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

Thrombocytopenia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT00903929
• Other study ID #: RSRB00028043
• Status: Active, not recruiting
• Phase: Phase 1
• First received: May 15, 2009
• Last updated: October 26, 2015
• Start date: December 2009

Study information

• Verified date: April 2012
• Source: University of Rochester
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 27
• Est. primary completion date: December 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Study Design

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Acute Radiation Syndrome
• Thrombocytopenia
• Thrombocytosis

Intervention

Drug:
• Eltrombopag
dose escalation

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Jane Liesveld

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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		1.5 years	Yes
Secondary	to collect preliminary efficacy data for accelerating the recovery of megakaryopoiesis and meaningful platelet production by eltrombopag		1.5 years	No