Avatrombopag for the Treatment of Thrombocytopenia After Donor Hematopoietic Stem Cell Transplant

A Phase II Trial of Avatrombopag for the Treatment of Thrombocytopenia After Allogeneic Hematopoietic Stem Cell Transplant

NCT04312789•Ayman Saad

View on ClinicalTrials.gov

Summary

This phase II trial studies the side effects and how well avatrombopag works for the treatment of thrombocytopenia after donor hematopoietic stem cell transplant. Thrombocytopenia is defined as abnormally low level of platelets in the blood. Avatrombopag is a small molecule thrombopoietin receptor agonist which stimulates thrombopoietin receptor leading to increase production of platelets.

Detailed Description

PRIMARY OBJECTIVE: I. To determine the safety and efficacy of avatrombopag for the treatment of thrombocytopenia after allogenic hematopoietic stem cell transplantation. SECONDARY OBJECTIVE: I. To identify predictors of response to avatrombopag. OUTLINE: Patients receive avatrombopag orally (PO) once daily (QD) for up to 1 year in the absence of disease progression or unacceptable toxicity. Avatrombopag will be titrated weekly until platelet count of greater than or equal to 60,000/uL is achieved and persists for 7 consecutive days, and the patient remains free from platelet transfusion. After completion of study treatment, patients are followed up weekly for 4 weeks and then monthly up to 1 year.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Failure to engraft platelets by day 30 (D30) after hematopoietic cell transplantation (HCT) defined as: Platelet count less than 20,000/uL and patient is still dependent on platelet transfusion support
•Patient must be able to start treatment with avatrombopag within 30-60 days following transplant
•Able to provide written informed consent from patient or legal representative

Exclusion Criteria

•Serious uncontrolled infections
•Steroid refractory graft versus host disease (GVHD)
•Patients with thrombotic microangiopathy
•Pregnant or lactating women
•Creatinine clearance \< 30 ml/min
•Active thromboembolism requiring anticoagulation
•Unable to understand the investigational nature of the study or provide informed consent
•Evidence of disease relapse by flow cytometry of chimerisms
•Concomitant use of other thrombopoietin receptor agonists (TPO-RA) medication during the treatment phase of the study or two weeks prior to enrollment

Key Dates

Start Date

December 1, 2022

Primary Completion Date

December 31, 2022

Completion Date

December 31, 2022

Last Updated

December 9, 2021

Conditions

Platelet Disorder

Interventions

Avatrombopag

DRUG

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: December 9, 2021Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

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Avatrombopag for the Treatment of Thrombocytopenia After Donor Hematopoietic Stem Cell Transplant

Inclusion Criteria

Exclusion Criteria

Protocol Title: Safety and Feasibility of Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis in Participants With RUNX1 Familial Platelet Disorder

Low-Dose Sirolimus to Increase Hematopoietic Function in Patients With RUNX1 Familial Platelet Disorder

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