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)

• Conditions: ow or Intermediate-1 Risk Myelodysplastic Syndrome (MDS); MedDRA version: 9.1Level: LLTClassification code 10028533Term: Myelodysplastic syndrome

• Registration Number: EUCTR2007-007258-75-SE
• Lead Sponsor: Amgen Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2008-08-28
• Last Update Posted: 1 May 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 240

Inclusion Criteria

Disease related:
• Diagnosis of MDS using the WHO classification
• Per MDS IPSS, Low or intermediate-1 risk MDS
• The mean of the two platelet counts taken within 4 weeks prior to randomization must be:
- = 20 x 109/L, with no individual count >30 x 109/L with or without a history of bleeding, OR
- = 50 x 109/L, with no individual count > 60 x 109/L with a history of bleeding .
A standard of care platelet count taken prior to Informed Consent may be used as 1 of the 2 counts taken within 4 weeks prior to randomization.

Demographic:
• 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 start.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.

Laboratory:
• Adequate liver function, as evidenced by a serum bilirubin = 2 times the laboratory normal range and unconjugated bilirubin = 90% of total bilirubin (except for patients with a confirmed diagnosis of Gilbert’s Disease), ALT = 3 times the laboratory normal range, and AST = 3 times the laboratory normal range.
• A serum creatinine concentration = 2 mg/dl (=176.8 µmol/L)
• Bone marrow biopsy and aspirate with cytogenetics within 3 months of the first dose of investigational product.

Ethical:
• Before any study-specific procedure, the appropriate written informed consent must be obtained.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Disease Related:
• Have ever received any disease-modifying treatment for MDS
• Previously diagnosed with intermediate-2 or high risk MDS with IPSS
• Prior history of leukemia, aplastic anemia, or other non-MDS related bone marrow stem cell disorder
• Prior history of hematopoietic stem cell transplantation
• Persistent peripheral blood monocytosis (= 3 months with an absolute monocyte count >1,000/µL)
• 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) in the past year
• History of venous thrombosis that currently requires anti-coagulation therapy

Laboratory:
• Pregnant or breast feeding

Medications
• Received IL-11 within 4 weeks of screening
• 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 investigational product.
• Planned receipt of peg-G-CSF or GM-CSF after the first dose of investigational product.

General:
• Subject of reproductive potential who is not using adequate contraceptive precautions, in the judgement 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 E coli-derived product (eg, Infergen®, Neupogen®, Somatropin, and Actimmune®).
• Subject previously has entered this study.
• 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.
• Subject currently is enrolled in or has not yet completed 30 days since ending other investigational device or drug study.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified