RecruitingNCT05823376

Avatrombopag for Platelet Recovery Post-UCBT in Patients With Bone Marrow Failure Disease

Efficacy and Safety of Avatrombopag for Platelet Recovery After Unrelated Cord Blood Transplantation in Patients With Bone Marrow Failure Disease: a Single-center, Single-arm, Phase II Trial


Sponsor

Anhui Provincial Hospital

Enrollment

40 participants

Start Date

May 4, 2023

Study Type

OBSERVATIONAL

Conditions

Summary

To evaluate the efficacy and safety of Avatrombopag for platelet recovery after unrelated cord blood transplantation (UCBT) in patients with bone marrow failure disease


Eligibility

Min Age: 12 Years

Inclusion Criteria4

  • Age ≥12 years old, male or female;
  • 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 Criteria13

  • Pregnant or lactating women;
  • 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).

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Interventions

DRUGAvatrombopag

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.


Locations(1)

The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)

Hefei, Anhui, China

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NCT05823376


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