: a multiethnic study. J Pediatr. 1993 Dec;123(6):893-8. doi: 10.1016/s0022-3476(05)80384-1.
- Background: PubMed 31540901Weyand AC, Flood VH, Shavit JA, Pipe SW. Efficacy of emicizumab in a pediatric patient with type 3 von Willebrand disease and alloantibodies. Blood Adv. 2019 Sep 24;3(18):2748-2750. doi: 10.1182/bloodadvances.2019000656.
- Background: PubMed 26917779Roberts JC, Morateck PA, Christopherson PA, Yan K, Hoffmann RG, Gill JC, Montgomery RR; Zimmerman Program Investigators. Rapid discrimination of the phenotypic variants of von Willebrand disease. Blood. 2016 May 19;127(20):2472-80. doi: 10.1182/blood-2015-11-664680. Epub 2016 Feb 25.
- Background: PubMed 3085499Miller CH, Hilgartner MW, Harris MB, Bussel JB, Aledort LM. Concurrence of von Willebrand's disease and hemophilia A: implications for carrier detection and prevalence. Am J Med Genet. 1986 May;24(1):83-94. doi: 10.1002/ajmg.1320240110.
- Background: PubMed 8339998Casonato A, Pontara E, Boscaro M, Dannhauser D, Sartori MT, Girolami A. Combined haemophilia A and type I von Willebrand's disease: a family study including an evaluation of the effects of DDAVP infusion. Haematologia (Budap). 1993;25(1):57-67.
- Background: PubMed 23023498Kitazawa T, Igawa T, Sampei Z, Muto A, Kojima T, Soeda T, Yoshihashi K, Okuyama-Nishida Y, Saito H, Tsunoda H, Suzuki T, Adachi H, Miyazaki T, Ishii S, Kamata-Sakurai M, Iida T, Harada A, Esaki K, Funaki M, Moriyama C, Tanaka E, Kikuchi Y, Wakabayashi T, Wada M, Goto M, Toyoda T, Ueyama A, Suzuki S, Haraya K, Tachibana T, Kawabe Y, Shima M, Yoshioka A, Hattori K. A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model. Nat Med. 2012 Oct;18(10):1570-4. doi: 10.1038/nm.2942. Epub 2012 Sep 30.
- Background: PubMed 27223146Shima M, Hanabusa H, Taki M, Matsushita T, Sato T, Fukutake K, Fukazawa N, Yoneyama K, Yoshida H, Nogami K. Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A. N Engl J Med. 2016 May 26;374(21):2044-53. doi: 10.1056/NEJMoa1511769.
- Background: PubMed 26626991Uchida N, Sambe T, Yoneyama K, Fukazawa N, Kawanishi T, Kobayashi S, Shima M. A first-in-human phase 1 study of ACE910, a novel factor VIII-mimetic bispecific antibody, in healthy subjects. Blood. 2016 Mar 31;127(13):1633-41. doi: 10.1182/blood-2015-06-650226. Epub 2015 Dec 1.
- Background: PubMed 28691557Oldenburg J, Mahlangu JN, Kim B, Schmitt C, Callaghan MU, Young G, Santagostino E, Kruse-Jarres R, Negrier C, Kessler C, Valente N, Asikanius E, Levy GG, Windyga J, Shima M. Emicizumab Prophylaxis in Hemophilia A with Inhibitors. N Engl J Med. 2017 Aug 31;377(9):809-818. doi: 10.1056/NEJMoa1703068. Epub 2017 Jul 10.
- Individual participant dataSharing planned: No
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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.
Jonathan C Roberts, MD principal investigator
Bleeding and Clotting Disorders Institute
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Emicizumab in people with Von Willebrand Disease, Type 3 and Concomitant VWD and Hemophilia, run by Bleeding and Clotting Disorders Institute Peoria, Illinois at 12 sites in United States.
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 10 inclusion and 16 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: looking for participants now, as of the sponsor's last update on 3 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Megan L Woodbury, PhD (309-692-5337 ext. 174, meganw@ilbcdi.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Emicizumab is efficacious for prophylaxis in severe VWD & concomitant VWD/hemophilia A (time frame: 18 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05500807, which also lists 19 linked publications.
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 3 Apr 2026; AzTrials last refreshed its copy on 8 Nov 2026.