Trial Outcomes & Findings for Eltrombopag for Post Transplant Thrombocytopenia (NCT NCT01000051)
NCT ID: NCT01000051
Last Updated: 2023-09-18
Results Overview
Comparison of the efficacy of eltrombopag and placebo in patients with thrombocytopenia post hematopoietic cell transplantation (HCT).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
64 participants
Primary outcome timeframe
Baseline to Day 57
Results posted on
2023-09-18
Participant Flow
Four participants that was enrolled was taken off the study before the randomization process, therefore not included in the participants that started.
Participant milestones
| Measure |
Eltrombopag
Starting dose 50 mg/day orally for 8 weeks
Eltrombopag: Starting daily dose 50 mg orally on empty stomach (1 hour before or 2 hours after a meal) for 8 weeks. East Asians start at 25 mg daily.
|
Placebo
Once a day orally for 8 weeks
Placebo: Once a day, orally for 8 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
42
|
18
|
|
Overall Study
COMPLETED
|
39
|
17
|
|
Overall Study
NOT COMPLETED
|
3
|
1
|
Reasons for withdrawal
| Measure |
Eltrombopag
Starting dose 50 mg/day orally for 8 weeks
Eltrombopag: Starting daily dose 50 mg orally on empty stomach (1 hour before or 2 hours after a meal) for 8 weeks. East Asians start at 25 mg daily.
|
Placebo
Once a day orally for 8 weeks
Placebo: Once a day, orally for 8 weeks.
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
1
|
|
Overall Study
Disease Progression
|
1
|
0
|
|
Overall Study
Serious noncompliance
|
1
|
0
|
Baseline Characteristics
Eltrombopag for Post Transplant Thrombocytopenia
Baseline characteristics by cohort
| Measure |
Eltrombopag
n=39 Participants
Starting dose 50 mg/day orally for 8 weeks
Eltrombopag: Starting daily dose 50 mg orally on empty stomach (1 hour before or 2 hours after a meal) for 8 weeks. East Asians start at 25 mg daily.
|
Placebo
n=17 Participants
Once a day orally for 8 weeks
Placebo: Once a day, orally for 8 weeks.
|
Total
n=56 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
|
37 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
51 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
22 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
33 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
33 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
46 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
5 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
28 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
42 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to Day 57Comparison of the efficacy of eltrombopag and placebo in patients with thrombocytopenia post hematopoietic cell transplantation (HCT).
Outcome measures
| Measure |
Eltrombopag
n=39 Participants
Starting dose 50 mg/day orally for 8 weeks
Eltrombopag: Starting daily dose 50 mg orally on empty stomach (1 hour before or 2 hours after a meal) for 8 weeks. East Asians start at 25 mg daily.
|
Placebo
n=17 Participants
Once a day orally for 8 weeks
Placebo: Once a day, orally for 8 weeks.
|
|---|---|---|
|
Comparing the Efficacy of Eltrombopaq and Placebo
Achieved platelet count >/= 30 x 10
|
15 Participants
|
5 Participants
|
|
Comparing the Efficacy of Eltrombopaq and Placebo
Achieved platelet count >/= 50 x 10
|
9 Participants
|
0 Participants
|
|
Comparing the Efficacy of Eltrombopaq and Placebo
Platelet count < 30x 10
|
15 Participants
|
12 Participants
|
Adverse Events
Eltrombopag
Serious events: 14 serious events
Other events: 31 other events
Deaths: 5 deaths
Placebo
Serious events: 3 serious events
Other events: 16 other events
Deaths: 4 deaths
Serious adverse events
| Measure |
Eltrombopag
n=39 participants at risk
Starting dose 50 mg/day orally for 8 weeks
Eltrombopag: Starting daily dose 50 mg orally on empty stomach (1 hour before or 2 hours after a meal) for 8 weeks. East Asians start at 25 mg daily.
|
Placebo
n=17 participants at risk
Once a day orally for 8 weeks
Placebo: Once a day, orally for 8 weeks.
|
|---|---|---|
|
Blood and lymphatic system disorders
Hemorrhage/Bleeding
|
7.7%
3/39 • Through study completion, average of 12 weeks
|
0.00%
0/17 • Through study completion, average of 12 weeks
|
|
Blood and lymphatic system disorders
Ataxia
|
2.6%
1/39 • Through study completion, average of 12 weeks
|
0.00%
0/17 • Through study completion, average of 12 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
7.7%
3/39 • Through study completion, average of 12 weeks
|
0.00%
0/17 • Through study completion, average of 12 weeks
|
|
Gastrointestinal disorders
Colitis
|
2.6%
1/39 • Through study completion, average of 12 weeks
|
0.00%
0/17 • Through study completion, average of 12 weeks
|
|
Gastrointestinal disorders
Vomiting
|
5.1%
2/39 • Through study completion, average of 12 weeks
|
5.9%
1/17 • Through study completion, average of 12 weeks
|
|
Gastrointestinal disorders
Diarrhea
|
2.6%
1/39 • Through study completion, average of 12 weeks
|
0.00%
0/17 • Through study completion, average of 12 weeks
|
|
Gastrointestinal disorders
GI graft vs host disease
|
2.6%
1/39 • Through study completion, average of 12 weeks
|
0.00%
0/17 • Through study completion, average of 12 weeks
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Death/Disease Progression
|
2.6%
1/39 • Through study completion, average of 12 weeks
|
5.9%
1/17 • Through study completion, average of 12 weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
2.6%
1/39 • Through study completion, average of 12 weeks
|
0.00%
0/17 • Through study completion, average of 12 weeks
|
|
Nervous system disorders
Seizure
|
2.6%
1/39 • Through study completion, average of 12 weeks
|
0.00%
0/17 • Through study completion, average of 12 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis/pulmonary infiltrates
|
0.00%
0/39 • Through study completion, average of 12 weeks
|
11.8%
2/17 • Through study completion, average of 12 weeks
|
|
Renal and urinary disorders
Acute kidney injury
|
0.00%
0/39 • Through study completion, average of 12 weeks
|
5.9%
1/17 • Through study completion, average of 12 weeks
|
|
Skin and subcutaneous tissue disorders
Cellulitis
|
2.6%
1/39 • Through study completion, average of 12 weeks
|
0.00%
0/17 • Through study completion, average of 12 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
2.6%
1/39 • Through study completion, average of 12 weeks
|
0.00%
0/17 • Through study completion, average of 12 weeks
|
|
Infections and infestations
Infections
|
12.8%
5/39 • Through study completion, average of 12 weeks
|
11.8%
2/17 • Through study completion, average of 12 weeks
|
Other adverse events
| Measure |
Eltrombopag
n=39 participants at risk
Starting dose 50 mg/day orally for 8 weeks
Eltrombopag: Starting daily dose 50 mg orally on empty stomach (1 hour before or 2 hours after a meal) for 8 weeks. East Asians start at 25 mg daily.
|
Placebo
n=17 participants at risk
Once a day orally for 8 weeks
Placebo: Once a day, orally for 8 weeks.
|
|---|---|---|
|
Renal and urinary disorders
Creatinine increased
|
12.8%
5/39 • Through study completion, average of 12 weeks
|
29.4%
5/17 • Through study completion, average of 12 weeks
|
|
Gastrointestinal disorders
Nausea
|
30.8%
12/39 • Through study completion, average of 12 weeks
|
17.6%
3/17 • Through study completion, average of 12 weeks
|
|
Gastrointestinal disorders
Abdominal pain
|
2.6%
1/39 • Through study completion, average of 12 weeks
|
0.00%
0/17 • Through study completion, average of 12 weeks
|
|
Gastrointestinal disorders
Diarrhea
|
17.9%
7/39 • Through study completion, average of 12 weeks
|
29.4%
5/17 • Through study completion, average of 12 weeks
|
|
Gastrointestinal disorders
Vomitting
|
7.7%
3/39 • Through study completion, average of 12 weeks
|
17.6%
3/17 • Through study completion, average of 12 weeks
|
|
Hepatobiliary disorders
Alanine aminotransferanse increased
|
7.7%
3/39 • Through study completion, average of 12 weeks
|
35.3%
6/17 • Through study completion, average of 12 weeks
|
|
Hepatobiliary disorders
Ascites
|
2.6%
1/39 • Through study completion, average of 12 weeks
|
0.00%
0/17 • Through study completion, average of 12 weeks
|
|
Hepatobiliary disorders
Aspatate aminotransferase increased
|
2.6%
1/39 • Through study completion, average of 12 weeks
|
5.9%
1/17 • Through study completion, average of 12 weeks
|
|
Blood and lymphatic system disorders
Hemorrhage/Bleeding
|
7.7%
3/39 • Through study completion, average of 12 weeks
|
23.5%
4/17 • Through study completion, average of 12 weeks
|
|
Renal and urinary disorders
Hemorrhage/Urinary Tract
|
5.1%
2/39 • Through study completion, average of 12 weeks
|
5.9%
1/17 • Through study completion, average of 12 weeks
|
|
Nervous system disorders
Headache
|
5.1%
2/39 • Through study completion, average of 12 weeks
|
0.00%
0/17 • Through study completion, average of 12 weeks
|
|
Infections and infestations
Infection
|
17.9%
7/39 • Through study completion, average of 12 weeks
|
35.3%
6/17 • Through study completion, average of 12 weeks
|
|
Blood and lymphatic system disorders
Petechiae
|
2.6%
1/39 • Through study completion, average of 12 weeks
|
17.6%
3/17 • Through study completion, average of 12 weeks
|
|
Hepatobiliary disorders
Bilirubin increased
|
25.6%
10/39 • Through study completion, average of 12 weeks
|
11.8%
2/17 • Through study completion, average of 12 weeks
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
2.6%
1/39 • Through study completion, average of 12 weeks
|
5.9%
1/17 • Through study completion, average of 12 weeks
|
Additional Information
Dr. Popat, Uday / Professor, Stem Cell Transplantation
MD Anderson Cancer Center
Phone: 713-745-3055
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place