Eltrombopag for Management of Thrombocytopenia

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by M.D. Anderson Cancer Center
Sponsor:
Collaborator:
GlaxoSmithKline
Information provided by (Responsible Party):
M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier:
NCT01428635
First received: September 1, 2011
Last updated: May 9, 2014
Last verified: May 2014
  Purpose

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.


Condition Intervention Phase
Leukemia
Drug: Eltrombopag
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Eltrombopag for the Management of Thrombocytopenia Associated With Tyrosine Kinase Therapy in Patients With Chronic Myeloid Leukemia (CML)

Resource links provided by NLM:


Further study details as provided by M.D. Anderson Cancer Center:

Primary Outcome Measures:
  • Number of Participants with Complete Platelet Response [ Time Frame: Every 3 to 4 months (Baseline to 3 months confirmed Complete Platelet Response, up to 18 months following treatment initiation.) ] [ Designated as safety issue: Yes ]
    Complete (platelet) response defined as a sustained (3 months) platelet count of ≥ 50 x 109/L and at least a 20% increase in platelet count from baseline with no more than 3 counts in that period being ≤ 50 * 109/L , while continuing imatinib or other TKI therapy. Complete Blood Counts (CBCs), including platelet count, weekly at beginning of treatment or with dose adjustments until stable platelet count achieved, monthly thereafter.


Estimated Enrollment: 39
Study Start Date: January 2012
Estimated Primary Completion Date: January 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Eltrombopag
Starting dose 50 mg by mouth daily. Dose escalation allowed every 2 weeks to 100 mg, 150 mg, 200 mg and 300 mg according to platelet response (except for patients of East Asian ancestry who receive starting dose of 25 mg daily). Treatment cycle defined as daily continuous dosing.
Drug: Eltrombopag
Starting dose 50 mg by mouth daily. Dose escalation allowed every 2 weeks to 100 mg, 150 mg, 200 mg and 300 mg according to platelet response (except for patients of East Asian ancestry who receive starting dose of 25 mg daily). Treatment cycle defined as daily continuous dosing.
Other Name: Promacta

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  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. CML patients in chronic phase receiving treatment with any 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%.
  2. 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.
  3. Subject is anticipated to have therapy with TKI continued for >/= 3 months
  4. Adequate organ function: Total bilirubin (except for Gilbert's Syndrome) </= 1.5 x ULN; ALT and AST < 3 x ULN; Creatinine </= 2 x ULN

Exclusion Criteria:

  1. 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.
  2. Thrombocytopenia that is considered to be unrelated to treatment with TKI or accelerated phase as defined above;
  3. Age < 18 years;
  4. Stem cell transplantation within preceding 60 days prior to registration;
  5. Patients with documented active hepatitis B or C infection;
  6. Patients with known bone marrow reticulin fibrosis (only applicable to patients with CML);
  7. Patients with palpable splenomegaly >/= 16cm below coastal margin (only applicable to patients with CML).
  8. Female subjects who are pregnant or breastfeeding. Women of childbearing potential are required to have a 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: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) 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.
  9. Patients with known risk factors for thromboembolism (e.g. Factor V Leiden mutation, ATIII deficiency, Protein C and S deficiency, antiphospholipid syndrome, portal hypertension, etc.)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01428635

Contacts
Contact: Gautam Borthakur, MBBS 713-563-1586

Locations
United States, Texas
UT MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
GlaxoSmithKline
Investigators
Principal Investigator: Gautam Borthakur, MBBS UT MD Anderson Cancer Center
  More Information

Additional Information:
No publications provided

Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT01428635     History of Changes
Other Study ID Numbers: 2011-0319, NCI-2011-03336
Study First Received: September 1, 2011
Last Updated: May 9, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by M.D. Anderson Cancer Center:
Leukemia
Thrombocytopenia
Tyrosine kinase therapy
TKI
Chronic myeloid leukemia
CML
Platelet count
Clonal evolution
Eltrombopag
Promacta

Additional relevant MeSH terms:
Leukemia
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Thrombocytopenia
Blood Platelet Disorders
Bone Marrow Diseases
Hematologic Diseases
Leukemia, Myeloid
Myeloproliferative Disorders
Neoplasms
Neoplasms by Histologic Type

ClinicalTrials.gov processed this record on October 20, 2014