Eltrombopag in Myelodysplastic Syndrome (MDS) Patients With Thrombocytopenia
|ClinicalTrials.gov Identifier: NCT01286038|
Recruitment Status : Active, not recruiting
First Posted : January 31, 2011
Last Update Posted : February 28, 2018
|Condition or disease||Intervention/treatment||Phase|
|Myelodysplastic Syndrome (MDS) Thrombocytopenia||Drug: Eltrombopag||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||37 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Sequential Two-Stage Dose Escalation Study to Evaluate the Safety and Efficacy of Eltrombopag in Myelodysplastic Syndrome (MDS) Patients With Thrombocytopenia Who Progressed or Are Resistant to Hypomethylating Agents|
|Actual Study Start Date :||March 21, 2011|
|Estimated Primary Completion Date :||December 2018|
|Estimated Study Completion Date :||December 2019|
Experimental: Eltrombopag Treatment
Phase I: Dose Escalation. Phase II: Treatment at Maximum Tolerated Dose (MTD)
In Stage 1 the best dose of eltrombopag will be found to take into Stage 2, which will treat 15 participants at this dose.
Other Name: Promacta®
- Maximum Tolerated Dose (MTD) [ Time Frame: 24 months ]Determine the maximum tolerated dose (MTD) of eltrombopag for the treatment of thrombocytopenia in participants with MDS who have either progressed or are resistant to Hypomethylating Agents (HMTA). The MTD is defined as the highest dose where less than 33% of participants experience a drug related predefined dose limited toxicity (DLT).
- Number of Participants with Complete or Major Platelet Response [ Time Frame: 24 months ]Proportion of participant achieving a complete or major platelet response utilizing International Working Group (IWG) 2006 response criteria and duration of platelets response.
- Number of Participants Achieving a Platelets Transfusion Response [ Time Frame: 24 months ]Proportion of participants achieving a platelets transfusion response.
- Number and Severity of Bleeding Events [ Time Frame: 24 months ]The incidence and severity of bleeding events, measured using the World Health Organization (WHO) Bleeding Scale, during the treatment and 4 week follow-up periods for eltrombopag.
- Number of Participants with Overall Survival(OS) [ Time Frame: 24 Months ]To evaluate the effect of eltrombopag on overall survival (OS).
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01286038
|United States, Florida|
|H. Lee Moffitt Cancer Center and Research Institute|
|Tampa, Florida, United States, 33612|
|Principal Investigator:||Rami Komrokji, M.D.||H. Lee Moffitt Cancer Center and Research Institute|