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An Open Label, Sequential, Dose Escalation Study to Evaluate the Safety and Efficacy of Romiplostim in Thrombocytopenic Subjects With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

Phase 2
18 Years
Not Enrolling
Thrombocytopenia, MDS, Myelodysplastic Syndromes, Refractory Cytopenias

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Trial Information

An Open Label, Sequential, Dose Escalation Study to Evaluate the Safety and Efficacy of Romiplostim in Thrombocytopenic Subjects With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

Inclusion Criteria:

- Diagnosis of MDS using the World Health Organization classification

- Low or Intermediate-1 risk MDS using the 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 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.

- Subjects 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

- 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),
ALT ≤ 3 times the laboratory normal range, and AST ≤ 3 times the laboratory normal

- 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 Criteria:

- 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, 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 >

- Unstable angina, congestive heart failure [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 IL-11 (oprelvekin) within 4 weeks before screening

- Concurrent use of granulocyte growth factors (i.e. G-CSF(Neupogen, Granocyte),
pegfilgrastim (Neulasta), GM-CSF (Leukine, Prokine, Sargramostim))

- Have ever previously received rTPO, PEG-rHuMGDF, eltrombopag, or romiplostim

- Less than 4 weeks since receipt of any therapeutic drug or device that is not FDA
approved for any indication

- Other investigational procedures are excluded

- History of arterial thrombosis (eg, stroke or transient ischemic attack) in the past

- History of venous thrombosis that currently requires anti-coagulation therapy

- Untreated B12 or folate deficiency

- Subject is evidently pregnant (eg, positive 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

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Adverse Event (Part A)

Outcome Description:

Occurrence of one or more adverse events in Part A

Outcome Time Frame:

Treatment period (4 weeks) plus treatment extension (1 year), plus 30 days if serious adverse event

Safety Issue:


Principal Investigator


Investigator Role:

Study Director

Investigator Affiliation:



France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Study ID:




Start Date:

January 2006

Completion Date:

May 2008

Related Keywords:

  • Thrombocytopenia
  • MDS
  • Myelodysplastic Syndromes
  • Refractory Cytopenias
  • MDS
  • Myelodysplastic Syndromes
  • Refractory Cytopenias
  • Thrombocytopenia
  • Myelodysplastic Syndromes
  • Preleukemia
  • Thrombocytopenia