Eltrombopag for Post Transplant Thrombocytopenia
18 Years
N/A
Both
Thrombocytopenia
Trial Information
Eltrombopag for Post Transplant Thrombocytopenia
The Study Drug:
Platelets are cells that help the blood clot. Platelet counts are often lower after
receiving a stem cell transplant. When the number of platelets in your body gets too low,
it can cause bleeding. Eltrombopag is designed to imitate a protein in your body that helps
make platelets. This may help your platelet counts stay normal.
Study Groups:
If you are found to be eligible to take part in this study, you will be randomly assigned
(as in the flip of a coin) to 1 of 2 study groups:
- If you are in Group 1, you will receive eltrombopag.
- If you are in Group 2, you will receive a placebo. A placebo is a substance that looks
like the study drug but has no active ingredients.
If you are 1 of the first 20 participants enrolled in this study, you will have an equal
chance of being assigned to either group. If you join the study after 20 participants have
been enrolled, you will have a better chance of being assigned to Group 1 if eltrombopag
has been shown to be more effective.
Neither you nor the study staff will know if you are receiving the study drug or the
placebo. However, if needed for your safety, the study staff will be able to find out what
you are receiving.
Study Drug Administration:
You will take the study drug/placebo pills by mouth 1 time each day while you are on study.
The study drug/placebo should be taken on an empty stomach (1 hour before or 2 hours after
food/drink). You may drink water, coffee, and/or tea with the study drug/placebo. You
should drink any coffee or tea without milk or cream.
The dose of study drug/placebo may be adjusted (raised or lowered) throughout the study
based on your reaction to the study drug/placebo.
Study Visits:
Every 2 weeks, the following tests and procedures will be performed:
- You will have a physical exam, including measurement of your weight and vital signs.
- You will be asked about any side effects you may have had.
- Blood (about 2 tablespoons) will be drawn for routine tests.
- Blood (about 1 teaspoon) will be drawn for PK testing.
- Blood (about 1 tablespoon) will be drawn to check your immune system.
If your blood tests show that you may have fiber deposits (build-up) in the bone marrow, you
will have a bone marrow biopsy to check the status of the disease
If you have symptoms of cataracts (clouding of the lens of the eye) anytime during the
study, you will have an eye exam.
Length of Study:
You will take the study drug for up to 8 weeks. You will have study visits for 12 weeks.
You will be taken off study if you have intolerable side effects.
End-of-Treatment Visit:
After completion of study drug, you will have the following tests and procedures performed:
- You will have a physical exam, including measurement of your weight and vital signs.
- You will be asked about any side effects you may have had.
- Blood (about 3 tablespoons) will be drawn for routine tests.
- Blood (about 1 tablespoon) will be drawn to check your blood's clotting ability.
- Blood (about 1 tablespoon) will be drawn to check your immune system.
Weeks 10 and 12 (+/- 3 days), you will have the following tests and procedures performed:
- You will have a telephone interview.
- You will be asked about any side effects you may have had.
- Blood (about 3 tablespoons) will be drawn for routine tests.
- Blood (about 1 tablespoon) will be drawn to check your blood's clotting ability.
- You will either have blood drawn locally or where you live regionally.
Within the 30 days after your last dose of study drug, you will have a bone marrow biopsy to
check the status of the disease and within 3 months after your last dose of study drug, you
will have an eye exam.
This is an investigational study. Eltrombopag is FDA approved and commercially available
for the treatment of low platelet counts in patients with idiopathic thrombocytopenic
purpura (ITP -- a severe bleeding disease). Its use in patients with low platelet counts
after a stem cell transplant is investigational.
Up to 60 patients will take part in this study. All will be enrolled at MD Anderson.
Inclusion Criteria:
1. Patients >/= 35 days post HCT with Platelet count days or patients are platelet transfusion dependent, and
2. Neutrophil count >/= 1.5 x 10^9/l anytime within the last seven days before
enrollment. Patients can be on myeloid or erythroid growth factors for example
filgrastim), and
3. Age >/= 18
Exclusion Criteria:
1. M7 AML
2. Primary or secondary myelofibrosis
3. Patients with persistent acute myeloid leukemia (>5% bone marrow blasts) at the time
of transplant
4. Recurrence or progression of primary malignancy after HCT
5. ALT >/= 2.5 times the ULN
6. Serum bilirubin >2mg/dl (unless due to Gilbert's syndrome)
7. Documented deep vein thrombosis within 1 year before enrollment on the study, except
if upper arm thrombosis related to central venous catheters, within 3 months before
enrollment on the study.
8. ECOG Performance status >2
9. Pregnancy: Women of child-bearing potential and men must agree to use contraception
prior to study entry and for the duration of study participation. A woman of
child-bearing potential is defined as a woman who has not been naturally
post-menopausal for at least 12 consecutive months or with no previous surgical
sterilization. A negative pregnancy test result will be required before any study
drug is given.
Type of Study:
Interventional
Study Design:
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment
Outcome Measure:
Number of Patients with Platelet Count >/= 50 X 10^9/l at day 57 without platelet transfusion
Outcome Time Frame:
Baseline to Day 57
Safety Issue:
No
Principal Investigator
Uday Popat, MD
Investigator Role:
Study Chair
Investigator Affiliation:
UT MD Anderson Cancer Center
Authority:
United States: Food and Drug Administration
Study ID:
2009-0106
NCT ID:
NCT01000051
Start Date:
February 2010
Completion Date:
Related Keywords:
- Thrombocytopenia
- Transplant Thrombocytopenia
- Hematopoietic cell transplantation
- HCT
- Low platelet count
- Bleeding
- Blood clot
- Stem Cell Transplant
- Blood And Marrow Transplantation
- Eltrombopag
- Promacta
- Thrombocytopenia
Name | Location |
|---|---|
| UT MD Anderson Cancer Center | Houston, Texas 77030 |
