ACTIVE_NOT_RECRUITING

Emicizumab in Patients With Acquired Hemophilia A

Conditions

Acquired Hemophilia A Acquire Hemophilia A emicizumab AHA

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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

This is a phase II multicenter open-label, single-arm prospective study to evaluate the efficacy of prophylactic emicizumab administered on a scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).

Official Title

Emicizumab in Patients With Acquired Hemophilia A: Multicenter, Single-arm, Open-label Clinical Trial

Quick Facts

Study Start:2022-08-31

Study Completion:2026-12

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT05345197

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:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* Signed Informed Consent/Assent Form * Age ≥18 years at time of signing Informed Consent Form * Ability to comply with the study protocol, in the investigator's judgment * Diagnosis of AHA based on a reduced FVIII activity (\<50 %) and positive FVIII inhibitor (\>0.6 BU/ml) at screening (local laboratory) * Current bleeding due to AHA at the time of screening * Plan to be adherent to emicizumab prophylaxis during the study * For women of childbearing potential who meet the following criteria: * Refrain from heterosexual intercourse or use contraceptive methods that result in a failure rate of \<1% per year during the study period A woman with ≥ 12 continuous months of amenorrhea with no identified cause other than menopause and has not undergone surgical sterilization (removal of ovaries and/or uterus). use of combined oral or injected hormonal contraceptive, bilateral tubal ligation, male sterilization, hormone- releasing intrauterine devices, and copper intrauterine devices.	* Congenital hemophilia A * Treatment with aPCC within the last 24 hours before first study treatment or planned treatment with aPCC during the course of the study * Known positive lupus anticoagulant at the time of screening * Severe uncontrolled infection at the time of screening * Signs of active disseminated intravascular coagulation at the time of screening - * Emicizumab ⎯ AHA Emi Version 1.0 20 * Current treatment for thromboembolic disease or signs of current thromboembolic disease at time of screening * Patients who are at high risk for TMA (e.g., have a previous medical or family history of TMA), in the investigator's judgment * Known severe congenital or acquired thrombophilia * Life expectancy \<3 months at the time of screening * Other conditions that substantially increase risk of bleeding or thrombosis by the discretion of the investigator * Contraindications according to the Investigator's Brochure of emicizumab * Current treatment with emicizumab at time of screening * History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection by the discretion of the investigator * Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose additional risk, or would, in the opinion of the local investigator, preclude the patient's safe participation in and completion of the study * Addiction or other diseases that preclude the patient from appropriately assessing the nature and scope as well as possible consequences of the clinical study by the discretion of the investigator * Pregnant or breast-feeding women * Would refuse treatment with blood or blood products, if necessary. * Subject is in custody by order of an authority or a court of law * 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 half-lives- 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

Inclusion Criteria

Exclusion Criteria

* Signed Informed Consent/Assent Form
* Age ≥18 years at time of signing Informed Consent Form
* Ability to comply with the study protocol, in the investigator's judgment
* Diagnosis of AHA based on a reduced FVIII activity (\<50 %) and positive FVIII inhibitor (\>0.6 BU/ml) at screening (local laboratory)
* Current bleeding due to AHA at the time of screening
* Plan to be adherent to emicizumab prophylaxis during the study
* For women of childbearing potential who meet the following criteria:
* Refrain from heterosexual intercourse or use contraceptive methods that result in a failure rate of \<1% per year during the study period A woman with ≥ 12 continuous months of amenorrhea with no identified cause other than menopause and has not undergone surgical sterilization (removal of ovaries and/or uterus). use of combined oral or injected hormonal contraceptive, bilateral tubal ligation, male sterilization, hormone- releasing intrauterine devices, and copper intrauterine devices.

* Congenital hemophilia A
* Treatment with aPCC within the last 24 hours before first study treatment or planned treatment with aPCC during the course of the study
* Known positive lupus anticoagulant at the time of screening
* Severe uncontrolled infection at the time of screening
* Signs of active disseminated intravascular coagulation at the time of screening -
* Emicizumab ⎯ AHA Emi Version 1.0 20
* Current treatment for thromboembolic disease or signs of current thromboembolic disease at time of screening
* Patients who are at high risk for TMA (e.g., have a previous medical or family history of TMA), in the investigator's judgment
* Known severe congenital or acquired thrombophilia
* Life expectancy \<3 months at the time of screening
* Other conditions that substantially increase risk of bleeding or thrombosis by the discretion of the investigator
* Contraindications according to the Investigator's Brochure of emicizumab
* Current treatment with emicizumab at time of screening
* History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection by the discretion of the investigator
* Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose additional risk, or would, in the opinion of the local investigator, preclude the patient's safe participation in and completion of the study
* Addiction or other diseases that preclude the patient from appropriately assessing the nature and scope as well as possible consequences of the clinical study by the discretion of the investigator
* Pregnant or breast-feeding women
* Would refuse treatment with blood or blood products, if necessary.
* Subject is in custody by order of an authority or a court of law
* 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 half-lives- 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

Contacts and Locations

Principal Investigator

Rebecca Kruse-Jarres, MD, MPH

PRINCIPAL_INVESTIGATOR

University of Washington

Study Locations (Sites)

UCSD Hemophilia and Thrombosis Treatment Center

San Diego, California, 92121

United States

Georgetown University

Washington D.C., District of Columbia, 20007

United States

Emory University

Atlanta, Georgia, 30308

United States

Bleeding and Clotting Disorders Institute

Peoria, Illinois, 61614

United States

Indiana Hemophilia and Thrombosis Center, Inc.

Indianapolis, Indiana, 46260

United States

Tulane University

New Orleans, Louisiana, 70112-2632

United States

Mayo Clinic

Rochester, Minnesota, 55905

United States

Washington University

St Louis, Missouri, 63110

United States

University of North Carolina

Chapel Hill, North Carolina, 27514

United States

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104

United States

Penn Blood Disorders Program, Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104

United States

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, 15213

United States

University of Vermont Medical Center

Burlington, Vermont, 05401

United States

UVA Comprehensive Cancer Center

Charlottesville, Virginia, 22908

United States

Washington Center for Bleeding Disorders

Seattle, Washington, 98101

United States

Versiti Inc.

Milwaukee, Wisconsin, 53226

United States

Collaborators and Investigators

Sponsor: University of Washington

Rebecca Kruse-Jarres, MD, MPH, PRINCIPAL_INVESTIGATOR, University of Washington

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 Date2022-08-31

Study Completion Date2026-12

Study Record Updates

Study Start Date2022-08-31

Study Completion Date2026-12

Terms related to this study

Keywords Provided by Researchers

Acquire Hemophilia A
emicizumab
AHA

Additional Relevant MeSH Terms

Acquired Hemophilia A