Skip to main content
OpenTrials
Completed

NCT Number: NCT00321711

Determination of Safe and Effective Dose of Romiplostim (AMG 531) in Subjects With Myelodysplastic Syndrome (MDS)Receiving Hypomethylating Agents

The purpose of this study is to evaluate the effect of Romiplostim (AMG 531) on the incidence of clinically significant thrombocytopenic events (grade 3 or 4 and/or receipt of platelet transfusions) in subjects with low or intermediate risk Myelodysplastic Syndrome (MDS) receiving hypomethylating agents. It is hypothesized that Romiplostim administration, at the appropriate dose and schedule, will result in reduction in the incidence of clinically significant thrombocytopenic events in low or intermediate risk MDS subjects receiving hypomethylating agents.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

MDS Blood Platelet Disorders Bone Marrow Diseases Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Myelodysplastic Syndromes Thrombocytopenia

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

- Diagnosis of MDS by bone marrow biopsy based on the World Health Organization (WHO) classification - Low, Intermediate-1 or Intermediate-2 risk category MDS using the IPSS (International Prognostic Scoring System) - Planned to receive either azacytidine 75 mg/m2 by subcutaneous administration each day for 7 days or decitabine 20 mg/m2 by intravenous administration each day for 5 days for at least 4 cycles Exclusion Criteria:

  • Prior exposure to >3 cycles hypomethylating agents
  • Prior history of leukemia or aplastic anemia
  • Prior history of bone marrow transplantation
  • Prior malignancy (other than in situ cervical cancer or basal cell cancer of the skin) unless treated with curative intent and without evidence of disease for ³ 3 years before randomization
  • Active or uncontrolled infections
  • Unstable angina, congestive heart failure [NYHA (New York Heart Association) > 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
  • Received IL-11 within 4 weeks of screening
  • Less than 4 weeks since receipt of any therapeutic drug or device that is not FDA approved for any indication
  • Have previously received any other thrombopoietic growth factor

Treatment and study plan

Placebo

Drug

Subjects in the control group will receive a placebo subcutaneous injection on a weekly basis during the 4 cycle treatment period.

AMG 531 (Romiplostim)

Biological

AMG 531 (Romiplostim) will be administered weekly by subcutaneous injection at a dose of 500 or 750 μg during Part A and 750 μg during Part B for the 4 cycle treatment period, depending on randomization.

Azacitidine

Drug

hypomethylating agent

decitabine

Drug

hypomethylating agent

Primary outcomes

  1. Occurrence of a Clinically Significant Thrombocytopenic Event

    Time frame: Treatment period (up to 20 weeks)

    Occurrence of a clinically significant thrombocytopenic event within the participant, defined as any platelet count obtained from day 15 of cycle 1 through the end of the interim follow-up visit that was less than 50 x 10^9/L or receipt of platelet transfusions at any time through the interim follow-up visit.

Secondary outcomes

  1. Hypomethylating Agent Dose Reduction and Delay Due to Thrombocytopenia

    Time frame: Treatment period (up to 20 weeks)

    Occurrence of hypomethylating agent dose reduction and delay due to thrombocytopenia

  2. Achieving an Overall Response (Complete or Partial Response, CR or PR) at the End of the Treatment Period

    Time frame: Treatment period (up to 20 weeks)

    CR = decrease in bone marrow blast (≤5%) and improvement in peripheral blood counts (Hgb ≥ 11 g/dL, platelets ≥ 100x10^9/L, neutrophils ≥ 1x10^9/L, peripheral blasts=0%). PR = improvement in peripheral blood counts plus a decrease in bone marrow blasts ≥50% but not ≤5, or decrease in International Prognostic Scoring System score.

  3. Platelet Transfusion

    Time frame: Study day 1 through the interim follow-up visit (up to 20 weeks)

    Occurrence of one or more platelet transfusions from study day 1 through the interim follow-up visit (16 weeks)

Sponsors and collaborators

Lead sponsor

Amgen

Industry

Registry information

Official study title

A Randomized, Double Blind, Placebo Controlled Study Evaluating the Efficacy and Safety of Romiplostim (AMG 531) Treatment of Subjects With Low or Intermediate Risk Myelodysplastic Syndrome (MDS) Receiving Hypomethylating Agents

Important dates

Study start
2006
Primary completion
2009
Study completion
2009
First posted
May 4, 2006
Registry last updated
Oct 17, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.