ThrombocytopeniaPhase 2NCT01000051
Eltrombopag for Post Transplant Thrombocytopenia
A Phase 2 study testing Eltrombopag in people with Thrombocytopenia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
The goal of this clinical research study is to learn if eltrombopag can help to improve platelet counts in patients with low platelets after they have had a stem cell transplant. The safety of this drug will also be studied. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thrombocytopenia
- What is being tested?
- Eltrombopag
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 64 participants
- Where
- 1 siteUnited States
- Study dates
- 17 Feb 2010to 4 Apr 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients >/= 35 days post HCT with Platelet count </= 20 x 10^9/l sustained for 7 days or patients are platelet transfusion dependent, and
- 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
- Age >/= 18
Who cannot join
- Recurrence or progression of primary malignancy after HCT
- ALT >/= 2.5 times the ULN
- Serum bilirubin >2mg/dl (unless due to Gilbert's syndrome)
- 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.
- ECOG Performance status >2
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
About this study
The goal of this clinical research study is to learn if eltrombopag can help to improve platelet counts in patients with low platelets after they have had a stem cell transplant. The safety of this drug will also be studied.
Read the detailed description
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 3 months after your last dose of study drug, you will have a bone marrow biopsy to check the status of the disease and an eye exam. The eye exam may be performed sooner if you develop symptoms of a cataract, such as blurry vision.
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 64 patients will take part in this study. All will be enrolled at MD Anderson.
- Conditions
- Thrombocytopenia
- Being tested
- Eltrombopag, Placebo
- Keywords
- Transplant Thrombocytopenia, Hematopoietic cell transplantation, HCT, Low platelet count, Bleeding, Blood clot, Stem Cell Transplant, Blood And Marrow Transplantation, Eltrombopag, Promacta
- Medical subject headings
- Thrombocytopenia, Hemorrhage, Thrombosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EltrombopagDrug
Also called Promacta
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.
PlaceboDrug
Once a day, orally for 8 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Eltrombopag | Experimental | Starting dose 50 mg/day orally for 8 weeks Receives: Drug: Eltrombopag |
| Placebo | Placebo comparator | Once a day orally for 8 weeks Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Comparing the Efficacy of Eltrombopaq and Placebo
Comparison of the efficacy of eltrombopag and placebo in patients with thrombocytopenia post hematopoietic cell transplantation (HCT).
Measured: Baseline to Day 57
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2009 (estimated)
- Study start17 Feb 2010
- Primary completion4 Apr 2018
- Study completion4 Apr 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Uday Popat, MD, Study chair, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01000051
- Sponsor's protocol ID
- 2009-0106
- Other IDs
- NCI-2011-01099
- Status last verified
- Sep 2023
- First submitted
- 20 Oct 2009
- Last updated
- 18 Sep 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Eltrombopag for Post Transplant Thrombocytopenia
- ClinicalTrials.gov ID
- NCT01000051
- Acronym / sponsor ID
- 2009-0106
- Conditions
- Thrombocytopenia
- Keywords
- Transplant Thrombocytopenia, Hematopoietic cell transplantation, HCT, Low platelet count, Bleeding, Blood clot, Stem Cell Transplant, Blood And Marrow Transplantation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 64 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Novartis Pharmaceuticals
- Study start
- 17 Feb 2010 (actual)
- Primary completion
- 4 Apr 2018 (actual)
- Study completion
- 4 Apr 2018 (actual)
- First posted
- 22 Oct 2009 (estimated)
- Results
- Posted 30 Jul 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical research study is to learn if eltrombopag can help to improve platelet counts in patients with low platelets after they have had a stem cell transplant. The safety of this drug will also be studied.
Detailed description
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 3 months after your last dose of study drug, you will have a bone marrow biopsy to check the status of the disease and an eye exam. The eye exam may be performed sooner if you develop symptoms of a cataract, such as blurry vision.
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 64 patients will take part in this study. All will be enrolled at MD Anderson.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Eltrombopag | Experimental | Drug: EltrombopagStarting dose 50 mg/day orally for 8 weeks |
| Placebo | Placebo comparator | Drug: PlaceboOnce a day orally for 8 weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Patients >/= 35 days post HCT with Platelet count </= 20 x 10^9/l sustained for 7 days or patients are platelet transfusion dependent, and
- 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
- Age >/= 18
Key exclusion criteria (6 listed)
- Recurrence or progression of primary malignancy after HCT
- ALT >/= 2.5 times the ULN
- Serum bilirubin >2mg/dl (unless due to Gilbert's syndrome)
- 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.
- ECOG Performance status >2
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EltrombopagAlso: PromactaStarting 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. | Drug | Eltrombopag |
| PlaceboOnce a day, orally for 8 weeks. | Drug | Placebo |
Primary outcome
Comparing the Efficacy of Eltrombopaq and Placebo
Time frame: Baseline to Day 57
Comparison of the efficacy of eltrombopag and placebo in patients with thrombocytopenia post hematopoietic cell transplantation (HCT).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- University of Texas MD Anderson Cancer Center WebsiteLink provided by the sponsor.
Related trials for Thrombocytopenia
Enrolling by invitationObservational study
Screening for Hematology Branch Protocols
Hematologic Disease and Disorders, Donors
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
Not yet recruitingObservational study
Platelet Transfusion Thresholds in Non-Bleeding Patients
Non Bleeding Patients With Thrombocytopenia
RecruitingPhase 3
A Study of Nipocalimab in Reducing the Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
Thrombocytopenia, Neonatal Alloimmune
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Uday Popat, MD study chair
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Eltrombopag in people with Thrombocytopenia, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Jul 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Comparing the Efficacy of Eltrombopaq and Placebo (time frame: Baseline to Day 57). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Sep 2023.