Emicizumab in Patients With Acquired Hemophilia A

NCT05345197 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: STUDY00013920
• Study Type: interventional
• Target: 51
• Locations: 1 country
• Sites: 16

Brief Summary

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).

Conditions

Acquired Hemophilia A

Keywords

Acquire Hemophilia A; emicizumab; AHA

Outcome Measures

Primary Outcomes (1)

• Primary Outcome Meassure

  Number of clinically significant bleeds after 12 weeks of study drug (emicizumab)

  12 weeks

Secondary Outcomes (5)

• Incidence and severity of adverse events

  duration of entire study

• Days of treatment with additional hemostatic agent

  12 weeks

• Remission Rate

  1 to 24 weeks

• Hospitalizations

  12 weeks

• Total dose of additional hemostatic agent

  12 weeks

Other Outcomes (2)

• Comparison of historic GTH-AH 01/2020 cohort/ Number of clinically significant bleeds

  12 to 24 weeks

• Comparison to a parallel German study cohort/ Number of clinically significant bleeds

  12 to 24 weeks

Study Arms (1)

Experimental-treatment

EXPERIMENTAL

Treatment with emicizumab

Drug: emicizumab

Interventions

emicizumab DRUG

This study design uses emicizumab as a prophylaxis treatment to prevent bleeding for all participants, bypassing agents (with the exception of aPCC) and treatment of concomitant diseases will be given as clinically indicated. All eligible subjects with AHA will receive the same study medication consisting of: two loading doses of the emicizumab on day 1 and 2 followed by once weekly subcutaneous emicizumab injections. Immunosuppressive therapy (IST) will be given concurrently as per investigator discretion.

Also known as: Hemlibra (R)

Experimental-treatment

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Washington: lead
• Genentech, Inc.: collaborator

Study Sites (16)

UCSD Hemophilia and Thrombosis Treatment Center

San Diego, California, 92121, United States

Location

Georgetown University

Washington D.C., District of Columbia, 20007, United States

Location

Emory University

Atlanta, Georgia, 30308, United States

Location

Bleeding and Clotting Disorders Institute

Peoria, Illinois, 61614, United States

Location

Indiana Hemophilia and Thrombosis Center, Inc.

Indianapolis, Indiana, 46260, United States

Location

Tulane University

New Orleans, Louisiana, 70112-2632, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

University of North Carolina

Chapel Hill, North Carolina, 27514, United States

Location

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States

Location

Penn Blood Disorders Program, Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, 15213, United States

Location

University of Vermont Medical Center

Burlington, Vermont, 05401, United States

Location

UVA Comprehensive Cancer Center

Charlottesville, Virginia, 22908, United States

Location

Washington Center for Bleeding Disorders

Seattle, Washington, 98101, United States

Location

Versiti Inc.

Milwaukee, Wisconsin, 53226, United States

Location

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MeSH Terms

Conditions

Factor 8 deficiency, acquired

Interventions

emicizumab

Study Officials

• Rebecca Kruse-Jarres, MD, MPH

  University of Washington

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor, School of Medicine: Hematology

Study Record Dates

• First Submitted: April 18, 2022
• First Posted: April 25, 2022
• Study Start: August 31, 2022
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: November 5, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: Data will be shared with the research group of the parallel European study (NCT04188639) at the end of the study and will be available for 2 years.
• Access Criteria: Only researchers of this or the parallel trial (NCT04188639) will have access to this data.

Locations

US (16)