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

NCT01428635 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: 2011-0319NCI-2011-03336
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

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.

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

Outcome Measures

Primary Outcomes (1)

• 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.

  Up to 9 years

Secondary Outcomes (1)

• Number of Participants With a Response to TKI Therapy After Eltrombopag

  Up to 9 years

Study Arms (1)

Supportive care (eltrombopag olamine)

EXPERIMENTAL

Patients receive eltrombopag olamine PO QD in the absence of disease progression or unacceptable toxicity.

Drug: Eltrombopag Olamine

Interventions

Eltrombopag Olamine DRUG

Given PO

Also known as: Promacta, SB-497115-GR

Supportive care (eltrombopag olamine)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.)

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

M D Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• University of Texas MD Anderson Cancer Center Website

MeSH Terms

Conditions

Leukemia, Myeloid, Accelerated Phase; Blast Crisis; Leukemia, Myeloid, Chronic-Phase; Primary Myelofibrosis; Thrombocytopenia

Interventions

eltrombopag

Condition Hierarchy (Ancestors)

Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Cell Transformation, Neoplastic; Carcinogenesis; Neoplastic Processes; Blood Platelet Disorders; Cytopenia

Results Point of Contact

• Title: Gautam Borthakur MD./Professor
• Organization: The University of Texas MD Anderson Cancer Center

GBorthak@mdanderson.org

713-563-1586

Study Officials

• Gautam Borthakur

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 1, 2011
• First Posted: September 5, 2011
• Study Start: January 13, 2012
• Primary Completion: January 3, 2022
• Study Completion: January 3, 2022
• Last Updated: September 26, 2023
• Results First Posted: February 1, 2023

Record last verified: 2023-09

Locations

US (1)