Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 PositivePhase 2/3NCT01428635
Eltrombopag Olamine in Treating Thrombocytopenia in Patients With Chronic Myeloid Leukemia or Myelofibrosis Receiving Tyrosine Kinase Therapy
A Phase 2/3 study testing Eltrombopag Olamine in people with Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive and Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
This phase II/III trial studies how well eltrombopag olamine works in treating thrombocytopenia in patients with chronic myeloid leukemia or myelofibrosis receiving tyrosine kinase inhibitor therapy. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
- What is being tested?
- Eltrombopag Olamine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- 13 Jan 2012to 3 Jan 2022
- Phase
- Phase 2/3
- 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
- CML patients in chronic phase receiving treatment with any Food and Drug Administration (FDA) approved TKI; or CML patients in accelerated or blastic phase who are considered to be in this phase because of thrombocytopenia or because of clonal evolution and with no other criteria for accelerated/blastic phase or patients with myelofibrosis receiving treatment with FDA approved TKI and with peripheral blood and/or bone marrow blasts =< 10%
- Grade >= 3 thrombocytopenia (platelets < 50 x 10^9/L) after the first 3 months of therapy with the TKI for patients with CML and platelets < 100 x 10^9/L for patients with MF after the first 3 months of therapy; thrombocytopenia must be either recurrent (i.e., second or greater episode of thrombocytopenia) or having required dose reductions of the TKI
- Subject is anticipated to have therapy with TKI continued for >= 3 months
- Total bilirubin =< 1.5 x upper limit of normal (ULN) (except for Gilbert's syndrome)
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) < 3 x ULN
- Creatinine =< 2 x ULN
Who cannot join
- CML patients in accelerated or blastic phase except for those who are considered to be in this phase because of thrombocytopenia or because of clonal evolution and with no other criteria for accelerated/blastic phase; or myelofibrosis patients who have transformed to acute leukemia or have >= 10% blasts in peripheral blood and/or in bone marrow
- Thrombocytopenia that is considered to be unrelated to treatment with TKI or accelerated phase as defined above
- Stem cell transplantation within preceding 60 days prior to registration
- Patients with documented active hepatitis B or C infection
- Patients with known bone marrow reticulin fibrosis (>= grade 2) (only applicable to patients with CML)
- Patients with palpable splenomegaly >= 16 cm below coastal margin (only applicable to patients with CML)
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- Female subjects who are pregnant or breastfeeding
- Women of childbearing potential are required to have a beta human chorionic gonadotropin (BHCG) serum or urine pregnancy test performed within 7 days prior to first study drug dose; a female of childbearing potential is a sexually mature woman who:
- Has not undergone a hysterectomy or bilateral oophorectomy
- Has not been naturally postmenopausal for at least 12 consecutive months (i.e., has had menses at any time in the preceding 12 consecutive months)
- Women of child-bearing potential and men must agree to use contraception prior to study entry and for the duration of study participation
- Patients with known risk factors for thromboembolism (e.g. Factor V Leiden mutation, antithrombin III (ATIII) deficiency, Protein C and S deficiency, antiphospholipid syndrome, portal hypertension, etc.)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Eltrombopag for the Management of Thrombocytopenia Associated With Tyrosine Kinase Therapy in Patients With Chronic Myeloid Leukemia (CML) and Myelofibrosis (MF)
This phase II/III trial studies how well eltrombopag olamine works in treating thrombocytopenia in patients with chronic myeloid leukemia or myelofibrosis receiving tyrosine kinase inhibitor therapy. Eltrombopag olamine may cause the body to make platelets after receiving treatment for chronic myeloid leukemia or myelofibrosis.
Read the detailed description
The goal of this clinical research study is learn if eltrombopag can help control or prevent low platelet counts in patients receiving treatment for CML or myelofibrosis. This is an investigational study. Eltrombopag is FDA approved and commercially available for the treatment of patients with low platelet counts. The use of eltrombopag for the treatment of low platelet counts in patients with CML and myelofibrosis is investigational. Eltrombopag will be provided at no cost to you during the study.
If you are found to be eligible to take part in this study, you will receive eltrombopag by mouth 1 time a day. Your dose may be increased every 2 weeks depending on your platelet count response. You should take eltrombopag on an empty stomach. You should not eat for 2 hours before taking eltrombopag. You should wait at least 4 hours between taking eltrombopag and taking other drugs (like antacids), dairy products, juices with calcium added, or supplements containing iron, calcium, aluminum, magnesium, selenium, or zinc.
Up to 39 patients will take part in this study. All will be enrolled at MD Anderson. As of June 6, 2017, the study is closed to new participants.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Primary Myelofibrosis, Thrombocytopenia
- Being tested
- Eltrombopag Olamine
- Medical subject headings
- Leukemia, Myeloid, Accelerated Phase, Blast Crisis, Leukemia, Myeloid, Chronic-Phase, Primary Myelofibrosis, Thrombocytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Eltrombopag OlamineDrug
Also called Promacta, SB-497115-GR
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Supportive care (eltrombopag olamine) | Experimental | Patients receive eltrombopag olamine PO QD in the absence of disease progression or unacceptable toxicity. Receives: Drug: Eltrombopag Olamine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With a Platelet Response
The primary endpoint is complete (platelet) response (yes/no). A complete (platelet) response will be defined as a sustained (3 months) platelet count of > 50 x 109/L for patients with CML and > 100 x 109/L for patients with MF and at least a 20% increase in platelet count from baseline.
Measured: Up to 9 years
Secondary outcomes (1)
Number of Participants With a Response to TKI Therapy After Eltrombopag
(CML) : Complete Hematologic Remission : Normalization for 4 weeks of the bone marrow (< 5% blasts) and peripheral blood with WBC within normal institutional limits with no blasts, promyelocytes or myelocytes, and basophils <5%,. Complete cytogenetic response: Ph positive 0%. Partial cytogenetic response: Ph positive 1-35%. Minor cytogenetic response: Ph positive 36-90%. No cytogenetic response: Ph positive 100%. Myelofibrosis : Complete Remission: absence of transfusion or growth factor support: complete resolution of disease-related symptoms/signs including palpable hepatosplenomegaly, hemoglobin > 11 g/dL, platelet count ≥100 x 10^9/L, absolute neutrophil count ≥ 1.0 x 10^9/L. Normal leukocyte differential with disappearance of nucleated red blood cells and immature myeloid cells in peripheral smear, in the absence of splenectomy. Bone marrow histological remission:presence of age-adjusted normocellularity, < 5% myeloblast , osteomyelofibrosis grade </= 1.
Measured: Up to 9 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Sep 2011 (estimated)
- Study start13 Jan 2012
- Primary completion3 Jan 2022
- Study completion3 Jan 2022
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 Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 PositiveStudies 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 2/3
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Gautam Borthakur, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01428635
- Sponsor's protocol ID
- 2011-0319
- Other IDs
- NCI-2011-03336, 2011-0319
- Status last verified
- Sep 2023
- First submitted
- 1 Sep 2011
- Last updated
- 26 Sep 2023
- 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 26 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Eltrombopag for the Management of Thrombocytopenia Associated With Tyrosine Kinase Therapy in Patients With Chronic Myeloid Leukemia (CML) and Myelofibrosis (MF)
- ClinicalTrials.gov ID
- NCT01428635
- Acronym / sponsor ID
- 2011-0319
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Primary Myelofibrosis, Thrombocytopenia
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 13 Jan 2012 (actual)
- Primary completion
- 3 Jan 2022 (actual)
- Study completion
- 3 Jan 2022 (actual)
- First posted
- 5 Sep 2011 (estimated)
- Results
- Posted 1 Feb 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase II/III trial studies how well eltrombopag olamine works in treating thrombocytopenia in patients with chronic myeloid leukemia or myelofibrosis receiving tyrosine kinase inhibitor therapy. Eltrombopag olamine may cause the body to make platelets after receiving treatment for chronic myeloid leukemia or myelofibrosis.
Detailed description
The goal of this clinical research study is learn if eltrombopag can help control or prevent low platelet counts in patients receiving treatment for CML or myelofibrosis. This is an investigational study. Eltrombopag is FDA approved and commercially available for the treatment of patients with low platelet counts. The use of eltrombopag for the treatment of low platelet counts in patients with CML and myelofibrosis is investigational. Eltrombopag will be provided at no cost to you during the study.
If you are found to be eligible to take part in this study, you will receive eltrombopag by mouth 1 time a day. Your dose may be increased every 2 weeks depending on your platelet count response. You should take eltrombopag on an empty stomach. You should not eat for 2 hours before taking eltrombopag. You should wait at least 4 hours between taking eltrombopag and taking other drugs (like antacids), dairy products, juices with calcium added, or supplements containing iron, calcium, aluminum, magnesium, selenium, or zinc.
Up to 39 patients will take part in this study. All will be enrolled at MD Anderson. As of June 6, 2017, the study is closed to new participants.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Supportive care (eltrombopag olamine) | Experimental | Drug: Eltrombopag OlaminePatients receive eltrombopag olamine PO QD in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- CML patients in chronic phase receiving treatment with any Food and Drug Administration (FDA) approved TKI; or CML patients in accelerated or blastic phase who are considered to be in this phase because of thrombocytopenia or because of clonal evolution and with no other criteria for accelerated/blastic phase or patients with myelofibrosis receiving treatment with FDA approved TKI and with peripheral blood and/or bone marrow blasts =< 10%
- Grade >= 3 thrombocytopenia (platelets < 50 x 10^9/L) after the first 3 months of therapy with the TKI for patients with CML and platelets < 100 x 10^9/L for patients with MF after the first 3 months of therapy; thrombocytopenia must be either recurrent (i.e., second or greater episode of thrombocytopenia) or having required dose reductions of the TKI
- Subject is anticipated to have therapy with TKI continued for >= 3 months
- Total bilirubin =< 1.5 x upper limit of normal (ULN) (except for Gilbert's syndrome)
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) < 3 x ULN
Key exclusion criteria (12 listed)
- CML patients in accelerated or blastic phase except for those who are considered to be in this phase because of thrombocytopenia or because of clonal evolution and with no other criteria for accelerated/blastic phase; or myelofibrosis patients who have transformed to acute leukemia or have >= 10% blasts in peripheral blood and/or in bone marrow
- Thrombocytopenia that is considered to be unrelated to treatment with TKI or accelerated phase as defined above
- Stem cell transplantation within preceding 60 days prior to registration
- Patients with documented active hepatitis B or C infection
- Patients with known bone marrow reticulin fibrosis (>= grade 2) (only applicable to patients with CML)
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 |
|---|---|---|
| Eltrombopag OlamineAlso: Promacta, SB-497115-GRGiven PO | Drug | Supportive care (eltrombopag olamine) |
Primary outcome
Number of Participants With a Platelet Response
Time frame: Up to 9 years
The primary endpoint is complete (platelet) response (yes/no). A complete (platelet) response will be defined as a sustained (3 months) platelet count of > 50 x 109/L for patients with CML and > 100 x 109/L for patients with MF and at least a 20% increase in platelet count from baseline.
1 secondary outcome
Number of Participants With a Response to TKI Therapy After Eltrombopag
Time frame: Up to 9 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Sep 2023.
- 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.
Gautam Borthakur 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 2/3 study testing Eltrombopag Olamine in people with Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive and Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, 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 6 inclusion and 12 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 26 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 1 Feb 2023). 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?
Number of Participants With a Platelet Response (time frame: Up to 9 years). 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 26 Sep 2023.