Erlotinib Study for Myelodysplastic Syndrome (MDS)
The purpose of this research study is to find out what effects, good and/or bad, erlotinib has on the patient and their myelodysplastic syndrome. Erlotinib has been approved by the Food and Drug Administration (FDA) to treat non-small cell lung cancer; however, erlotinib use in this study is considered investigational as the FDA has not approved it for the treatment of myelodysplastic syndrome.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase II Study Evaluating the Role of Erlotinib an Epidermal Growth Factor Receptor (EGFR) Inhibitor in the Treatment of Myelodysplastic Syndrome|
- Combined Overall Response Rate (ORR) [ Time Frame: Up to 21 Months ] [ Designated as safety issue: No ]Best Response Categories: Marrow complete response (CR), Bone marrow: ≤ 5% myeloblasts and decrease by ≥ 50% over pretreatment; Hematological improvement (HI), Hgb increase by ≥ 1.5 g/dL, Absolute increase of ≥ 30 x 10^9/L for patients starting with > 20 x 10^9/L, At least 100% increase and an absolute increase of > 0.5 x 10^9/L, as defined by the International Working Group (IWG) 2006 criteria.
- Median Overall Survival (OS) [ Time Frame: Up to 21 Months ] [ Designated as safety issue: No ]OS: The time from randomization until death from any cause. Kaplan-Meier estimates were used for secondary endpoint analysis.
- Median Progression Free Survival (PFS) [ Time Frame: Up to 21 Months ] [ Designated as safety issue: No ]PFS: The time elapsed between treatment initiation and tumor progression or death from any cause. Kaplan-Meier estimates were used for secondary endpoint analysis. Disease Progression is defined using International Working Group (IWG) Response Criteria for MDS, as at least 50% decrement from maximum remission/response levels in granulocytes or platelets; reduction in hemoglobin (Hgb) concentration by ≥ 2 g/dL; transfusion dependence.
- Leukemia Free Survival (LFS) [ Time Frame: Up to 21 Months ] [ Designated as safety issue: No ]LFS: Survival without evidence of relapse at any time post-transplant. Kaplan-Meier estimates were used for secondary endpoint analysis.
|Study Start Date:||September 2009|
|Study Completion Date:||June 2012|
|Primary Completion Date:||June 2012 (Final data collection date for primary outcome measure)|
Experimental: Erlotinib Treatment
Erlotinib was given as an oral 150 mg daily dose for 16 weeks. The dose was adjusted for diarrhea, rash and pulmonary toxicity.
Participants took erlotinib at least 1 hour before, or 2 hours after they ate a meal or snack. Participants were advised to take erlotinib at around the same time every day.
Screening Period: Informed consent, physical examination, medical history report, blood tests, pregnancy test (if applicable), list of current medications, description of symptoms, chest x-ray, ECG, bone marrow aspirate/biopsy within 4 weeks of study start.
Weeks 2,6,10 and 14: Blood tests.
Weeks 4 and 12: Blood tests, physical exam, patients will answer question about how they are feeling and if there are any changes to medication they have taken.
Weeks 8 and 16: Blood tests, physical exam, patients will answer question about how they are feeling and if there are any changes to medication they have taken, bone marrow aspirate/biopsy (if physician has determined the patient has had a clinical response or partial response to treatment.
After week 16 (if responding to treatment): Have a bone marrow aspirate/biopsy (will be repeated at time of relapse, i.e., more than 50% increase in the percentage of myeloblasts [leukemia cells] or drop in blood counts after they improved or requiring regular blood transfusions after not requiring them for at least 8 weeks, or after 1 year in study).
After the patient has stopped taking erlotinib: Periodic follow-up on patients' status.
|United States, Florida|
|H. Lee Moffitt Cancer Center & Research Institute|
|Tampa, Florida, United States, 33612|
|Principal Investigator:||Rami Komrokji, M.D.||H. Lee Moffitt Cancer Center and Research Institute|