Overview

This trial is active, not recruiting.

Condition idiopathic thrombocytopenic purpura
Treatment promacta (eltrombopag)
Phase phase 2
Sponsor Weill Medical College of Cornell University
Collaborator GlaxoSmithKline
Start date May 2009
End date January 2014
Trial size 12 participants
Trial identifier NCT00902018, 0809009980

Summary

The purpose of this study is to further evaluate the effects that eltrombopag has on platelets in subjects with chronic ITP. Eltrombopag is approved by the Food and Drug Administration (FDA) for the treatment of low platelets in patients with chronic ITP. It is being further studied by GlaxoSmithKline in other conditions associated with low platelets. This research study is being done because eltrombopag has been shown to increase platelet counts in a different way than other therapies for ITP. The investigators want to further study how eltrombopag affects subjects and their platelets to determine how the study drug should best be used in ITP treatment.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Other)
No Arm
promacta (eltrombopag)
Subjects will be treated with eltrombopag 75 mg once daily. Patients will be monitored 3 times a week for the first 2 weeks, and then monitored as clinically indicated as they continue eltrombopag dosing for 3-4 months.

Primary Outcomes

Measure
To determine how eltrombopag affects platelet counts.
time frame: 2-4 weeks.

Secondary Outcomes

Measure
To determine effect of eltrombopag on platelet apoptosis.
time frame: First two weeks of study.
To determine how eltrombopag affects platelet function and platelet survival.
time frame: First two weeks of study.
To continue to assess the safety and efficacy of eltrombopag.
time frame: First two weeks of study.

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: - Subject has signed and dated a written informed consent - Male or female adults (≥18 years) diagnosed with either primary ITP according to the American Society for Hematology or British Committee for Standards in Haematology (ASH/BCSH) guidelines [Blood, 1996; British Journal of Haematology, 2003] for at least three months prior to study entry or with ITP secondary to Evans syndrome, SLE, or Common Variable Immunodeficiency (including hypogammaglobulinemia). - Subjects must have responded with a platelet count > 30,000/µL to a previous ITP therapy including thrombopoietic agents. - Platelet count < 30,000/µL - Female subjects of childbearing potential are practicing an acceptable method of contraception or are completely abstinent from intercourse. Exclusion Criteria: - Active infection - Previously treated with thrombopoietic agents IF either no response at a therapeutic dose (peak platelet count < 50k) OR treatment with the agent within the past 4 weeks - Currently treated with concomitant ITP medication that has not been stable in dose for at least 2 weeks - only prednisone, azathioprin, and danazol are allowed. - Female subjects who are nursing or pregnant - Thrombosis of any kind within past 6 months or on blood thinners because of thrombosis. - IVIG, IV anti-D, bolus corticosteroids or vinca alkaloids within the past week - Other cytotoxic or immunosuppressive ITP therapy within the past 8 weeks or rituximab within the past 12 weeks - Active non-dermatologic malignancy defined as presence of known tumor ie. visible by radiography or evident on blood or bone marrow testing OR receiving chemotherapy within past 2 months - Hemoglobin < 10 gm/dl or WBC < 2,500/ul - Liver function tests (ALT, AST, or T Bili) > 3X ULN - Creatinine > 2X ULN

Additional Information

Official title The Effect of Eltrombopag on Platelet Survival: the Role of the BcL-xL Pathway
Principal investigator James B Bussel, MD
Description The Bcl-xL/Bak balance has been identified as an intrinsic mechanism that is critical in determining platelet lifespan (Mason, Cell 2007). There is evidence that Bcl-xL protein expression in megakaryocytes is regulated by TPO-mediated activation of Akt pathways mediated by Jak2 and Stat 5 (possibly by Stat 3 as well). (e.g. Kozuma et al, J Thromb Haemost 2007). Little is known about the Bcl-xL / Bak axis in patients with ITP, or the effect of TPO-R stimulation on platelet survival in patients with ITP. The TPO effect may be a result of stimulation of TPO-R signalling in megakaryocytes altering the packaging of Bcl-xl into platelets, or be a direct effect of platelet TPO-R stimulation as described above.
Trial information was received from ClinicalTrials.gov and was last updated in November 2012.
Information provided to ClinicalTrials.gov by Weill Medical College of Cornell University.