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

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:

Drug Information available for: Eltrombopag

U.S. FDA Resources

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:

Age ≥ 18
Able to give written informed consent for a clinical trial
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
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
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.
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).
Karnofsky performance status must be ≥70%.

Exclusion Criteria:

TBI dose less than 400 cGY
Cord blood transplantation
HIV infection
Pregnancy or breastfeeding
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
Active infection requiring systemic antibiotic therapy with antibacterial, antifungal, or antiviral agents
Concomitant enrollment in another therapeutic clinical study except with PI approval
Must not have previously received eltrombopag
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
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

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

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:

Radiation Injuries
Thrombocytopenia
Acute Radiation Syndrome

Wounds and Injuries
Blood Platelet Disorders
Hematologic Diseases

ClinicalTrials.gov processed this record on May 19, 2013