Emicizumab treatment for severe Von Willebrand Disease and Hemophilia A

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial investigates the safety and efficacy of Emicizumab, a monoclonal bispecific antibody, for the prophylactic treatment of severe Von Willebrand Disease (VWD) and patients with both VWD and Hemophilia A. The study aims to provide a simplified subcutaneous treatment option, as current therapies often require intravenous administration. Participants will include individuals diagnosed with severe VWD or VWD with Hemophilia A, who will receive Emicizumab to prevent severe bleeding events. The trial will assess the effectiveness of this treatment compared to existing therapies.

Why it matters

Who can join: the full rules

Inclusion 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 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
* 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.

Exclusion Criteria:

* 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
* 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

Where it's running

• The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders (CIBD)
  Orange, California, United States Recruiting
• Stanford University: Stanford Children's Health
  Redwood City, California, United States Recruiting
• University of Miami - Miller School of Medicine
  Coral Gables, Florida, United States Recruiting
• St. Joseph's Children's Hospital - Center for Bleeding and Clotting Disorders
  Tampa, Florida, United States Recruiting
• Bleeding and Clotting Disorders Institute (BCDI)
  Peoria, Illinois, United States Recruiting
• Innovative Hematology, Inc. (IHI)
  Indianapolis, Indiana, United States Recruiting

Study contacts

• Principal investigator: Jonathan C Roberts, MD — Bleeding and Clotting Disorders Institute
• Study coordinator: Megan L Woodbury, PhD
• Email: meganw@ilbcdi.org
• Phone: 309-692-5337

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Emicizumab prophylaxis for people with Type 3 von Willebrand disease

  This trial will test whether regular emicizumab injections reduce bleeds compared with on-demand standard therapy for people aged 1 month and older with Type 3 von Willebrand disease, while also studying people already on prophylaxis using their prior data.

  Recruiting at 29 sites Phase 3 Hoffmann-La Roche · Sacramento, California and 28 other locations · NCT06998524

• NXT007 versus emicizumab to prevent bleeds in people with Hemophilia A

  This will test whether NXT007 prevents bleeding better than emicizumab in people aged 12 and older with Hemophilia A, including those with or without factor VIII inhibitors.

  Recruiting at 63 sites Phase 3 Hoffmann-La Roche · Orange, California and 62 other locations · NCT07416604

• Switching from Emicizumab to Marstacimab in Severe Hemophilia A

  This study is testing if switching from emicizumab to marstacimab is safe and how well the body handles the new medication in people with severe hemophilia A.

  Recruiting at 7 sites Phase 1 Pfizer · La Jolla, California and 9 other locations · NCT06703606

• Testing fitusiran to prevent bleeds in severe hemophilia A patients switching from emicizumab

  This study is testing if fitusiran can help prevent bleeding in men with severe hemophilia A who are switching from another treatment called emicizumab.

  Recruiting at 3 sites Phase 4 Sanofi · Los Angeles, California and 2 other locations · NCT06145373

• Using Nuwiq to manage bleeding in hemophilia A patients during major surgery

  This study is testing if Nuwiq can help prevent bleeding during major surgery for boys and men over 12 with severe hemophilia A who are already using another treatment called emicizumab.

  Recruiting at 17 sites Phase 4 Octapharma · Dallas, Texas and 17 other locations · NCT05935358

• Understanding antibody changes in hemophilia A patients treated with emicizumab

  This study looks at how the immune system reacts to emicizumab treatment in people with severe hemophilia A by checking leftover samples from their regular doctor visits.

  Recruiting Goethe University · Frankfurt am Main, Hesse · NCT05802836

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.