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Phase I Study of Eltrombopag for Promoting Thrombopoiesis After Total Body Irradiation

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00903929
Recruitment Status : Completed
First Posted : May 19, 2009
Results First Posted : May 5, 2016
Last Update Posted : May 5, 2016
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:

Patients who undergo total body irradiation (TBI) for stem cell transplantation have prolonged periods of low counts of specific blood cells called platelets. These low platelets counts can cause bleeding and infection. Thus far, no drug is available for use to speed the recovery of platelets, and therefore transfusions are often necessary.

The purpose of this study is to test the safety of a drug called eltrombopag in patients who have received TBI. The investigators want to find out what effects, good or bad, it has on people with low platelet counts due to treatment with TBI. The investigators will also be testing how well eltrombopag may work at different doses and determine if this drug speeds up the recovery of the platelets.


Condition or disease Intervention/treatment Phase
Thrombocytopenia Acute Radiation Syndrome Drug: Eltrombopag Phase 1

Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 19 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I Study of Eltrombopag for Promoting Thrombopoiesis in Patients Undergoing Stem Cell Transplantation After Total Body Irradiation
Study Start Date : December 2009
Primary Completion Date : December 2015
Study Completion Date : December 2015

Resource links provided by the National Library of Medicine

Drug Information available for: Eltrombopag
U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Experimental: Eltrombopag Drug: Eltrombopag
dose escalation


Outcome Measures

Primary Outcome Measures :
  1. Maximum Tolerated Dose (MTD) of Eltrombopag [ Time Frame: 1.5 years ]
    The MTD was defined as the highest dose if no dose limiting toxicity was observed, or the highest dose at which less than one-third of the patients experienced toxicities not expected in the standard stem cell transplantation setting.


Secondary Outcome Measures :
  1. Median Time to Platelet Engraftment [ Time Frame: 1.5 years ]
    Determined for all participants who completed at least 75% of the planned doses. Platelet engraftment was as defined by the Center for International Blood and Marrow Transplant Research as the first of 3 consecutive days of a platelet count 20,000/mL without platelet trans-fusions for 7 days and/or the first day of a platelet count 100,000/mL without platelet transfusions for 7 days.

  2. Median Number of Platelet Transfusions up to the Day of Engraftment [ Time Frame: baseline to day of engraftment ]

Eligibility Criteria

Information from the National Library of Medicine

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

Inclusion Criteria:

  1. Age ≥ 18
  2. Able to give written informed consent for a clinical trial
  3. Scheduled to undergo an autologous or an allogeneic stem cell transplantation from a sibling, related donor, or unrelated donor using a conditioning regimen containing at least 400 cGy TBI
  4. Transplantation is being performed for one of the following medical conditions:

    • Acute myelogenous leukemia
    • Acute lymphoblastic leukemia
    • Chronic myelogenous leukemia in chronic, accelerated, or blastic phase
    • Myelodysplastic syndrome
    • Myeloproliferative diseases such as chronic myelomonocytic leukemia, agnogeneic myeloid metaplasia with myelofibrosis, polycythemia vera, or essential thrombocythemia
    • Hodgkin's lymphoma
    • Non-Hodgkin's lymphoma
    • Multiple myeloma
    • Chronic lymphocytic leukemia
    • Other malignancies or marrow disease such as aplastic anemia where transplant would be appropriate with approval of principal investigator
  5. Patients with therapy-related AML or MDS may be included if their prior malignancy has been in remission for at least 12 months. If the remission is less than 12 months, approval of the principal investigator is required. Entry could be allowed if the malignancy is controlled and not expected to relapse e.g. localized prostate cancer treated with XRT.
  6. Patients must meet all other pre-transplantation criteria of the transplant center including acceptable tests of heart, liver, kidney, and lung function (Standard screening for HSCT per PI, and co-investigators).
  7. Karnofsky performance status must be ≥70%.

Exclusion Criteria:

  1. TBI dose less than 400 cGY
  2. Cord blood transplantation
  3. HIV infection
  4. Pregnancy or breastfeeding
  5. Creatinine or bilirubin or ALT or AST greater than two times the upper limit of normal, unless the abnormal bilirubin is due to Gilbert's syndrome
  6. Active infection requiring systemic antibiotic therapy with antibacterial, antifungal, or antiviral agents
  7. Concomitant enrollment in another therapeutic clinical study except with PI approval
  8. Must not have previously received eltrombopag
  9. Patients with moderate or severe liver disease (ALT, AST, or bilirubin ≥ 2X the upper limit of normal, unless the abnormal bilirubin is due to Gilbert's syndrome) will be excluded
  10. Patients with high risk of thromboembolism based on genetic syndromes , or past thromboembolic disease in the past 6 months will be excluded from the study, with the exception of those with catheter related clots
Contacts and Locations

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): NCT00903929


Sponsors and Collaborators
Jane Liesveld
Investigators
Study Director: Yuhchyau Chen, MD,PhD University of Rochester
Principal Investigator: Jane Liesveld, MD University of Rochester
More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Jane Liesveld, Professor M&D-Hematology/Oncology, University of Rochester
ClinicalTrials.gov Identifier: NCT00903929     History of Changes
Other Study ID Numbers: RSRB00028043
First Posted: May 19, 2009    Key Record Dates
Results First Posted: May 5, 2016
Last Update Posted: May 5, 2016
Last Verified: April 2016

Additional relevant MeSH terms:
Thrombocytopenia
Radiation Injuries
Acute Radiation Syndrome
Blood Platelet Disorders
Hematologic Diseases
Wounds and Injuries