RECRUITING

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

Conditions

Von Willebrand Disease, Type 3 Concomitant VWD and Hemophilia

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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. 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.

Official Title

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

Quick Facts

Study Start:2022-11-01

Study Completion:2026-03

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05500807

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:2 Years to 90 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* Signed informed consent * age \>/= 2 * 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 ono-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 mensturating 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).	* Patients age \<2 years of age. * 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: * 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

Inclusion Criteria

Exclusion Criteria

* Signed informed consent
* age \>/= 2
* 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 ono-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 mensturating 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).

* Patients age \<2 years of age.
* 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:
* 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

Contacts and Locations

Study Contact

Sarah E Gonzales, MD

CONTACT

309-692-5337

sarah@ilbcdi.org

Lisa Weber, BS

CONTACT

309-392-5337

lisaw@ilbcdi.org

Principal Investigator

Jonathan C Roberts, MD

PRINCIPAL_INVESTIGATOR

Bleeding and Clotting Disorders Institute

Study Locations (Sites)

Bleeding and Clotting Disorders Institute

Peoria, Illinois, 61614

United States

Collaborators and Investigators

Sponsor: Bleeding and Clotting Disorders Institute Peoria, Illinois

Jonathan C Roberts, MD, PRINCIPAL_INVESTIGATOR, Bleeding and Clotting Disorders Institute

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-11-01

Study Completion Date2026-03

Study Record Updates

Study Start Date2022-11-01

Study Completion Date2026-03

Terms related to this study

Additional Relevant MeSH Terms

Von Willebrand Disease, Type 3
Concomitant VWD and Hemophilia