Clinical trial NCT05823376: Avatrombopag for Platelet Recovery Post-UCBT in Patients With Bone Marrow Failure Disease
Official title: 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
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Platelet Recovery After Umbilical Cord Blood Transplantation
Keywords: Avatrobopag
Lead sponsor: Anhui Provincial Hospital
Sponsor class: OTHER_GOV
First posted: 2023-04-21
Enrollment: 40
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients diagnosed with bone marrow failure and receiving umbilical cord blood transplantation

Brief 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

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* 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 Criteria:

* 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).

Primary outcomes:
measure: Time of platelet engraftment
timeFrame: 28 days
measure: The cumulative incidence of platelet engraftment at 28 days after transplantation
timeFrame: 28 days
Secondary outcomes:
measure: The cumulative incidence of platelet recovery rate at 28 days after transplantation
timeFrame: 28 days
measure: Total platelet transfusion during 4 weeks of treatment
timeFrame: 28 days
measure: neutrophil reconstitution
timeFrame: 28 days
measure: erythroid reconstitution
timeFrame: 28 days
measure: Megakaryocyte levels in bone marrow smears 4 weeks after treatment
timeFrame: 4 weeks
measure: Time of platelet recovery
timeFrame: 100 days
measure: Time of platelet ≥100×10^9/L.
timeFrame: 100 days
Other outcomes:
Locations:
city: Hefei
country: China
facility: The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
state: Anhui
Study officials:
affiliation: The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
name: Xiaoyu Zhu, ph.D
role: PRINCIPAL_INVESTIGATOR
References: