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

Официальное название

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

Обзор

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

Подробное описание

Persistent thrombocytopenia is a common complication after umbilical cord blood transplantation (UCBT), which necessitates platelet transfusion and leads to increased transplant-related mortality. This is a single-center, single-arm, phase II clinical trial aimed at evaluating the efficacy and safety of Avatrombopag for platelet recovery after UCBT in patients with bone marrow failure disease (BMFD). The study was conducted in patients diagnosed with BFMD and receiving UCBT. After the subjects signed and informed and qualified, they received Avatrombopag treatment from the first day to the 28th day after transplantation. In this study, the cumulative of platelet implantation rate at 28 days after transplantation was used as the main therapeutic index, and 40 patients were planned to be enrolled.

Вмешательства

• Препарат 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.

Первичные конечные точки

• Time of platelet engraftment [Срок оценки: 28 days]
• The cumulative incidence of platelet engraftment at 28 days after transplantation [Срок оценки: 28 days]

Вторичные конечные точки (7)

• The cumulative incidence of platelet recovery rate at 28 days after transplantation [Срок оценки: 28 days]
• Total platelet transfusion during 4 weeks of treatment [Срок оценки: 28 days]
• neutrophil reconstitution [Срок оценки: 28 days]
• erythroid reconstitution [Срок оценки: 28 days]
• Megakaryocyte levels in bone marrow smears 4 weeks after treatment [Срок оценки: 4 weeks]
• Time of platelet recovery [Срок оценки: 100 days]
• Time of platelet ≥100×10^9/L. [Срок оценки: 100 days]

Критерии участия

Критерии включения

• 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；

Критерии исключения

• 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).

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Китай · 1 центр

• The First Affiliated Hospital of University of Science and Technology of China (Anhui Prov — Хэфэй

Идентификаторы

NCT: NCT05823376 · AVA&MDS-001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗