CompletedPhase 2

Eltrombopag With or Without Hypomethylating Agent After Hypomethylating Agent Failure For Patients With Myelodysplastic Syndrome (MDS)

United States29 participantsStarted 2013-10

Plain-language summary

The goal of this clinical research study is to learn if eltrombopag can help to control MDS. The safety of this drug will also be studied.

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Signed, informed consent must be obtained prior to any study specific procedures.
✓. Subjects with a histologically confirmed diagnosis of MDS by FAB criteria, including both MDS and RAEB-T (AML with 20-30% blasts and multilineage dysplasia) by World Health Organization (WHO) classification are eligible.
✓. Patients must have completed at least 4 cycles of hypomethylating agent therapy (e.g azacitidine or decitabine) with failure to achieve at least a partial response, or with the presence of ongoing cytopenias per International Working Group (IWG) (platelet count \< 100x10\^9/L, hemoglobin \<11g/L or Absolute Neutrophil Count (ANC) \<1x10\^9/L). Patients with progressive disease on HMA-therapy prior to this time point are also eligible at the time of documented progression. Therapy with decitabine analogs (i.e. SGI-110) will be considered as decitabine for the purposes of this study.
✓. Platelet count \<100x10\^9/L
✓. Low, intermediate-1, intermediate-2 or High-risk category by International Prognostic Scoring System (IPSS)
✓. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2
✓. Adequate liver function, as evidenced by a serum bilirubin \</=2x the ULN (except for patients with a confirmed diagnosis of Gilbert's Disease) and an Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST) \</=3x the laboratory Upper Limit of Normal (ULN).
✓. Serum creatinine \</=2x upper limit of normal

✕. Subjects with any prior exposure to a thrombopoietin-receptor agonist
✕. Any prior or co-existing medical condition that in the investigator's judgment will substantially increase the risk associated with the subject's participation in the study
✕. Psychiatric disorders or altered mental status precluding understanding of the informed consent process and/or completion of the necessary study procedures
✕. Active uncontrolled serious infection or sepsis at study enrollment
✕. Clinically significant gastrointestinal disorders that may interfere with absorption of drug.
✕. History of arterial thrombosis (i.e. stroke) in the past year
✕. History of venous thrombosis currently requiring anti-coagulation therapy
✕. Unstable angina, congestive heart failure (New York Heart Association (NYHA) \> Class II), uncontrolled hypertension (diastolic blood pressure \> 100mmHg), or recent (within 1 year) myocardial infarction

Overall Response Rate (ORR)

Timeframe: After second 28 day cycle

Overall Survival (OS)

Timeframe: Through study completion. Up to 3 years, 3 months.

NCT IDNCT01893372

SponsorM.D. Anderson Cancer Center

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2019-01-06

Results submitted2020-01-23

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Signed, informed consent must be obtained prior to any study specific procedures.
✓. Subjects with a histologically confirmed diagnosis of MDS by FAB criteria, including both MDS and RAEB-T (AML with 20-30% blasts and multilineage dysplasia) by World Health Organization (WHO) classification are eligible.
✓. Patients must have completed at least 4 cycles of hypomethylating agent therapy (e.g azacitidine or decitabine) with failure to achieve at least a partial response, or with the presence of ongoing cytopenias per International Working Group (IWG) (platelet count \< 100x10\^9/L, hemoglobin \<11g/L or Absolute Neutrophil Count (ANC) \<1x10\^9/L). Patients with progressive disease on HMA-therapy prior to this time point are also eligible at the time of documented progression. Therapy with decitabine analogs (i.e. SGI-110) will be considered as decitabine for the purposes of this study.
✓. Platelet count \<100x10\^9/L
✓. Low, intermediate-1, intermediate-2 or High-risk category by International Prognostic Scoring System (IPSS)
✓. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2
✓. Adequate liver function, as evidenced by a serum bilirubin \</=2x the ULN (except for patients with a confirmed diagnosis of Gilbert's Disease) and an Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST) \</=3x the laboratory Upper Limit of Normal (ULN).
✓. Serum creatinine \</=2x upper limit of normal

✕. Subjects with any prior exposure to a thrombopoietin-receptor agonist
✕. Any prior or co-existing medical condition that in the investigator's judgment will substantially increase the risk associated with the subject's participation in the study
✕. Psychiatric disorders or altered mental status precluding understanding of the informed consent process and/or completion of the necessary study procedures
✕. Active uncontrolled serious infection or sepsis at study enrollment
✕. Clinically significant gastrointestinal disorders that may interfere with absorption of drug.
✕. History of arterial thrombosis (i.e. stroke) in the past year
✕. History of venous thrombosis currently requiring anti-coagulation therapy
✕. Unstable angina, congestive heart failure (New York Heart Association (NYHA) \> Class II), uncontrolled hypertension (diastolic blood pressure \> 100mmHg), or recent (within 1 year) myocardial infarction