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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04312789
Recruitment Status : Recruiting
First Posted : March 18, 2020
Last Update Posted : September 16, 2020
Sponsor:
Collaborator:
Dova Pharmaceuticals
Information provided by (Responsible Party):
Basem William, MD, Ohio State University Comprehensive Cancer Center

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.

Condition or disease Intervention/treatment Phase
Platelet Disorder Drug: Avatrombopag Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 27 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Trial of Avatrombopag for the Treatment of Thrombocytopenia After Allogeneic Hematopoietic Stem Cell Transplant
Actual Study Start Date : August 10, 2020
Estimated Primary Completion Date : December 31, 2022
Estimated Study Completion Date : December 31, 2022

Arm Intervention/treatment
Experimental: Treatment (avatrombopag)
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.
Drug: Avatrombopag
Given PO
Other Names:
  • AKR-501
  • AS 1670542
  • Doptelet
  • E5501
  • YM 477




Primary Outcome Measures :
  1. Incidence of adverse events of avatrombopag treatment [ Time Frame: Up to 30 days after the last dose ]
    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.

  2. Failure rate of platelet recovery [ Time Frame: At day 90 ]
    The proportion will be provided with 95% exact binomial confidence interval.


Secondary Outcome Measures :
  1. Independence from platelet transfusion [ Time Frame: Up to 1 year ]
  2. Duration of platelet response [ Time Frame: Up to 1 year ]
    Will be presented in a descriptive manner.

  3. Platelet count >= 50,000/uL for 7 consecutive days without transfusion support [ Time Frame: Up to 1 year ]
  4. Duration of exposure to avatrombopag [ Time Frame: Up to 1 year ]
    Will be presented in a descriptive manner.

  5. Incidence of adverse events associated with avatrombopag treatment [ Time Frame: Up to 30 days after last dose ]
  6. Transplant-related mortality [ Time Frame: At day 100 and 1 year post-hematopoietic stem cell transplant (HCT) ]
  7. Progression-free survival (PFS) of underlying malignant hematologic disorder [ Time Frame: From the time of HCT to progression and death, assessed up to 1 year ]
    The method of Kaplan-Meier will be used to estimate PFS.

  8. Overall survival (OS) [ Time Frame: From the time of HCT to death from any cause, assessed up to 1 year ]
    The method of Kaplan-Meier will be used to estimate OS.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04312789


Contacts
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Contact: Ohio State Comprehensive Cancer Center 800-293-5066 OSUCCCClinicaltrials@osumc.edu

Locations
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United States, Ohio
Ohio State University Comprehensive Cancer Center Recruiting
Columbus, Ohio, United States, 43210
Contact: Basem M. William, MD MRCP FACP    614-688-7942    basem.William@osumc.edu   
Principal Investigator: Basem M. William, MD MRCP FACP         
Sponsors and Collaborators
Basem William, MD
Dova Pharmaceuticals
Investigators
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Principal Investigator: Basem M William, MD MRCP FACP Ohio State University Comprehensive Cancer Center
Additional Information:
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Responsible Party: Basem William, MD, Principal Investigator, Ohio State University Comprehensive Cancer Center
ClinicalTrials.gov Identifier: NCT04312789    
Other Study ID Numbers: OSU-19328
NCI-2020-01035 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
First Posted: March 18, 2020    Key Record Dates
Last Update Posted: September 16, 2020
Last Verified: September 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Thrombocytopenia
Blood Platelet Disorders
Hematologic Diseases