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

Primary Purpose

Platelet Disorder

Status
Withdrawn
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Avatrombopag
Sponsored by
Ayman Saad
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Platelet Disorder

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Treatment (avatrombopag)

    Arm Description

    Patients receive avatrombopag PO 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.

    Outcomes

    Primary Outcome Measures

    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.
    Failure rate of platelet recovery
    The proportion will be provided with 95% exact binomial confidence interval.

    Secondary Outcome Measures

    Independence from platelet transfusion
    Duration of platelet response
    Will be presented in a descriptive manner.
    Platelet count >= 50,000/uL for 7 consecutive days without transfusion support
    Duration of exposure to avatrombopag
    Will be presented in a descriptive manner.
    Incidence of adverse events associated with avatrombopag treatment
    Transplant-related mortality
    Progression-free survival (PFS) of underlying malignant hematologic disorder
    The method of Kaplan-Meier will be used to estimate PFS.
    Overall survival (OS)
    The method of Kaplan-Meier will be used to estimate OS.

    Full Information

    First Posted
    March 16, 2020
    Last Updated
    November 26, 2021
    Sponsor
    Ayman Saad
    Collaborators
    Sobi, Inc.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT04312789
    Brief Title
    Avatrombopag for the Treatment of Thrombocytopenia After Donor Hematopoietic Stem Cell Transplant
    Official Title
    A Phase II Trial of Avatrombopag for the Treatment of Thrombocytopenia After Allogeneic Hematopoietic Stem Cell Transplant
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    November 2021
    Overall Recruitment Status
    Withdrawn
    Why Stopped
    Drug availability
    Study Start Date
    December 2022 (Anticipated)
    Primary Completion Date
    December 31, 2022 (Anticipated)
    Study Completion Date
    December 31, 2022 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor-Investigator
    Name of the Sponsor
    Ayman Saad
    Collaborators
    Sobi, Inc.

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    Yes
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    Yes

    5. Study Description

    Brief 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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Platelet Disorder

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    0 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Treatment (avatrombopag)
    Arm Type
    Experimental
    Arm Description
    Patients receive avatrombopag PO 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.
    Intervention Type
    Drug
    Intervention Name(s)
    Avatrombopag
    Other Intervention Name(s)
    AKR-501, AS 1670542, Doptelet, E5501, YM 477
    Intervention Description
    Given PO
    Primary Outcome Measure Information:
    Title
    Incidence of adverse events of avatrombopag treatment
    Description
    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.
    Time Frame
    Up to 30 days after the last dose
    Title
    Failure rate of platelet recovery
    Description
    The proportion will be provided with 95% exact binomial confidence interval.
    Time Frame
    At day 90
    Secondary Outcome Measure Information:
    Title
    Independence from platelet transfusion
    Time Frame
    Up to 1 year
    Title
    Duration of platelet response
    Description
    Will be presented in a descriptive manner.
    Time Frame
    Up to 1 year
    Title
    Platelet count >= 50,000/uL for 7 consecutive days without transfusion support
    Time Frame
    Up to 1 year
    Title
    Duration of exposure to avatrombopag
    Description
    Will be presented in a descriptive manner.
    Time Frame
    Up to 1 year
    Title
    Incidence of adverse events associated with avatrombopag treatment
    Time Frame
    Up to 30 days after last dose
    Title
    Transplant-related mortality
    Time Frame
    At day 100 and 1 year post-hematopoietic stem cell transplant (HCT)
    Title
    Progression-free survival (PFS) of underlying malignant hematologic disorder
    Description
    The method of Kaplan-Meier will be used to estimate PFS.
    Time Frame
    From the time of HCT to progression and death, assessed up to 1 year
    Title
    Overall survival (OS)
    Description
    The method of Kaplan-Meier will be used to estimate OS.
    Time Frame
    From the time of HCT to death from any cause, assessed up to 1 year

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    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
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Ayman Saad, MB/BCH
    Organizational Affiliation
    Ohio State University Comprehensive Cancer Center
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Plan to Share IPD
    No
    Links:
    URL
    http://cancer.osu.edu
    Description
    The Jamesline

    Learn more about this trial

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

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