Eltrombopag in Elderly Acute Myelogenous Leukemia (AML)
|ClinicalTrials.gov Identifier: NCT01113502|
Recruitment Status : Active, not recruiting
First Posted : April 30, 2010
Last Update Posted : July 19, 2016
|Condition or disease||Intervention/treatment||Phase|
|Male and Female Subjects, Greater Than 60 Years of Age With Non-M3 AML||Drug: Eltrombopag||Phase 1 Phase 2|
Primary Objectives (Phase I Portion): 1). To determine the safety and tolerability of eltrombopag in elderly subjects with AML 2). To determine the maximally tolerated initial starting dose of eltrombopag for elderly subjects with AML Primary Objectives (Phase II portion): 1). To better define the safety and tolerability of eltrombopag in elderly patients with AML at the maximally tolerated starting dose Page 9 of 18 determined in Phase I portion of study. 2). To determine the incidence of peripheral platelet count improvement (using baseline and response parameters as defined below) for subjects with disease related thrombocytopenia. Secondary Objectives (Phase I and II): 1). To preliminarily determine the efficacy (using AML response criteria as defined below) of eltrombopag in elderly subjects with AML.
2). To perform ex-vivo analyses using subject AML samples and stock eltrombopag to 1) assess leukemic proliferative capacity and 2) investigate potential eltrombopag induced cytoxic mechanisms for leukemic cell death. 3). To perform pharmacodynamic assessments of drug activity in leukemic cells using subject samples collected at various time points before and during drug exposure. 4). To preliminarily correlate pharmacodynamic findings with clinical response.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||44 participants|
|Official Title:||A Phase I/II Study of Eltrombopag in Elderly Patients With AML|
|Study Start Date :||April 2010|
|Actual Primary Completion Date :||March 2013|
- Phase I PortionThe maximally tolerated initial starting dose of eltrombopag for elderly subjects with AML. Clinical assessment and laboratory evaluation of Adverse Events and DLTs will be done according to the Common Terminology Criteria for Adverse Events (CTCAE), version 4.0 of the National Cancer Institute (NCI) Cancer Therapy Evaluation Program(CTEP).
- Phase II portion:The safety and tolerability of eltrombopag in elderly patients with AML at the maximally tolerated starting dose determined in Phase I portion of study. Clinical assessment and laboratory evaluation of Adverse Events and DLTs will be done according to the Common Terminology Criteria for Adverse Events (CTCAE), version 4.0 of the National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP).
- Overall response rate (Phase I and Phase II)This will include subjects who achieve a complete remission (CR), CR with incomplete count recovery, partial response (PR), and hematologic improvement (HI) based on definitions by the International Working Group (IWG) Of note: subjects who only have HI-platelet (HIp) will not be considered to have had a disease response as this may be due to direct stimulation of meagkaryocytes and not cytotoxic effect
- Survival, Response and Progression
Overall survival (OS) is defined as the time from first day of therapy to death due to any cause or last patient contact.
Progression-free survival (PFS) is defined as the time from first day of therapy to disease progression, death due to any cause, or last patient contact.
PFS is defined for all patients entered on the study, following an intent-to-treat analysis plan.
Relapse free survival (RFS) Time from complete remission to disease relapse. The time to response is defined as the time from the first day of therapy until the time that measurement criteria are met for response.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01113502
|United States, Pennsylvania|
|Abramson Cancer Center of the University of Pennsylvania|
|Philadelphia, Pennsylvania, United States, 19104|
|Principal Investigator:||Noelle Frey, MD||Abramson Cancer Center of the University of Pennsylvania|