- ICH GCP
- US Clinical Trials Registry
- Klinisk forsøg NCT07789587
A Study of Bometase Alfa for Bleeding Control in Acquired Hemophilia A
24. august 2026 opdateret af: Institute of Hematology & Blood Diseases Hospital, China
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.
Studieoversigt
Status
Ikke rekrutterer endnu
Betingelser
Intervention / Behandling
Undersøgelsestype
Interventionel
Tilmelding (Anslået)
20
Fase
- Ikke anvendelig
Kontakter og lokationer
Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.
Studiekontakt
- Navn: Wei Liu
- Telefonnummer: +8613820261971
- E-mail: liuwei1@ihcams.ac.cn
Undersøgelse Kontakt Backup
- Navn: Lei Zhang
- E-mail: zhanglei1@ihcams.ac.cn
Studiesteder
-
-
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Tianjin, Kina
- Chinese Academy of Medical Science and Blood Disease Hospital
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Kontakt:
- Wei Liu
- Telefonnummer: +8613820261971
- E-mail: liuwei1@ihcams.ac.cn
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-
Deltagelseskriterier
Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.
Berettigelseskriterier
Aldre berettiget til at studere
- Voksen
- Ældre voksen
Tager imod sunde frivillige
Ingen
Beskrivelse
Inclusion Criteria:
- Age ≥18 years;
Confirmed diagnosis of acquired hemophilia A, meeting the following criteria:
- Isolated prolongation of activated partial thromboplastin time (APTT) with a normal prothrombin time (PT);
- Reduced factor VIII coagulant activity (FVIII:C <50%);
- 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;
- 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.
Studieplan
Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
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Eksperimentel: 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.
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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.
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Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Tidsramme |
|---|---|
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Incidence of effective hemostasis rate at 8 hours after the first administration
Tidsramme: 8 hours
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8 hours
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Sekundære resultatmål
Resultatmål |
Tidsramme |
|---|---|
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Incidence of effective hemostasis rate at 12 hours after the first administration
Tidsramme: 12 hours
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12 hours
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Time to achieve clinical hemostasis
Tidsramme: 30 days
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30 days
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Amount of blood product use
Tidsramme: 30 days
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30 days
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Dose of Bometase Alfa administered
Tidsramme: 30 days
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30 days
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Rate of rescue therapy
Tidsramme: 30 days
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30 days
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Incidence of Treatment-Emergent Adverse Events (AES)
Tidsramme: AES was assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0.
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AES was assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0.
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Samarbejdspartnere og efterforskere
Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.
Efterforskere
- Ledende efterforsker: Lei Zhang, Chinese Academy of Medical Science and Blood Disease Hospital
Datoer for undersøgelser
Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.
Studer store datoer
Studiestart (Anslået)
1. august 2026
Primær færdiggørelse (Anslået)
1. oktober 2026
Studieafslutning (Anslået)
1. oktober 2026
Datoer for studieregistrering
Først indsendt
24. august 2026
Først indsendt, der opfyldte QC-kriterier
24. august 2026
Først opslået (Faktiske)
27. august 2026
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
27. august 2026
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
24. august 2026
Sidst verificeret
1. august 2026
Mere information
Begreber relateret til denne undersøgelse
Nøgleord
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- IIT2026087
Plan for individuelle deltagerdata (IPD)
Planlægger du at dele individuelle deltagerdata (IPD)?
JA
IPD-delingstidsramme
from 12 months to 36 months after study completion
IPD-delingsadgangskriterier
From corresponding author
IPD-deling Understøttende informationstype
- STUDY_PROTOCOL
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
Studerer et amerikansk FDA-reguleret lægemiddelprodukt
Ingen
Studerer et amerikansk FDA-reguleret enhedsprodukt
Ingen
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