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Therapy of Myelodysplastic Syndrome (MDS) With Azacitidine Given in Combination With Etanercept: A Phase I/II Study.

Phase 1/Phase 2
18 Years
Not Enrolling
de Novo Myelodysplastic Syndromes, Previously Treated Myelodysplastic Syndromes, Secondary Myelodysplastic Syndromes

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Trial Information

Therapy of Myelodysplastic Syndrome (MDS) With Azacitidine Given in Combination With Etanercept: A Phase I/II Study.


I. Determine the frequency of hematologic responses in patients with MDS to 5-aza
(azacitidine) plus etanercept.

II. Determine the efficacy of 5-aza combined with etanercept in patients with low or
intermediate (int)-1 risk who fail to respond to anti-thymocyte globulin (ATG) plus
etanercept and for the purpose of this trial are considered as having progressive or "more
advanced" disease.

III. Correlate results of ex vivo/in vitro studies on phenotypic, cytogenetic and functional
disease characteristics with in vivo treatment responses, to identify parameters that are
associated with a high probability of response.


Patients receive etanercept subcutaneously (SC) twice weekly during weeks 1 and 2 and
azacitidine SC or intravenously (IV) over 10-40 minutes on days 1-7. Treatment repeats every
28 days for at least 3 courses. Treatment continues in the absence of disease progression or
unacceptable toxicity.

Inclusion Criteria:

- Int-2 or high risk MDS patients

- Patients with low-risk or int-1 risk MDS by International Prognostic Scoring System
(IPSS) criteria with:

- Single or multilineage cytopenia (absolute neutrophil count [ANC] < 1500/μL,
hemoglobin [Hgb],10g/dL, or platelet count < 100,000/μL); or

- Transfusion requirement of at least 2 units of packed red blood cells over an 8
week period

- Serum creatinine =< 1.5x ULN (upper limit of normal)

- Aspartate aminotransferase (AST)/alanine aminotransferase (ALT) =< 2x ULN

- Performance status =< 2 (Eastern Cooperative Oncology Group [ECOG] scale, 0-5)

Exclusion Criteria:

- Patients who have previously received hematopoietic stem cell transplants,
specifically for MDS

- Patients with a diagnosis of acute myeloid leukemia (AML) by World Health
Organization (WHO) criteria (i.e >= 20% blasts) at time of enrollment

- Women of child bearing potential who are currently pregnant, lactating or who are not
willing to use contraception during the entire duration of the study

- Men who are unwilling to use contraception while receiving 5-aza

- Patients with severe disease other than MDS which is expected to prevent compliance
with the present protocol

- Patients with severe infections (pneumonia, septicemia, etc) within the 2 weeks prior
to the anticipated start of protocol treatment

- Patients who are currently receiving or within the preceding 2 weeks have received
cytotoxic therapy, hemopoietic growth factors, immunomodulatory therapy, or other
experimental therapy for the treatment of MDS

- Current evidence of uncontrolled cardiac arrhythmia or congestive heart failure

- Platelet count =< 10,000/mcl

- Absolute neutrophil count =< 250/mcl

- Prior treatment with 5-aza

- Known or suspected hypersensitivity to azacitidine or mannitol

Type of Study:


Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Frequency of hematologic responses, as defined by International Working Group (IWG) criteria

Outcome Description:

A two-stage Simon design for phase II trials will be followed. The operating characteristics of this design yield a 90% chance of declaring a treatment ineffective if they true response rate is 0.30. If the true response rate is 0.50, there is an 80% chance of reaching the threshold of 13 responses out of 32 patients.

Outcome Time Frame:

Up to 2 years

Safety Issue:


Principal Investigator

Bart Scott

Investigator Role:

Principal Investigator

Investigator Affiliation:

Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium


United States: Federal Government

Study ID:




Start Date:

April 2005

Completion Date:

Related Keywords:

  • de Novo Myelodysplastic Syndromes
  • Previously Treated Myelodysplastic Syndromes
  • Secondary Myelodysplastic Syndromes
  • Myelodysplastic Syndromes
  • Preleukemia



Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium Seattle, Washington  98109