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Eltrombopag Olamine in Increasing Platelet Counts in Patients Undergoing Transplant

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ClinicalTrials.gov Identifier: NCT01927731
Recruitment Status : Active, not recruiting
First Posted : August 23, 2013
Last Update Posted : November 28, 2018
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Bone Marrow Transplantation Recipient Cord Blood Transplant Recipient Hematopoietic Cell Transplantation Recipient Drug: Eltrombopag Olamine Other: Laboratory Biomarker Analysis Phase 2

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  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Efficacy of Eltrombopag in Patients Undergoing Cord Blood or Haploidentical Bone Marrow Transplantation
Actual Study Start Date : October 14, 2013
Estimated Primary Completion Date : October 31, 2019
Estimated Study Completion Date : October 31, 2019

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Arm I (cord blood transplant patients)
Patients receive eltrombopag olamine PO QD for 60 days beginning on day -1.
Drug: Eltrombopag Olamine
Given PO
Other Names:
  • Promacta
  • SB-497115-GR

Other: Laboratory Biomarker Analysis
Correlative studies

Experimental: Arm II (haploidentical donor stem cell transplant patients)
Patients receive eltrombopag olamine PO QD for 60 days beginning on day 5.
Drug: Eltrombopag Olamine
Given PO
Other Names:
  • Promacta
  • SB-497115-GR

Other: Laboratory Biomarker Analysis
Correlative studies




Primary Outcome Measures :
  1. Rate of platelet engraftment, evaluated as the number of patients experiencing platelet engraftment of greater than 50K/ul [ Time Frame: Up to day 60 ]
    Cumulative incidence will be estimated with death or relapse as a competing risk. Logistic regression will be used to model the association between engraftment endpoints and covariates of interest.


Secondary Outcome Measures :
  1. Incidence of platelet engraftment greater than 20K/ul [ Time Frame: At day 60 ]
    Cumulative incidence will be estimated with death or relapse as a competing risk. Logistic regression will be used to model the association between engraftment endpoints and covariates of interest.

  2. Incidence of platelet engraftment greater than 20K/ul [ Time Frame: At day 30 ]
    Cumulative incidence will be estimated with death or relapse as a competing risk. Logistic regression will be used to model the association between engraftment endpoints and covariates of interest.

  3. Incidence of platelet engraftment greater than 50K/ul [ Time Frame: At day 30 ]
    Cumulative incidence will be estimated with death or relapse as a competing risk. Logistic regression will be used to model the association between engraftment endpoints and covariates of interest.

  4. Incidence of neutrophil engraftment defined as the first of three consecutive days that the absolute neutrophil count is greater than 0.5 k/ul [ Time Frame: Up to day 60 ]
    Cumulative incidence will be estimated with death or relapse as a competing risk. Logistic regression will be used to model the association between engraftment endpoints and covariates of interest.

  5. Time to platelet engraftment [ Time Frame: Up to day 60 ]
    Logistic regression will be used to model the association between engraftment endpoints and covariates of interest.

  6. Time to neutrophil engraftment [ Time Frame: Up to day 60 ]
    Logistic regression will be used to model the association between engraftment endpoints and covariates of interest.

  7. Overall survival [ Time Frame: Up to 1 year ]
    The method of Kaplan and Meier will be used to estimate the distribution of survival time, and distributions will be compared using the log-rank test. Cox proportional hazards regression methods will be used to model survival parameters as a function of disease and demographic covariates of interest.

  8. Progression free survival [ Time Frame: Up to 1 year ]
    The method of Kaplan and Meier will be used to estimate the distribution of progression-free survival times, and distributions will be compared using the log-rank test. Cox proportional hazards regression methods will be used to model survival parameters as a function of disease and demographic covariates of interest.

  9. Cumulative incidence of acute graft-versus-host disease [ Time Frame: Up to day 60 ]
    The method of Gooley et al will be used to estimate the cumulative incidence of graft-versus-host disease.

  10. Cumulative incidence of chronic graft-versus-host disease [ Time Frame: Up to 1 year ]
    The method of Gooley et al will be used to estimate the cumulative incidence of graft-versus-host disease.

  11. Incidence of toxicity, assessed using the National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0 [ Time Frame: Up to day 60 ]
    The rate of grade 4 and higher non-hematologic toxicity attributable to the drug through day 60 in this trial will be monitored by using the method of Thall, Simon, and Estey. Descriptive summaries of toxicity will be provided by arm and dose.



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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:

  • Patients undergoing a cord blood or haploidentical transplantation on any protocol or standard of care treatment plan
  • 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:

  • Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) >= 2.5 upper limit of normal (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 < 30 ml/min; creatinine clearance will be calculated using the Modification of Diet in Renal Disease (MDRD) method
  • Arterial or venous thrombosis in the last year except for line-related venous thrombosis more than 3 months ago
  • Positive beta human chorionic gonadotropin (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

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): NCT01927731


Locations
United States, Texas
M D Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
National Cancer Institute (NCI)
Investigators
Principal Investigator: Uday Popat M.D. Anderson Cancer Center

Additional Information:
Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT01927731     History of Changes
Other Study ID Numbers: 2012-0920
NCI-2013-02348 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
2012-0920 ( Other Identifier: M D Anderson Cancer Center )
P30CA016672 ( U.S. NIH Grant/Contract )
First Posted: August 23, 2013    Key Record Dates
Last Update Posted: November 28, 2018
Last Verified: November 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No