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

NCT00903929 | Completed Phase 1 Results DMC

• 1 other identifier: RSRB00028043
• Study Type: interventional
• Target: 19
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Thrombocytopenia; Acute Radiation Syndrome

Outcome Measures

Primary Outcomes (1)

• 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.

  1.5 years

Secondary Outcomes (2)

• Median Time to Platelet Engraftment

  1.5 years

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

  baseline to day of engraftment

Study Arms (1)

Eltrombopag

EXPERIMENTAL

Drug: Eltrombopag

Interventions

Eltrombopag DRUG

dose escalation

Eltrombopag

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Jane Liesveld: lead

Related Publications (1)

• 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.

  PMID: 24120380 DERIVED

MeSH Terms

Conditions

Thrombocytopenia; Acute Radiation Syndrome

Interventions

eltrombopag

Condition Hierarchy (Ancestors)

Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia; Radiation Injuries; Wounds and Injuries

Results Point of Contact

• Title: Jane L. Liesveld
• Organization: University of Rochester

jane_liesveld@urmc.rochester.edu

585-275-5823

Study Officials

• Yuhchyau Chen, MD,PhD

  University of Rochester

  STUDY DIRECTOR

• Jane Liesveld, MD

  University of Rochester

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor M&D-Hematology/Oncology

Study Record Dates

• First Submitted: May 15, 2009
• First Posted: May 19, 2009
• Study Start: December 1, 2009
• Primary Completion: December 1, 2015
• Study Completion: December 1, 2015
• Last Updated: May 5, 2016
• Results First Posted: May 5, 2016

Record last verified: 2016-04