Avatrombopag for Platelet Recovery Post-UCBT in Patients With Bone Marrow Failure Disease
NCT ID: NCT05823376
Last Updated: 2024-12-31
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
40 participants
OBSERVATIONAL
2023-05-04
2025-02-28
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Avatrombopag group
The duration of the study is 4 weeks, from +1 day to +28 days after UCBT, patients will be given Avatrombopag with a recommended dose of 40mg per day. After 28 days of administration, the patients may continue, change or discontinue medication based on the patients' platelet status.
Avatrombopag
Indications for drug reduction and withdrawal: excluding blood transfusion factors, the reduction will be 20mg/d when PLT≥50×10\^9/L; Drug administration will be stopped when PLT≥100×10\^9/L.
Interventions
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Avatrombopag
Indications for drug reduction and withdrawal: excluding blood transfusion factors, the reduction will be 20mg/d when PLT≥50×10\^9/L; Drug administration will be stopped when PLT≥100×10\^9/L.
Eligibility Criteria
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Inclusion Criteria
* Patients diagnosed with bone marrow failure disease including aplastic anemia (AA), Fanconi anemia (FA), paroxysmal nocturnal hemoglobinuria (PNH) and undergoing UCBT;
* ECOG score ≤2;
* Voluntary participation in this clinical trial, patients fully understand the trial content and sign the informed consent；
Exclusion Criteria
* Known allergy to Avatrombopag;
* A history of severe thrombotic events or known risk factors for thrombosis or active thromboembolism requiring anticoagulation;
* A history of platelet dysfunction or bleeding prone disease or severe bleeding (requiring more than 2 units of red blood cell infusion or a hematocrit drop of ≥10%) within 7 days prior to screening;
* Chronic active hepatitis B and C;
* Repeat or multiple transplantation or multiple organ transplantation;
* HIV positive, EBV-DNA positive, CMV-DNA positive;
* Have severe infection or complicated with serious heart, liver, lung, kidney, neurological or metabolic diseases;
* A serious adverse event (Level 4 or 5) as defined by the Standard General Terminology for Adverse Events Version 4.0 occurred during the pre-treatment period;
* Participants participate in another clinical study with any exploratory drug or device within 30 days prior to baseline visit; Observational studies are allowed;
* Subjects with cognitive impairment or uncontrolled mental illness;
* Subjects and/or authorized family members to refuse treatment with Avatrombopag;
* Those who are judged by the researchers as not suitable for inclusion (such as accompanying medical history, which may affect the safety of the subjects or it is estimated that the treatment cannot be adhered to due to financial problems).
12 Years
ALL
No
Sponsors
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Anhui Provincial Hospital
OTHER_GOV
Responsible Party
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Xiaoyu Zhu
Chief physician
Principal Investigators
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Xiaoyu Zhu, ph.D
Role: PRINCIPAL_INVESTIGATOR
The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
Locations
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The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
Hefei, Anhui, China
Countries
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Central Contacts
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Facility Contacts
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Xiaoyu Zhu, ph.D
Role: primary
Aijie Huang, M.D
Role: backup
Other Identifiers
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AVA&MDS-001
Identifier Type: -
Identifier Source: org_study_id