Status:

COMPLETED

Determination of Safe Dose of Romiplostim (AMG 531) in Patients With Myelodysplastic Syndromes (MDS)

Lead Sponsor:

Amgen

Conditions:

Thrombocytopenia

MDS

Eligibility:

All Genders

18+ years

Phase:

PHASE2

Brief Summary

The purpose of this study is to evaluate the safety and tolerability of romiplostim in thrombocytopenic patients with low or Intermediate-1 risk MDS. In addition, the study will evaluate the platelet ...

Eligibility Criteria

Inclusion

  • Diagnosis of MDS using the World Health Organization classification
  • Low or Intermediate-1 risk MDS using the International Prognostic Scoring System (IPSS)
  • The mean of two platelet counts taken during the screening period must be ≤ 50 x 10\^9/L, with no individual count \> 55 x 10\^9/L (The mean platelet counts of 5 subjects enrolled at the maximum tolerated dose (MTD) must be ≤ 20 x 10\^9/L). Standard of care platelet assessments taken prior to Informed Consent may be used as 1 of the 2 counts taken within 3 weeks prior to study day 1.
  • Must be ≥ 18 years of age at the time of obtaining informed consent
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 at the time of screening
  • Adequate Liver Function, as evidenced by a serum bilirubin ≤ 1.5 times the laboratory normal range (except for patients with a confirmed diagnosis of Gilbert's Disease), alanine aminotransferase (ALT) ≤ 3 times the laboratory normal range, and aspartate aminotransferase (AST) ≤ 3 times the laboratory normal range
  • A serum creatinine concentration ≤ 2 mg/dL (≤ 176.6 µmol/L)
  • Before any study-specific procedure, the appropriate written informed consent must be obtained (see Section 12.1)

Exclusion

  • Currently receiving any treatment for MDS other than transfusions and erythropoietic growth factors. If granulocyte growth factors are currently being received, they cannot be used on or after study day 1
  • Clinically significant bleeding within 2 weeks prior to screening (eg, gastrointestinal (GI) bleeds, intracranial hemorrhage)
  • Prior malignancy (other than controlled prostate cancer, in situ cervical cancer or basal cell cancer of the skin) unless treated with curative intent and without evidence of disease for ≥ 3 years before screening
  • Prior history of bone marrow transplantation
  • Persistent peripheral blood monocytosis (≥ 3 months with an absolute monocyte count \> 1,000/µL)
  • Unstable angina, congestive heart failure (New York Heart Association \[NYHA\] \> class II), uncontrolled hypertension (diastolic \> 100 mmHg), uncontrolled cardiac arrhythmia, or recent (within 1 year) myocardial infarction
  • Received Anti-Thymocyte Globuline (ATG) within 6 months of screening
  • Received hypomethylating agents, immunomodulating agents, histone deacetylase inhibitors, cyclosporine or mycophenolate within 6 weeks of screening
  • Received interleukin (IL)-11 (oprelvekin) within 4 weeks before screening
  • Concurrent use of granulocyte growth factors (i.e. granulocyte-colony stimulating factor \[G-CSF; Neupogen, Granocyte\], pegfilgrastim \[Neulasta\], granulocyte macrophage-colony stimulating factor \[GM-CSF; Leukine, Prokine, Sargramostim\])
  • Have ever previously received recombinant thrombopoietin (rTPO), pegylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), eltrombopag, or romiplostim
  • Less than 4 weeks since receipt of any therapeutic drug or device that is not Food and Drug Administration (FDA) approved for any indication
  • Other investigational procedures are excluded
  • History of arterial thrombosis (eg, stroke or transient ischemic attack) in the past year
  • History of venous thrombosis that currently requires anti-coagulation therapy
  • Untreated B12 or folate deficiency
  • Subject is evidently pregnant (eg, positive human chorionic gonadotropin \[HCG\] test) or is breast feeding
  • Subject is not using adequate contraceptive precautions
  • Subject has known hypersensitivity to any recombinant E coli-derived product
  • Subject previously has enrolled in this study
  • Subject will not be available for follow-up assessment
  • Subject has any kind of disorder that compromises the ability of the subject to give written informed consent and/or to comply with study procedures

Key Trial Info

Start Date :

February 1 2006

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

May 1 2008

Estimated Enrollment :

72 Patients enrolled

Trial Details

Trial ID

NCT00303472

Start Date

February 1 2006

End Date

May 1 2008

Last Update

December 16 2013

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