Acquired Hemophilia A
Conditions
Keywords
Acquired Hemophilia A, Bometase Alfa
Brief summary
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.
Interventions
DRUGBometase 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.
Sponsors
Institute of Hematology & Blood Diseases Hospital, China
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of effective hemostasis rate at 8 hours after the first administration | 8 hours |
Secondary
| 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. |
Countries
China
Contacts
CONTACTWei Liu
CONTACTLei Zhang
PRINCIPAL_INVESTIGATORLei Zhang
Chinese Academy of Medical Science and Blood Disease Hospital
Outcome results
None listed