Phase I Study of Eltrombopag for Promoting Thrombopoiesis in Patients Undergoing Stem Cell Transplantation After Total Body Irradiation

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2011 by University of Rochester
Sponsor:
Information provided by:
University of Rochester
ClinicalTrials.gov Identifier:
NCT00903929
First received: May 15, 2009
Last updated: August 2, 2011
Last verified: August 2011
  Purpose

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 Intervention Phase
Thrombocytopenia
Acute Radiation Syndrome
Drug: Eltrombopag
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by University of Rochester:

Primary Outcome Measures:
  • to determine the safety, side effect profile, and toxicity of eltrombopag when administered to patients undergoing stem cell transplantation from autologous or allogeneic donor sources, who have received myeloablative doses of TBI [ Time Frame: 1.5 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • to collect preliminary efficacy data for accelerating the recovery of megakaryopoiesis and meaningful platelet production by eltrombopag [ Time Frame: 1.5 years ] [ Designated as safety issue: No ]

Estimated Enrollment: 27
Study Start Date: December 2009
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Eltrombopag Drug: Eltrombopag
dose escalation

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
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
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: NCT00903929

Contacts
Contact: Therese Smudzin, BS CCRC 585-275-7848 therese_smudzin@urmc.rochester.edu

Locations
United States, New York
University of Rochester, Dept. Radiation Oncology Recruiting
Rochester, New York, United States, 14642
Contact: Yuhchayau Chen, MD, Ph.D.    585-275-0793    yuhchyau_chen@urmc.rochester.edu   
Principal Investigator: Yuhchyau Chen, MD, Ph.D         
University of Rochester, Medical Center Recruiting
Rochester, New York, United States, 14642
Sponsors and Collaborators
University of Rochester
  More Information

No publications provided by University of Rochester

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Yuhchyau Chen MD,PhD, University of Rochester
ClinicalTrials.gov Identifier: NCT00903929     History of Changes
Other Study ID Numbers: RSRB00028043
Study First Received: May 15, 2009
Last Updated: August 2, 2011
Health Authority: United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on October 29, 2014