Bone Marrow Transplantation Recipient, Cord Blood Transplant Recipient, Hematopoietic Cell Transplantation Recipient
Conditions
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.
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.
Interventions
Given PO
OTHERLaboratory Biomarker Analysis
Correlative studies
Sponsors
National Cancer Institute (NCI)
M.D. Anderson Cancer Center
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants That Achieved Platelet Engraftment Greater Than 50K/ul | Up to day 60 | Number of participants that achieved platelet engraftment greater than 50K/ul 60 days post transplant. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Overall Survival | Up to 1 year | Number of participants that survived at one year post transplant. |
| Progression Free Survival | Up to 1 year | Number of participants without disease progression at 1 year post transplant. |
Countries
United States
Participant flow
Recruitment details
October 2013 to November 2016. All participants were registered in University of Texas MD Anderson Cancer Center.
Participants by arm
| Arm | Count |
|---|---|
| Arm I (Cord Blood Transplant Patients) Patients receive eltrombopag olamine PO QD for 60 days beginning on day -1.
Eltrombopag Olamine: Given PO
Laboratory Biomarker Analysis: Correlative studies | 6 |
| Arm II (Haploidentical Donor Stem Cell Transplant Patients) Patients receive eltrombopag olamine PO QD for 60 days beginning on day 5.
Eltrombopag Olamine: Given PO
Laboratory Biomarker Analysis: Correlative studies | 42 |
| Total | 48 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Death | 0 | 3 |
| Overall Study | Graft Failure | 1 | 2 |
| Overall Study | Physician Decision | 2 | 3 |
| Overall Study | Withdrawal by Subject | 0 | 2 |
Baseline characteristics
| Characteristic | Arm I (Cord Blood Transplant Patients) | Arm II (Haploidentical Donor Stem Cell Transplant Patients) | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 6 Participants | 6 Participants |
| Age, Categorical Between 18 and 65 years | 6 Participants | 36 Participants | 42 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants | 7 Participants | 8 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 5 Participants | 35 Participants | 40 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Region of Enrollment United States | 6 participants | 42 participants | 48 participants |
| Sex: Female, Male Female | 1 Participants | 18 Participants | 19 Participants |
| Sex: Female, Male Male | 5 Participants | 24 Participants | 29 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 6 | 3 / 42 |
| other Total, other adverse events | 2 / 6 | 8 / 42 |
| serious Total, serious adverse events | 2 / 6 | 2 / 42 |
Outcome results
Primary
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.
Time frame: Up to day 60
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Arm I (Cord Blood Transplant Patients) | Number of Participants That Achieved Platelet Engraftment Greater Than 50K/ul | 3 Participants |
| Arm II (Haploidentical Donor Stem Cell Transplant Patients) | Number of Participants That Achieved Platelet Engraftment Greater Than 50K/ul | 30 Participants |
Secondary
Overall Survival
Number of participants that survived at one year post transplant.
Time frame: Up to 1 year
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Arm I (Cord Blood Transplant Patients) | Overall Survival | 3 Participants |
| Arm II (Haploidentical Donor Stem Cell Transplant Patients) | Overall Survival | 32 Participants |
Secondary
Progression Free Survival
Number of participants without disease progression at 1 year post transplant.
Time frame: Up to 1 year
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Arm I (Cord Blood Transplant Patients) | Progression Free Survival | 3 Participants |
| Arm II (Haploidentical Donor Stem Cell Transplant Patients) | Progression Free Survival | 30 Participants |