Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A

Part of paid clinical trials in Orange, California.

Sponsor
Bleeding and Clotting Disorders Institute Peoria, Illinois
Study ID
NCT05500807
Phase
PHASE1
Status
Recruiting
Apply to this trial

Conditions

  • Concomitant VWD and Hemophilia
  • Von Willebrand Disease, Type 3

Eligibility Criteria

Sex
ALL
Age
0 Years - 90 Years
Healthy Volunteers
Not accepted

Interventions

  • Emicizumab — DRUG
    Subcutaneous injection of emicizumab for prophylaxis

Study Details

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.

Key Dates

Start date
Nov 1, 2022
Status verified
Mar 2026
Primary completion
Jun 30, 2027
Completion
Feb 29, 2028

Study Design

Enrollment
40 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Other: Open Label Emicizumab
    Emicizumab prophylaxis

Primary Outcome Measure

Emicizumab is efficacious for prophylaxis in severe VWD & concomitant VWD/hemophilia A [ Time Frame: 18 months ]

Central Contacts

Locations (12)

FacilityCityStateZIPSite coordinators
The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders (CIBD)OrangeCalifornia92868
Alyssa Barron
[email protected]
Nicole Crook
[email protected]
Vanessa Salinas, MD (PRINCIPAL_INVESTIGATOR)
Stanford University: Stanford Children's HealthRedwood CityCalifornia94063
Stephanie Gam
[email protected]
Riko Lee
[email protected]
May Chien, MD (PRINCIPAL_INVESTIGATOR)
University of Miami - Miller School of MedicineCoral GablesFlorida33146
Leandro Pisani
[email protected]
Fernando Francisco Corrales-Medina, MD (PRINCIPAL_INVESTIGATOR)
St. Joseph's Children's Hospital - Center for Bleeding and Clotting DisordersTampaFlorida33607
Denise Cardwell
[email protected]
Erin M Cockrell, DO (PRINCIPAL_INVESTIGATOR)
Bleeding and Clotting Disorders Institute (BCDI)PeoriaIllinois61614
Dayna Lenski, BS
[email protected]
309-692-5337
Sara Malik, MPH
[email protected]
309-692-5337
Jonathan C Roberts, MD (PRINCIPAL_INVESTIGATOR)
Michael D Tarantino, MD (SUB_INVESTIGATOR)
Innovative Hematology, Inc. (IHI)IndianapolisIndiana46260
Shannon Mears
[email protected]
Young Chong
[email protected]
Sweta L Gupta, MD (PRINCIPAL_INVESTIGATOR)
University of Michigan Medical SchoolAnn ArborMichigan48109
Jordan Kindshoven
[email protected]
Adam Comstock
[email protected]
Angela Weyand, MD (PRINCIPAL_INVESTIGATOR)
Central Michigan University: Children's Hospital of MichiganMount PleasantMichigan48859
Negin Salehi
[email protected]
Meera Chitlur, MD (PRINCIPAL_INVESTIGATOR)
Children's Mercy HospitalKansas CityMissouri64108
Anna Wisemann
[email protected]
Shannon L Carpenter, MD (PRINCIPAL_INVESTIGATOR)
Penn State College of MedicineHersheyPennsylvania17033
Erica Miller
[email protected]
Brennen Hartig
[email protected]
Huseyin Erdemir, MD (PRINCIPAL_INVESTIGATOR)
Washington Center for Bleeding DisordersSeattleWashington98101
Sophia Beyer
[email protected]
Laura Masserman
[email protected]
Rebecca Kruse-Jarres, MD, MPH (PRINCIPAL_INVESTIGATOR)
UW Health Comprehensive Program for Bleeding DisordersMadisonWisconsin53792
Hannah Ranous
[email protected]
Alec J Anderson
[email protected]
John Sheehan, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Orange, CA

By research site
The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders (CIBD)· Orange, CAStanford University: Stanford Children's Health· Redwood City, CAUniversity of Miami - Miller School of Medicine· Coral Gables, FLSt. Joseph's Children's Hospital - Center for Bleeding and Clotting Disorders· Tampa, FLBleeding and Clotting Disorders Institute (BCDI)· Peoria, ILInnovative Hematology, Inc. (IHI)· Indianapolis, IN

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