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Eltrombopag Olamine in Treating Thrombocytopenia in Patients With Chronic Myeloid Leukemia or Myelofibrosis Receiving Tyrosine Kinase Therapy

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ClinicalTrials.gov Identifier: NCT01428635
Recruitment Status : Completed
First Posted : September 5, 2011
Results First Posted : February 1, 2023
Last Update Posted : February 1, 2023
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Study Description
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Brief 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. Eltrombopag olamine may cause the body to make platelets after receiving treatment for chronic myeloid leukemia or myelofibrosis.

Condition or disease Intervention/treatment Phase
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 Drug: Eltrombopag Olamine Phase 2 Phase 3

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
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 21 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Supportive Care
Official Title: Eltrombopag for the Management of Thrombocytopenia Associated With Tyrosine Kinase Therapy in Patients With Chronic Myeloid Leukemia (CML) and Myelofibrosis (MF)
Actual Study Start Date : January 13, 2012
Actual Primary Completion Date : January 3, 2022
Actual Study Completion Date : January 3, 2022

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Supportive care (eltrombopag olamine)
Patients receive eltrombopag olamine PO QD in the absence of disease progression or unacceptable toxicity.
 Drug: Eltrombopag Olamine
Given PO
Other Names:
  • Promacta
  • SB-497115-GR


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Number of Participants With a Response to TKI Therapy After Eltrombopag [ Time Frame: Up to 9 years ]
    (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.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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)
  • 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.)
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01428635


Locations
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United States, Texas
M D Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
National Cancer Institute (NCI)
Investigators
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Principal Investigator: Gautam Borthakur M.D. Anderson Cancer Center
  Study Documents (Full-Text)

Documents provided by M.D. Anderson Cancer Center:
Study Protocol and Statistical Analysis Plan  [PDF] August 14, 2018

More Information
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Additional Information:

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Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT01428635    
Other Study ID Numbers: 2011-0319
NCI-2011-03336 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
2011-0319 ( Other Identifier: M D Anderson Cancer Center )
First Posted: September 5, 2011    Key Record Dates
Results First Posted: February 1, 2023
Last Update Posted: February 1, 2023
Last Verified: January 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Leukemia
Leukemia, Myeloid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Leukemia, Myeloid, Chronic-Phase
Leukemia, Myeloid, Accelerated Phase
Blast Crisis
Thrombocytopenia
Primary Myelofibrosis
Neoplasms by Histologic Type
 Neoplasms
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
Blood Platelet Disorders
Cell Transformation, Neoplastic
Carcinogenesis
Neoplastic Processes
Pathologic Processes