LeukemiaPhase 2NCT02010645
Eltrombopag With Decitabine in Advanced Myelodysplastic Syndrome (MDS)
A Phase 2 study testing Eltrombopag and Decitabine in people with Leukemia, 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 given in combination with decitabine can help to control advanced MDS. The safety of this study drug combination will also be studied. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia
- What is being tested?
- Eltrombopag and Decitabine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2014to 2 Jan 2019
- 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
- Signed, informed consent must be obtained prior to any study specific procedures.
- Subjects with a histologically confirmed diagnosis of MDS by FAB criteria, including both MDS and RAEB-T (AML with 20-30% blasts and multilineage dysplasia) and chronic myelomonocytic leukemia (CMML) with at least 10% bone marrow blasts by World Health Organization (WHO) classification are eligible.
- Advanced MDS by virtue of intermediate-2 or high-risk MDS by IPSS score, or high or very-high risk by IPSS-R.
- Platelet count </= 100 x 10^9/L at baseline
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2
- Adequate liver function, as evidenced by a serum bilirubin </= 2x the ULN (except for patients with a confirmed diagnosis of Gilbert's Disease) and an ALT or AST </= 3x the laboratory ULN.
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- Serum creatinine </= 2.5x upper limit of normal
- Subjects must be >/= 18 years of age at the time of informed consent
- Subject is practicing an acceptable method of contraception (documented in chart). Female subjects (or female partners of male subject) must either be of non-childbearing potential (hysterectomy, bilateral oophorectomy, bilateral tubal ligation or post-menopausal > 1 year), or of childbearing potential and use one of the following highly effective methods of contraception (i.e. Pearl index < 1.0%) from 2 weeks prior to administration of study medication, throughout the study, and 28 days after completion or premature discontinuation from the study: - Complete abstinence from intercourse; - Intrauterine device (IUD); - Two forms of barrier contraception (diaphragm plus spermicide, and for males condom plus spermicide); - Male partner is sterile prior to entry into the study and is the only partner of the female; - Systemic contraceptives (combined or progesterone only).
Who cannot join
- Subjects with any prior exposure to a thrombopoietin-receptor agonist
- Prior hypomethylating agent treatment for MDS
- Any prior or co-existing medical condition that in the investigator's judgment will substantially increase the risk associated with the subject's participation in the study
- Psychiatric disorders or altered mental status precluding understanding of the informed consent process and/or completion of the necessary study procedures
- Active uncontrolled serious infection or sepsis at study enrollment
- Clinically significant gastrointestinal disorders that may interfere with absorption of drug.
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- History of arterial thrombosis (i.e. stroke) in the past year
- History of venous thrombosis currently requiring anti-coagulation therapy
- Unstable angina, congestive heart failure (New York Heart Association (NYHA) > Class II), uncontrolled hypertension (diastolic blood pressure > 100mmHg), or recent (within 1 year) myocardial infarction
- Subjects with a QTc > 480 msec (QTc > 510 msec for subjects with Bundle Branch Block) at baseline
- Pregnant or breast-feeding
- Subjects with known history of human immunodeficiency virus (HIV) or active infection with hepatitis C virus (HCV) or hepatitis B virus (HBV), because eltrombopag is hepatically cleared, and underlying hepatic impairment may lead to an increased risk of hepatotoxicity. Eltrombopag has not been evaluated with combination antiretroviral regimens.
- Subjects with liver cirrhosis (as determined by the investigator)
- Subjects with hypersensitivity to study drugs or their excipients.
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
Official title: Phase II Study of Eltrombopag in Combination With Decitabine in Subjects With Advanced Myelodysplastic Syndrome
The goal of this clinical research study is to learn if eltrombopag given in combination with decitabine can help to control advanced MDS. The safety of this study drug combination will also be studied.
Read the detailed description
Study Drug Administration:
Each cycle is 28 days.
If you are found to be eligible to take part in this study, you will receive decitabine by vein over 1 hour on Days 1-5 of each cycle.
You will also take eltrombopag capsules by mouth 1 time every day of each cycle. You should take it on an empty stomach (1 hour before a meal or 2 hours after a meal) with 8 ounces (1 cup) of water.
Do not eat calcium-rich foods (such as dairy products and juices with added calcium), or take other drugs (such as antacids) or supplements containing iron, calcium, aluminum, magnesium, selenium, and/or zinc for 4 hours before or 4 hours after taking eltrombopag.
If a dose of eltrombopag is vomited, you should not make it up or re-take it on the same day. If the morning dose is missed, it may be taken up until 5:00 PM on the same day.
Study Visits:
On Day 1 of each cycle:
- You will have a physical exam.
- Blood (about 2-3 teaspoons) will be drawn for routine tests.
On Days 8, 15, and 22 of Cycle 1 only, blood (about 2-3 teaspoons) will be drawn for routine tests.
If the doctor thinks it is needed, on Day 1 of Cycles 2-4, then every 3 cycles (Cycles 7, 10, 13, and so on), you will also have a bone marrow aspirate/biopsy to check the status of the disease and for cytogenetic testing.
If the doctor thinks it is needed, once each week blood (about 2-3 teaspoons) will be drawn for routine tests.
End-of-Treatment Visit:
Within 5 days of your last dose of study drug, you will come to the clinic for an end-of-treatment visit. The following procedures will be performed:
- You will have a physical exam.
- Blood (about 2-3 teaspoons) will be drawn for routine tests.
- You will have a bone marrow aspirate/biopsy to check the status of the disease and for cytogenetic testing.
Length of Study:
You may continue taking the study drugs for as long as the doctor thinks it is in your best interest. You will no longer be able to take the study drugs if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.
Your participation on the study will be over after the follow-up visits.
Follow-up Visit:
About 28 days after your last dose of study drugs, you will come to the clinic for a follow-up visit. The following procedures will be performed:
- You will have a physical exam.
- Blood (about 2-3 teaspoons) will be drawn for routine tests.
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). Decitabine is FDA approved for the treatment of MDS and is commercially available. The combination of eltrombopag and decitabine to treat MDS is investigational.
Up to 50 patients will take part in this study. All will be enrolled at MD Anderson.
- Conditions
- Leukemia
- Being tested
- Eltrombopag, Decitabine
- Keywords
- Leukemia, Advanced Myelodysplastic Syndrome, MDS, Intermediate-2 Myelodysplastic Syndrome, High-Risk Myelodysplastic Syndrome, Chronic myelomonocytic leukemia, CMML, Eltrombopag, Promacta, Decitabine, Dacogen
- Medical subject headings
- Leukemia, Leukemia, Myelomonocytic, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EltrombopagDrug
Also called Promacta
Starting dose of Eltrombopag is 100 mg by mouth daily for each 28 day cycle. East Asians will start at 50 mg by mouth daily for each 28 day cycle.
DecitabineDrug
Also called Dacogen
Starting dose of Decitabine is 20 mg/m2 by vein on Days 1-5 for each 28 day cycle.
| Study group | Role | What they receive |
|---|---|---|
| Eltrombopag + Decitabine | Experimental | Starting dose of Eltrombopag is 100 mg by mouth daily for each 28 day cycle. East Asians will start at 50 mg by mouth daily for each 28 day cycle.
Starting dose of Decitabine is 20 mg/m2 by vein on Days 1-5 for each 28 day cycle. Receives: Drug: Eltrombopag; Drug: Decitabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate (ORR)
The primary endpoint is the overall response rate (ORR) based on the IWG-2006 criteria, which includes complete remission (CR), partial remission (PR), and hematologic improvement (HI).
Measured: 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Dec 2013 (estimated)
- Study startMar 2014
- Primary completion2 Jan 2019
- Study completion2 Jan 2019
Why it stopped: PI Request
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 LeukemiaStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- GlaxoSmithKline
- Responsible party
- Sponsor
- Investigators
- Courtney DiNardo, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02010645
- Sponsor's protocol ID
- 2013-0590
- Other IDs
- NCI-2014-01276
- Status last verified
- Dec 2019
- First submitted
- 5 Dec 2013
- Last updated
- 2 Jan 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Eltrombopag in Combination With Decitabine in Subjects With Advanced Myelodysplastic Syndrome
- ClinicalTrials.gov ID
- NCT02010645
- Acronym / sponsor ID
- 2013-0590
- Conditions
- Leukemia
- Keywords
- Leukemia, Advanced Myelodysplastic Syndrome, MDS, Intermediate-2 Myelodysplastic Syndrome, High-Risk Myelodysplastic Syndrome, Chronic myelomonocytic leukemia, CMML, Eltrombopag
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: PI Request
- Enrolment
- 6 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- GlaxoSmithKline
- Study start
- Mar 2014 (actual)
- Primary completion
- 2 Jan 2019 (actual)
- Study completion
- 2 Jan 2019 (actual)
- First posted
- 13 Dec 2013 (estimated)
- Results
- Posted 2 Jan 2020
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 given in combination with decitabine can help to control advanced MDS. The safety of this study drug combination will also be studied.
Detailed description
Study Drug Administration:
Each cycle is 28 days.
If you are found to be eligible to take part in this study, you will receive decitabine by vein over 1 hour on Days 1-5 of each cycle.
You will also take eltrombopag capsules by mouth 1 time every day of each cycle. You should take it on an empty stomach (1 hour before a meal or 2 hours after a meal) with 8 ounces (1 cup) of water.
Do not eat calcium-rich foods (such as dairy products and juices with added calcium), or take other drugs (such as antacids) or supplements containing iron, calcium, aluminum, magnesium, selenium, and/or zinc for 4 hours before or 4 hours after taking eltrombopag.
If a dose of eltrombopag is vomited, you should not make it up or re-take it on the same day. If the morning dose is missed, it may be taken up until 5:00 PM on the same day.
Study Visits:
On Day 1 of each cycle:
- You will have a physical exam.
- Blood (about 2-3 teaspoons) will be drawn for routine tests.
On Days 8, 15, and 22 of Cycle 1 only, blood (about 2-3 teaspoons) will be drawn for routine tests.
If the doctor thinks it is needed, on Day 1 of Cycles 2-4, then every 3 cycles (Cycles 7, 10, 13, and so on), you will also have a bone marrow aspirate/biopsy to check the status of the disease and for cytogenetic testing.
If the doctor thinks it is needed, once each week blood (about 2-3 teaspoons) will be drawn for routine tests.
End-of-Treatment Visit:
Within 5 days of your last dose of study drug, you will come to the clinic for an end-of-treatment visit. The following procedures will be performed:
- You will have a physical exam.
- Blood (about 2-3 teaspoons) will be drawn for routine tests.
- You will have a bone marrow aspirate/biopsy to check the status of the disease and for cytogenetic testing.
Length of Study:
You may continue taking the study drugs for as long as the doctor thinks it is in your best interest. You will no longer be able to take the study drugs if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.
Your participation on the study will be over after the follow-up visits.
Follow-up Visit:
About 28 days after your last dose of study drugs, you will come to the clinic for a follow-up visit. The following procedures will be performed:
- You will have a physical exam.
- Blood (about 2-3 teaspoons) will be drawn for routine tests.
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). Decitabine is FDA approved for the treatment of MDS and is commercially available. The combination of eltrombopag and decitabine to treat MDS is investigational.
Up to 50 patients will take part in this study. All will be enrolled at MD Anderson.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Eltrombopag + Decitabine | Experimental | Drug: Eltrombopag, Drug: DecitabineStarting dose of Eltrombopag is 100 mg by mouth daily for each 28 day cycle. East Asians will start at 50 mg by mouth daily for each 28 day cycle. Starting dose of Decitabine is 20 mg/m2 by vein on Days 1-5 for each 28 day cycle. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Signed, informed consent must be obtained prior to any study specific procedures.
- Subjects with a histologically confirmed diagnosis of MDS by FAB criteria, including both MDS and RAEB-T (AML with 20-30% blasts and multilineage dysplasia) and chronic myelomonocytic leukemia (CMML) with at least 10% bone marrow blasts by World Health Organization (WHO) classification are eligible.
- Advanced MDS by virtue of intermediate-2 or high-risk MDS by IPSS score, or high or very-high risk by IPSS-R.
- Platelet count </= 100 x 10^9/L at baseline
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2
Key exclusion criteria (14 listed)
- Subjects with any prior exposure to a thrombopoietin-receptor agonist
- Prior hypomethylating agent treatment for MDS
- Any prior or co-existing medical condition that in the investigator's judgment will substantially increase the risk associated with the subject's participation in the study
- Psychiatric disorders or altered mental status precluding understanding of the informed consent process and/or completion of the necessary study procedures
- Active uncontrolled serious infection or sepsis at study enrollment
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 dose of Eltrombopag is 100 mg by mouth daily for each 28 day cycle. East Asians will start at 50 mg by mouth daily for each 28 day cycle. | Drug | Eltrombopag + Decitabine |
| DecitabineAlso: DacogenStarting dose of Decitabine is 20 mg/m2 by vein on Days 1-5 for each 28 day cycle. | Drug | Eltrombopag + Decitabine |
Primary outcome
Overall Response Rate (ORR)
Time frame: 28 days
The primary endpoint is the overall response rate (ORR) based on the IWG-2006 criteria, which includes complete remission (CR), partial remission (PR), and hematologic improvement (HI).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jan 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- University of Texas MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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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.
Courtney DiNardo, MD principal investigator
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 and Decitabine in people with Leukemia, 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 9 inclusion and 14 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: stopped early, as of the sponsor's last update on 2 Jan 2020. 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. 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 2 Jan 2020). 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?
Overall Response Rate (ORR) (time frame: 28 days). 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 2 Jan 2020.