The Role of Erlotinib an Epidermal Growth Factor Receptor (EGFR) Inhibitor in the Treatment of Myelodysplastic Syndrome
The purpose of this research study is to find out what effects, good and/or bad, Erlotinib has on Myelodysplastic syndrome. Myelodysplastic syndrome is a group of blood diseases where the bone marrow (spongy space in long bones which is the factory for blood cell production) does not make enough blood cells and therefore there is a lack of healthy blood cells in the body. This can result in anemia, risk for infection and/or bleeding..
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase II of Erlotinib an Epidermal Growth Factor Receptor Inhibitor in the Treatment of Myelodysplastic Syndrome|
- First 20 patients will be evaluated for overall response rate (CR, PR or HI). [ Time Frame: Estimated to be about 1 year. ] [ Designated as safety issue: No ]
- If the first analysis of 20 patients warrant further enrollment, an additional 15 eligible patients will be registered. If 8 or more of the 35 patients achieve CR, PR or HI, then Phase III study will be warranted. [ Time Frame: Estimated to be about 1 year. ] [ Designated as safety issue: No ]
|Study Start Date:||November 2007|
|Estimated Study Completion Date:||November 2012|
|Estimated Primary Completion Date:||November 2009 (Final data collection date for primary outcome measure)|
Experimental: Single Arm
All patients will receive 150 mg of Erlotinib
150 mg, PO, QD beginning day 1 week 1. Patients will receive treatment for 16 weeks as long as there is no evidence of disease progression. In no response is noted after 16 weeks of treatment, patients will be taken off the study. Patients achieving response (HI, CR, or PR) will continue on treatment until evidence of disease progression or relapse.
MDS is a neoplastic clonal stem disorder characterized by bone marrow failure with cytopenia, dyslastic morphological features and tendency to progress to acute myeloid leukemia. It is estimated that MDS is the most common hematological malignancy in the USA. Several treatment options are available for MDS ranging from supportive care, growth factor use, chemotherapy, stem cell transplantation, and newer novel agents such as thalidomide, lenalidomide, and hypomethylating agents. Each of the different available treatments for MDS work in certain subset and relatively small percentage of patients, keeping the door open for novel therapeutic strategies to be explored. The NIH has published requests for applications on myeloproliferative and myelopdysplastic syndrome emphasizing the need for more research in this area.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00570375
|United States, Ohio|
|University of Cincinnati|
|Cincinnati, Ohio, United States, 45267|
|Principal Investigator:||Carl W Siegrist, M.D.||University of Cincinnati|