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New treatment option for Von Willebrand Disease, Type 3
Official title Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A
ClinicalTrials.gov ID: NCT05500807
What this study is testing
What is Emicizumab?
Emicizumab is an investigational medicine, given as an once-weekly injection under the skin, being studied as a potential treatment for von willebrand disease, type 3.
Also referred to as Hemlibra.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 0 to 90
You may be able to join if
- 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...
- 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...
You likely can't join if
- 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...
- Other conditions (e.g., certain autoimmune diseases, including, but not limited to diseases such as systemic lupus erythematosus, inflammatory bowel...
See the full eligibility criteria
- 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...
- 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
- 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...
- 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...
- 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...
- 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...
- 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...
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Orange, California, United States
- Redwood City, California, United States
- Coral Gables, Florida, United States
- Tampa, Florida, United States
- Peoria, Illinois, United States
- Indianapolis, Indiana, United States
- Ann Arbor, Michigan, United States
- Mount Pleasant, Michigan, United States
- Kansas City, Missouri, United States
- Hershey, Pennsylvania, United States
- Seattle, Washington, United States
- Madison, Wisconsin, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0 years to 90 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Orange, California, United States; Redwood City, California, United States; Coral Gables, Florida, United States; Tampa, Florida, United States; Peoria, Illinois, United States; Indianapolis, Indiana, United States and 6 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.