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

• Sponsor: Ayman Saad (Other)
• Overall Status: Withdrawn
• CT.gov ID: NCT04312789
• Collaborator: Sobi, Inc. (Industry)
• Enrollment: 0
• Arm: 1
• Anticipated Duration (Days): 30

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Platelet Disorder	Drug: Avatrombopag	Phase 2

Detailed Description

PRIMARY OBJECTIVE:

1. To determine the safety and efficacy of avatrombopag for the treatment of thrombocytopenia after allogenic hematopoietic stem cell transplantation.

SECONDARY OBJECTIVE:

1. 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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 0 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
• Anticipated Study Start Date: Dec 1, 2022
• Anticipated Primary Completion Date: Dec 31, 2022
• Anticipated Study Completion Date: Dec 31, 2022

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Incidence of adverse events of avatrombopag treatment [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 [At day 90]

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

Secondary Outcome Measures

1. Independence from platelet transfusion [Up to 1 year]

2. Duration of platelet response [Up to 1 year]

   Will be presented in a descriptive manner.

3. Platelet count >= 50,000/uL for 7 consecutive days without transfusion support [Up to 1 year]

4. Duration of exposure to avatrombopag [Up to 1 year]

   Will be presented in a descriptive manner.

5. Incidence of adverse events associated with avatrombopag treatment [Up to 30 days after last dose]

6. Transplant-related mortality [At day 100 and 1 year post-hematopoietic stem cell transplant (HCT)]

7. Progression-free survival (PFS) of underlying malignant hematologic disorder [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) [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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

• Ayman Saad
• Sobi, Inc.

Investigators

• Principal Investigator: Ayman Saad, MB/BCH, Ohio State University Comprehensive Cancer Center

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• The Jamesline

Publications

None provided.

• Responsible Party: Ayman Saad, Principal Investigator, Ohio State University Comprehensive Cancer Center
• ClinicalTrials.gov Identifier: NCT04312789
• Other Study ID Numbers: OSU-19328; NCI-2020-01035
• First Posted: Mar 18, 2020
• Last Update Posted: Dec 9, 2021
• Last Verified: Nov 1, 2021

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Thrombocytopenia; Blood Platelet Disorders

Study Results

No Results Posted as of Dec 9, 2021