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

Primary Purpose

Thrombocytopenia, Acute Radiation Syndrome

Status

Completed

Phase

Phase 1

Locations

Study Type

Interventional

Intervention

Eltrombopag

About this trial

This is an interventional treatment trial for Thrombocytopenia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Eltrombopag

Arm Description

Outcomes

Primary Outcome Measures

Maximum Tolerated Dose (MTD) of Eltrombopag

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

Median Time to Platelet Engraftment

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

Full Information

NCT ID

NCT00903929

First Posted

May 15, 2009

Last Updated

April 1, 2016

Sponsor

Jane Liesveld

1. Study Identification

Unique Protocol Identification Number

NCT00903929

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2016

Overall Recruitment Status

Completed

Study Start Date

December 2009 (undefined)

Primary Completion Date

December 2015 (Actual)

Study Completion Date

December 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor-Investigator

Name of the Sponsor

Jane Liesveld

4. Oversight

Data Monitoring Committee

Yes

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Thrombocytopenia, Acute Radiation Syndrome

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

19 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Eltrombopag

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

Eltrombopag

Intervention Description

dose escalation

Primary Outcome Measure Information:

Title

Maximum Tolerated Dose (MTD) of Eltrombopag

Description

Time Frame

1.5 years

Secondary Outcome Measure Information:

Title

Median Time to Platelet Engraftment

Description

Time Frame

1.5 years

Title

Median Number of Platelet Transfusions up to the Day of Engraftment

Time Frame

baseline to day of engraftment

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Yuhchyau Chen, MD,PhD

Organizational Affiliation

University of Rochester

Official's Role

Study Director

First Name & Middle Initial & Last Name & Degree

Jane Liesveld, MD

Organizational Affiliation

University of Rochester

Official's Role

Principal Investigator

12. IPD Sharing Statement

Citations:

PubMed Identifier

24120380

Citation

Liesveld JL, Phillips GL 2nd, Becker M, Constine LS, Friedberg J, Andolina JR, Milner LA, DeBolt J, Smudzin T, Hyrien O, Erickson-Miller CL, Johnson BM, Dawson KL, Chen Y. A phase 1 trial of eltrombopag in patients undergoing stem cell transplantation after total body irradiation. Biol Blood Marrow Transplant. 2013 Dec;19(12):1745-52. doi: 10.1016/j.bbmt.2013.10.002. Epub 2013 Oct 8.

Results Reference

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

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