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

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.

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Key information

Conditions

Thrombocytopenia Acute Radiation Syndrome Blood Platelet Disorders Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Radiation Injuries Wounds and Injuries

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

eltrombopag

Drug

dose escalation

Primary outcomes

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 outcomes

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.
Median Number of Platelet Transfusions up to the Day of Engraftment

Time frame: baseline to day of engraftment

Sponsors and collaborators

Lead sponsor

Jane Liesveld

Other

Registry information

Official study title

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

Important dates

Study start: 2009
Primary completion: 2015
Study completion: 2015
First posted: May 19, 2009
Registry last updated: May 5, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed