A Study of Bometase Alfa for Bleeding Control in Acquired Hemophilia A

Efficacy and Safety of Bometase Alfa for the Treatment of Bleeding in Acquired Hemophilia A: A Prospective, Single-Arm, Exploratory Study

This is a single-center, prospective, single-arm, exploratory study designed to evaluate the efficacy and safety of Bometase Alfa for the on-demand treatment of bleeding episodes in patients with acquired hemophilia A. A total of 20 patients with acquired hemophilia A experiencing bleeding events will be enrolled. Bometase Alfa will be administered at 0.1 U/kg for non-severe bleeding and 0.16 U/kg for severe bleeding, with consecutive doses given at 4-hour intervals until hemostasis is achieved. Treatment will be discontinued once hemostasis is achieved or if symptoms suggestive of arterial thrombosis occur, followed by a safety assessment. Rescue therapy will be initiated if bleeding remains uncontrolled after three consecutive administrations for a single bleeding episode, or if bleeding continues to worsen during treatment and the investigator determines that further treatment with Bometase Alfa is unlikely to provide clinical benefit and may pose a medical risk.

Study Overview

• Status: Not yet recruiting
• Conditions: Acquired Hemophilia A
• Intervention / Treatment: Drug: Bometase Alfa

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Wei Liu; Phone Number: +8613820261971; Email: liuwei1@ihcams.ac.cn
• Study Contact Backup: Name: Lei Zhang; Email: zhanglei1@ihcams.ac.cn

Study Locations

• China
  • Tianjin, China
    • Chinese Academy of Medical Science and Blood Disease Hospital
    • Contact: Wei Liu; Phone Number: +8613820261971; Email: liuwei1@ihcams.ac.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Age ≥18 years;

• Confirmed diagnosis of acquired hemophilia A, meeting the following criteria:

  1. Isolated prolongation of activated partial thromboplastin time (APTT) with a normal prothrombin time (PT);
  2. Reduced factor VIII coagulant activity (FVIII:C <50%);
  3. Positive FVIII inhibitor, defined as ≥0.6 BU/mL as measured by the Bethesda assay or Nijmegen-modified Bethesda assay, or failure of a 1:1 mixing study with normal plasma to achieve complete correction;
  4. No evidence of congenital hemophilia, von Willebrand disease, or lupus anticoagulant;
• Presence of clinically significant active bleeding, including, but not limited to, muscle or subcutaneous hematoma, gastrointestinal or genitourinary bleeding, postpartum or postoperative bleeding, or deep-seated or life-threatening organ bleeding;
• Provision of written informed consent by the patient and/or a legally authorized representative;
• Ability to comply with the study follow-up schedule for at least 30 da

Exclusion Criteria:

• Congenital hemophilia A or B, or any other confirmed congenital coagulation factor deficiency;
• Isolated prolonged activated partial thromboplastin time (APTT) due to lupus anticoagulant or antiphospholipid syndrome, with a negative FVIII inhibitor and normal FVIII activity; or coagulation abnormalities caused by disseminated intravascular coagulation (DIC) or severe liver disease that do not fulfill the diagnostic criteria for acquired hemophilia A;
• A history or symptoms of any arterial or venous thromboembolic event within 3 months before enrollment, including atherosclerosis, myocardial infarction, ischemic stroke, transient ischemic attack, deep vein thrombosis, or pulmonary embolism, or the presence of DIC;
• Use of factor VII (FVII), activated factor VII (FVIIa), tranexamic acid, or aminocaproic acid within 1 day before the planned administration of the study drug; or use of prothrombin complex concentrate (PCC) or factor VIII (FVIII) within 3 days before the first administration;
• Female participants who are pregnant or breastfeeding, or have a positive pregnancy test;
• Known hypersensitivity to the investigational product or any of its excipients;
• Inability to obtain informed consent, including patients unable to express their wishes and without a legally authorized representative;
• Inability to comply with the study follow-up requirements or investigator-determined poor compliance;
• Any other condition for which the investigator considers the participant unsuitable for study participation.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Acquired hemophilia A receiving Bometase Alfa for bleeding control; Bometase Alfa will be administered at 0.1 U/kg for non-severe bleeding or up to 0.16 U/kg for severe bleeding, with repeated doses given at 4-hour intervals until hemostasis is achieved.

Drug: Bometase Alfa; For non-severe bleeding, Bometase Alfa will be administered at a dose of 0.1 U/kg, while patients with severe bleeding will receive 0.16 U/kg. The study drug will be administered consecutively at 4-hour intervals until hemostasis is achieved. Treatment will be discontinued once hemostasis is achieved or if symptoms suggestive of arterial thrombosis occur, after which the patient will enter the safety assessment process.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of effective hemostasis rate at 8 hours after the first administration	8 hours

Secondary Outcome Measures

Outcome Measure	Time Frame
Incidence of effective hemostasis rate at 12 hours after the first administration	12 hours
Time to achieve clinical hemostasis	30 days
Amount of blood product use	30 days
Dose of Bometase Alfa administered	30 days
Rate of rescue therapy	30 days
Incidence of Treatment-Emergent Adverse Events (AES)	AES was assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0.

Collaborators and Investigators

• Sponsor: Institute of Hematology & Blood Diseases Hospital, China
• Investigators: Principal Investigator: Lei Zhang, Chinese Academy of Medical Science and Blood Disease Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): August 1, 2026
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): October 1, 2026

Study Registration Dates

• First Submitted: August 24, 2026
• First Submitted That Met QC Criteria: August 24, 2026
• First Posted (Actual): August 27, 2026

Study Record Updates

• Last Update Posted (Actual): August 27, 2026
• Last Update Submitted That Met QC Criteria: August 24, 2026
• Last Verified: August 1, 2026

More Information

Terms related to this study

• Keywords: Acquired Hemophilia A; Bometase Alfa
• Additional Relevant MeSH Terms: Factor 8 deficiency, acquired
• Other Study ID Numbers: IIT2026087

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Sharing Time Frame: from 12 months to 36 months after study completion
• IPD Sharing Access Criteria: From corresponding author
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No