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

NCT04312789 | Withdrawn Phase 2 DMC FDA Drug

• 2 other identifiers: OSU-19328NCI-2020-01035
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Platelet Disorder

Outcome Measures

Primary Outcomes (2)

• 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

• Failure rate of platelet recovery

  The proportion will be provided with 95% exact binomial confidence interval.

  At day 90

Secondary Outcomes (8)

• Independence from platelet transfusion

  Up to 1 year

• Duration of platelet response

  Up to 1 year

• Platelet count >= 50,000/uL for 7 consecutive days without transfusion support

  Up to 1 year

• Duration of exposure to avatrombopag

  Up to 1 year

• Incidence of adverse events associated with avatrombopag treatment

  Up to 30 days after last dose

Study Arms (1)

Treatment (avatrombopag)

EXPERIMENTAL

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

Interventions

Avatrombopag DRUG

Given PO

Also known as: AKR-501, AS 1670542, Doptelet, E5501, YM 477

Treatment (avatrombopag)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Ayman Saad: lead
• Sobi, Inc.: collaborator

Related Links

• The Jamesline

MeSH Terms

Conditions

Blood Platelet Disorders

Interventions

avatrombopag

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Ayman Saad, MB/BCH

  Ohio State University Comprehensive Cancer Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: March 16, 2020
• First Posted: March 18, 2020
• Study Start: December 1, 2022
• Primary Completion: December 31, 2022
• Study Completion: December 31, 2022
• Last Updated: December 9, 2021

Record last verified: 2021-11

Data Sharing

• IPD Sharing: Will not share