Phase 2
N=40
Determination of Safe and Effective Dose of Romiplostim (AMG 531) in Subjects With Myelodysplastic Syndrome (MDS)Receiving Hypomethylating Agents
MDS · Myelodysplastic Syndromes · Thrombocytopenia
Bottom Line
View on ClinicalTrials.gov: NCT00321711 ↗Enrolled (actual)
40
Serious AEs
56.5%
Results posted
Apr 2011
Primary outcome: Primary: Occurrence of a Clinically Significant Thrombocytopenic Event — 11; 8; 10; 11 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Placebo (Drug); AMG 531 (Romiplostim) (Biological); Azacitidine (Drug); Decitabine (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Amgen
- Primary completion
- Oct 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Occurrence of a Clinically Significant Thrombocytopenic Event |
11; 8; 10; 11; 12 | — |
| SECONDARY Hypomethylating Agent Dose Reduction and Delay Due to Thrombocytopenia |
1; 1; 0; 0; 0 | — |
| SECONDARY Achieving an Overall Response (Complete or Partial Response, CR or PR) at the End of the Treatment Period |
2; 1; 1; 3; 5 | — |
| SECONDARY Platelet Transfusion |
9; 6; 5; 8; 7 | — |
Summary
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.
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT00321711). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.