Trial Outcomes & Findings for Eltrombopag Olamine in Increasing Platelet Counts in Patients Undergoing Transplant (NCT NCT01927731)
NCT ID: NCT01927731
Last Updated: 2024-04-23
Results Overview
Number of participants that achieved platelet engraftment greater than 50K/ul 60 days post transplant.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
48 participants
Primary outcome timeframe
Up to day 60
Results posted on
2024-04-23
Participant Flow
October 2013 to November 2016. All participants were registered in University of Texas MD Anderson Cancer Center.
Participant milestones
| Measure |
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
|
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
|
|---|---|---|
|
Overall Study
STARTED
|
6
|
42
|
|
Overall Study
COMPLETED
|
3
|
32
|
|
Overall Study
NOT COMPLETED
|
3
|
10
|
Reasons for withdrawal
| Measure |
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
|
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
|
|---|---|---|
|
Overall Study
Graft Failure
|
1
|
2
|
|
Overall Study
Physician Decision
|
2
|
3
|
|
Overall Study
Withdrawal by Subject
|
0
|
2
|
|
Overall Study
Death
|
0
|
3
|
Baseline Characteristics
Eltrombopag Olamine in Increasing Platelet Counts in Patients Undergoing Transplant
Baseline characteristics by cohort
| Measure |
Arm I (Cord Blood Transplant Patients)
n=6 Participants
Patients receive eltrombopag olamine PO QD for 60 days beginning on day -1.
Eltrombopag Olamine: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
Arm II (Haploidentical Donor Stem Cell Transplant Patients)
n=42 Participants
Patients receive eltrombopag olamine PO QD for 60 days beginning on day 5.
Eltrombopag Olamine: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
Total
n=48 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
6 Participants
n=5 Participants
|
36 Participants
n=7 Participants
|
42 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
19 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
24 Participants
n=7 Participants
|
29 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
5 Participants
n=5 Participants
|
35 Participants
n=7 Participants
|
40 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=5 Participants
|
42 participants
n=7 Participants
|
48 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to day 60Number of participants that achieved platelet engraftment greater than 50K/ul 60 days post transplant.
Outcome measures
| Measure |
Arm I (Cord Blood Transplant Patients)
n=6 Participants
Patients receive eltrombopag olamine PO QD for 60 days beginning on day -1.
Eltrombopag Olamine: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
Arm II (Haploidentical Donor Stem Cell Transplant Patients)
n=42 Participants
Patients receive eltrombopag olamine PO QD for 60 days beginning on day 5.
Eltrombopag Olamine: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|---|
|
Number of Participants That Achieved Platelet Engraftment Greater Than 50K/ul
|
3 Participants
|
30 Participants
|
SECONDARY outcome
Timeframe: Up to 1 yearNumber of participants that survived at one year post transplant.
Outcome measures
| Measure |
Arm I (Cord Blood Transplant Patients)
n=3 Participants
Patients receive eltrombopag olamine PO QD for 60 days beginning on day -1.
Eltrombopag Olamine: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
Arm II (Haploidentical Donor Stem Cell Transplant Patients)
n=32 Participants
Patients receive eltrombopag olamine PO QD for 60 days beginning on day 5.
Eltrombopag Olamine: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|---|
|
Overall Survival
|
3 Participants
|
32 Participants
|
SECONDARY outcome
Timeframe: Up to 1 yearNumber of participants without disease progression at 1 year post transplant.
Outcome measures
| Measure |
Arm I (Cord Blood Transplant Patients)
n=3 Participants
Patients receive eltrombopag olamine PO QD for 60 days beginning on day -1.
Eltrombopag Olamine: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
Arm II (Haploidentical Donor Stem Cell Transplant Patients)
n=32 Participants
Patients receive eltrombopag olamine PO QD for 60 days beginning on day 5.
Eltrombopag Olamine: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|---|
|
Progression Free Survival
|
3 Participants
|
30 Participants
|
Adverse Events
Arm I (Cord Blood Transplant Patients)
Serious events: 2 serious events
Other events: 2 other events
Deaths: 0 deaths
Arm II (Haploidentical Donor Stem Cell Transplant Patients)
Serious events: 2 serious events
Other events: 8 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
Arm I (Cord Blood Transplant Patients)
n=6 participants at risk
Patients receive eltrombopag olamine PO QD for 60 days beginning on day -1.
Eltrombopag Olamine: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
Arm II (Haploidentical Donor Stem Cell Transplant Patients)
n=42 participants at risk
Patients receive eltrombopag olamine PO QD for 60 days beginning on day 5.
Eltrombopag Olamine: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|---|
|
Infections and infestations
Sepsis
|
33.3%
2/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
33.3%
2/6 • Up to 1 year
|
4.8%
2/42 • Up to 1 year
|
|
Infections and infestations
Infections and infestations
|
33.3%
2/6 • Up to 1 year
|
4.8%
2/42 • Up to 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Adult respiratory distress syndrome
|
16.7%
1/6 • Up to 1 year
|
0.00%
0/42 • Up to 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration
|
16.7%
1/6 • Up to 1 year
|
0.00%
0/42 • Up to 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
Other adverse events
| Measure |
Arm I (Cord Blood Transplant Patients)
n=6 participants at risk
Patients receive eltrombopag olamine PO QD for 60 days beginning on day -1.
Eltrombopag Olamine: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
Arm II (Haploidentical Donor Stem Cell Transplant Patients)
n=42 participants at risk
Patients receive eltrombopag olamine PO QD for 60 days beginning on day 5.
Eltrombopag Olamine: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|---|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Renal and urinary disorders
Acute kidney injury
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Adult respiratory distress syndrome
|
16.7%
1/6 • Up to 1 year
|
0.00%
0/42 • Up to 1 year
|
|
Investigations
Blood bilirubin increased
|
0.00%
0/6 • Up to 1 year
|
4.8%
2/42 • Up to 1 year
|
|
Infections and infestations
Bronchial infection
|
16.7%
1/6 • Up to 1 year
|
0.00%
0/42 • Up to 1 year
|
|
Infections and infestations
Catheter related infection
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Cardiac disorders
Chest pain - cardiac
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Investigations
Creatinine increased
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Renal and urinary disorders
Cystitis noninfective
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/6 • Up to 1 year
|
11.9%
5/42 • Up to 1 year
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
General disorders
Edema limbs
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Infections and infestations
Enterocolitis infectious
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
General disorders
Fever
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
General disorders
General disorders and administration site conditions
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Nervous system disorders
Headache
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Renal and urinary disorders
Hematuria
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Infections and infestations
Infections and infestations
|
0.00%
0/6 • Up to 1 year
|
19.0%
8/42 • Up to 1 year
|
|
Nervous system disorders
Lethargy
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/6 • Up to 1 year
|
9.5%
4/42 • Up to 1 year
|
|
Nervous system disorders
Nervous system disorders
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
General disorders
Pain
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Infections and infestations
Pleural infection
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Renal and urinary disorders
Renal and urinary disorders
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Infections and infestations
Soft tissue infection
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Blood and lymphatic system disorders
Thrombotic thrombocytopenic purpura
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Infections and infestations
Upper respiratory infection
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/6 • Up to 1 year
|
9.5%
4/42 • Up to 1 year
|
|
Renal and urinary disorders
Urinary tract pain
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
|
Cardiac disorders
Ventricular tachycardia
|
0.00%
0/6 • Up to 1 year
|
2.4%
1/42 • Up to 1 year
|
Additional Information
Uday Popat, M.D. / Stem Cell Transplantation
The University of Texas MD Anderson Cancer Center
Phone: 713-792-8750
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place