A Randomized, Double Blind, Placebo Controlled Study Evaluating the Efficacy and Safety of Romiplostim Treatment of Thrombocytopenia in Subjects With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)
- Diagnosis of MDS using the WHO classification for myeloid neoplasms as assessed
during the screening period.
- Per MDS IPSS, low or intermediate-1 risk MDS as assessed during the screening period.
- The mean of the two platelet counts taken within 4 weeks prior to randomization must
- ≤ 20 x 10^9/L, (with no individual count >30 x 10^9/L during the screening
period), with or without a history of bleeding associated with the diagnosis of
- ≤ 50 x 10^9/L, (with no individual count >60 x 10^9/L during the screening
period) with a history of bleeding associated with the diagnosis of MDS.
- Subjects must be ≥18 and ≤ 90 years of age at the time of informed consent. Subjects
between 85 and 90 years of age must have been diagnosed with MDS ≤ 5 years from study
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
- Adequate liver function, as evidenced by ALT ≤ 3 times the laboratory normal range,
AST ≤ 3 times the laboratory normal range and total bilirubin ≤ 2.0 times the
laboratory normal range.
(Adequate liver function for patients with a confirmed diagnosis of Gilbert's Disease
evidenced by ALT ≤ 3 times the laboratory normal range, and AST ≤ 3 times the laboratory
- A serum creatinine concentration ≤ 2 mg/dl (≤176.8 μmol/L).
- Bone marrow biopsy and aspirate with cytogenetics within 3 months of starting first
dose of investigational product.
- Written Informed Consent.
- Have ever received any disease-modifying treatment for MDS.
- Previously diagnosed with intermediate-2 or high risk MDS using the IPSS.
- Prior history of leukemia, aplastic anemia, or other non-MDS related bone marrow stem
- Prior history of hematopoietic stem cell transplantation.
- Persistent peripheral blood monocytosis (≥ 3 months with an absolute monocyte count
>1,000/μL) or known diagnosis of Chronic Myelomonocytic Leukemia per FAB criteria.
- Prior malignancy (other than in situ cervical cancer, non-melanoma skin cancer, or in
situ carcinoma) unless treated with curative intent and without evidence of disease
for ≥ 3 years before randomization.
- Active or uncontrolled infections.
- Unstable angina, congestive heart failure (NYHA > class II), uncontrolled
hypertension (diastolic >100 mmHg), uncontrolled cardiac arrhythmia, or recent
(within 1 year) myocardial infarction.
- History of arterial thrombosis (eg, stroke or transient ischemic attack) within the
- History of venous thrombosis that currently requires anti-coagulation therapy.
- Received IL-11 within 4 weeks of the first dose of investigational product.
- Have previously received any thrombopoietic growth factor.
- Receipt of G-CSF, peg-G-CSF, or GM-CSF within 4 weeks of the first dose of
- Planned receipt of peg-G-CSF or GM-CSF after the first dose of investigational
- Pregnant or breast feeding.
- Subjects of reproductive potential who are not using adequate contraceptive
precautions, in the judgment of the investigator. Amgen recommends double barrier
contraception is used for all applicable patients enrolled on this study. A double
barrier method is defined as two methods of contraception, for example 2 actual
barrier methods, or one actual barrier method and one hormonal method.
- Subject has known sensitivity to any recombinant E coli-derived product (eg,
Infergen®, Neupogen®, Somatropin, and Actimmune).
- Previously enrolled into the 20060198 study or another romiplostim study.
- Inability to comply with study procedures.
- Subject currently is enrolled in or has not yet completed at least 30 days since
ending other investigational device or drug study.