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

Platelet Disorder Clinical Trial

Official title:

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT04312789
• Other study ID #: OSU-19328
• Secondary ID: NCI-2020-01035
• Status: Withdrawn
• Phase: Phase 2
• Start date: December 2022
• Est. completion date: December 31, 2022

Study information

• Verified date: November 2021
• Source: Ohio State University Comprehensive Cancer Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: December 31, 2022
• Est. primary completion date: December 31, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Blood Platelet Disorders
• Platelet Disorder
• Thrombocytopenia

Intervention

Drug:
• Avatrombopag
Given PO

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Ayman Saad	Dova Pharmaceuticals

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of adverse events of avatrombopag treatment	Toxicities will be evaluated using the Common Terminology Criteria for Adverse Events (CTCAE) version (v).5 standard toxicity grading. Frequency and other descriptive statistics will be used to present the toxicity pattern.	Up to 30 days after the last dose
Primary	Failure rate of platelet recovery	The proportion will be provided with 95% exact binomial confidence interval.	At day 90
Secondary	Independence from platelet transfusion		Up to 1 year
Secondary	Duration of platelet response	Will be presented in a descriptive manner.	Up to 1 year
Secondary	Platelet count >= 50,000/uL for 7 consecutive days without transfusion support		Up to 1 year
Secondary	Duration of exposure to avatrombopag	Will be presented in a descriptive manner.	Up to 1 year
Secondary	Incidence of adverse events associated with avatrombopag treatment		Up to 30 days after last dose
Secondary	Transplant-related mortality		At day 100 and 1 year post-hematopoietic stem cell transplant (HCT)
Secondary	Progression-free survival (PFS) of underlying malignant hematologic disorder	The method of Kaplan-Meier will be used to estimate PFS.	From the time of HCT to progression and death, assessed up to 1 year
Secondary	Overall survival (OS)	The method of Kaplan-Meier will be used to estimate OS.	From the time of HCT to death from any cause, assessed up to 1 year

External Links

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