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

NCT ID: NCT04312789

Last Updated: 2021-12-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

2022-12-31

Study Completion Date

2022-12-31

Brief Summary

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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

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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.

Conditions

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Platelet Disorder

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

Avatrombopag

Intervention Type DRUG

Given PO

Interventions

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Avatrombopag

Given PO

Intervention Type DRUG

Other Intervention Names

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AKR-501 AS 1670542 Doptelet E5501 YM 477

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sobi, Inc.

INDUSTRY

Sponsor Role collaborator

Ayman Saad

OTHER

Sponsor Role lead

Responsible Party

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Ayman Saad

Principal Investigator

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

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Ayman Saad, MB/BCH

Role: PRINCIPAL_INVESTIGATOR

Ohio State University Comprehensive Cancer Center

Countries

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United States

Related Links

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Other Identifiers

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NCI-2020-01035

Identifier Type: REGISTRY

Identifier Source: secondary_id

OSU-19328

Identifier Type: -

Identifier Source: org_study_id