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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 : Active, not recruiting
First Posted : September 5, 2011
Last Update Posted : January 15, 2021
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

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:

PRIMARY OBJECTIVES:

I. To determine the efficacy of eltrombopag (eltrombopag olamine) for patients with chronic myeloid leukemia (CML) or myelofibrosis (MF) who have developed thrombocytopenia during the course of therapy with tyrosine kinase inhibitors (TKI) as measured by recovery of platelet count.

SECONDARY OBJECTIVES:

I. To determine the safety of eltrombopag for patients with CML or MF who have developed thrombocytopenia during the course of therapy with TKI.

II. To determine the dose intensity of TKI after start of therapy with eltrombopag.

III. To determine response to TKI after start of therapy with eltrombopag.

OUTLINE:

Patients receive eltrombopag olamine orally (PO) once daily (QD) in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up at 30 days.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 39 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
Estimated Primary Completion Date : January 31, 2021
Estimated Study Completion Date : January 31, 2022


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




Primary Outcome Measures :
  1. Efficacy of eltrombopag olamine to increase platelet count as indicated by at least 30% of subjects having a complete (platelet) response: proportions of subjects with complete (platelet) response [ Time Frame: Up to 30 days after the last dose of study drug ]
    The proportions of subjects with complete (platelet) response will be reported together with exact 95% confidence intervals. The denominator will include all subjects who received eltrombopag olamine. Platelet counts over time and in relationship to exposure to eltrombopag and tyrosine kinase inhibitor (TKI) will be summarized using descriptive statistics.


Secondary Outcome Measures :
  1. Risk of leukemia defined as suboptimal response and progression to accelerated or blastic phase over time [ Time Frame: Up to 30 days after the last dose of study drug ]
    Kaplan-Meier methods will be used.

  2. Determining the extent of exposure to eltrombopag olamine [ Time Frame: Up to 30 days after the last dose of study drug ]
    Summarized using descriptive statistics. Number of days needed for response and number of days on Eltrombopag

  3. Number of subjects with adverse events, serious adverse events, and adverse events leading to discontinuation, scored using Common Terminology Criteria for Adverse Events version 4.0 [ Time Frame: Up to 30 days after the last dose of study drug ]
    Adverse events will be reported by type, severity and frequency.



Information from the National Library of Medicine

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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.)

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
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 )
P30CA016672 ( U.S. NIH Grant/Contract )
First Posted: September 5, 2011    Key Record Dates
Last Update Posted: January 15, 2021
Last Verified: January 2021

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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
Primary Myelofibrosis
Thrombocytopenia
Neoplasms by Histologic Type
Neoplasms
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
Blood Platelet Disorders
Cell Transformation, Neoplastic
Carcinogenesis
Neoplastic Processes
Pathologic Processes