Eltrombopag Olamine in Increasing Platelet Counts in Patients Undergoing Transplant

Safety and Efficacy of Eltrombopag in Patients Undergoing Cord Blood or Haploidentical Bone Marrow Transplantation

This phase II trial studies how well eltrombopag olamine works in increasing platelet counts in patients undergoing transplant. Eltrombopag olamine may help platelet counts and the immune system recover from blood or bone marrow transplant.

Study Overview

• Status: Completed
• Conditions: Hematopoietic Cell Transplantation Recipient; Bone Marrow Transplantation Recipient; Cord Blood Transplant Recipient
• Intervention / Treatment: Other: Laboratory Biomarker Analysis; Drug: Eltrombopag Olamine

Detailed Description

PRIMARY OBJECTIVES:

I. To estimate the rate of platelet engraftment by day 60 in patients undergoing cord blood transplant (CBT) or haploidentical donor stem cell transplantation treated with eltrombopag (eltrombopag olamine).

SECONDARY OBJECTIVES:

I. To assess safety of eltrombopag in this population. II. To assess neutrophil engraftment with eltrombopag in this population. III. To characterize immune reconstitution. IV. To assess overall survival (OS). V. To assess progression free survival (PFS). VI. To assess incidence of acute graft-versus-host disease (GVHD).

OUTLINE: Patients are assigned to 1 of 2 arms.

ARM I (CORD BLOOD TRANSPLANT): Patients receive eltrombopag olamine orally (PO) once daily (QD) for 60 days beginning on day -1.

ARM II (HAPLOIDENTICAL DONOR STEM CELL TRANSPLANT): Patients receive eltrombopag olamine PO QD for 60 days beginning on day 5.

After completion of study treatment, patients are followed up at 1, 2, 3, 6, and 12 months.

• Study Type: Interventional
• Enrollment (Actual): 48
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Texas
    • Houston, Texas, United States, 77030
      • M D Anderson Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Patients undergoing a cord blood or haploidentical transplantation on any protocol or standard of care treatment plan.
• Age >/= 18.
• Females of child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization or breast-feeding must be willing to use an effective contraceptive measure until 30 days after the last dose of eltrombopag. Males who have had sexual contact with female of child-bearing potential must be willing to use contraceptive techniques until 30 days after the last dose of eltrombopag.
• Patient or patient's legal representative(s) is/are able to provide written informed consent to participate.

Exclusion Criteria:

• ALT and AST >/= 2.5 ULN.
• Serum direct bilirubin >/= 1 mg/dl (except Gilbert's syndrome or hemolysis).
• Patients of east Asian ancestry (Chinese, Japanese, or Korean Origin).
• Calculated creatinine clearance < 30ml./min. Creatinine clearance will be calculated using the MDRD method.
• Arterial or venous thrombosis in the last year except for line-related venous thrombosis more than 3 months ago.
• Positive beta HCG within 7 days prior to consent in female of child-bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization or breast-feeding.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Arm I (cord blood transplant patients); Patients receive eltrombopag olamine PO QD for 60 days beginning on day -1.	Other: Laboratory Biomarker Analysis; Correlative studies; Drug: Eltrombopag Olamine; Given PO; Other Names: Promacta; SB-497115-GR
Experimental: Arm II (haploidentical donor stem cell transplant patients); Patients receive eltrombopag olamine PO QD for 60 days beginning on day 5.	Other: Laboratory Biomarker Analysis; Correlative studies; Drug: Eltrombopag Olamine; Given PO; Other Names: Promacta; SB-497115-GR

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants That Achieved Platelet Engraftment Greater Than 50K/ul	Number of participants that achieved platelet engraftment greater than 50K/ul 60 days post transplant.	Up to day 60

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival	Number of participants that survived at one year post transplant.	Up to 1 year
Progression Free Survival	Number of participants without disease progression at 1 year post transplant.	Up to 1 year

Collaborators and Investigators

• Sponsor: M.D. Anderson Cancer Center
• Collaborators: National Cancer Institute (NCI)
• Investigators: Principal Investigator: Uday Popat, M.D. Anderson Cancer Center

Publications and helpful links

Helpful Links

• MD Anderson Cancer Center Website

Study record dates

Study Major Dates

• Study Start (Actual): October 14, 2013
• Primary Completion (Actual): June 6, 2023
• Study Completion (Actual): June 6, 2023

Study Registration Dates

• First Submitted: August 20, 2013
• First Submitted That Met QC Criteria: August 22, 2013
• First Posted (Estimated): August 23, 2013

Study Record Updates

• Last Update Posted (Actual): April 23, 2024
• Last Update Submitted That Met QC Criteria: March 27, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Other Study ID Numbers: 2012-0920 (Other Identifier: M D Anderson Cancer Center); NCI-2013-02348 (Registry Identifier: CTRP (Clinical Trial Reporting Program))

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No