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A Phase II Study of 5-Azacitidine and Sargramostim as Maintenance Treatment After Definitive Therapy for Poor-risk AML or MDS

Information source: Sidney Kimmel Comprehensive Cancer Center
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Acute Myeloid Leukemia; Myelodysplastic Syndrome

Intervention: 5-azacitidine and sargramostim (Drug)

Phase: Phase 2

Status: Recruiting

Sponsored by: Sidney Kimmel Comprehensive Cancer Center

Overall contact:
Margaret Showel, MD, Phone: 410-614-7059


We propose a phase II study to determine the impact of maintenance therapy with 5-azacytidine and GM-CSF in patients with poor-risk AML or MDS, who are in remission after definitive treatment with either stem cell transplant or cytarabine-based consolidation chemotherapy. In order to precede relapse and to avoid lead time bias, treatment would need to commence within 185 days of definitive therapy. Furthermore, approximately 50% of relapses occur within the first year and up to 80% within two years after SCT, therefore we would limit the duration of maintenance therapy to one year, followed by two years of follow-up.

Clinical Details

Official title: A Phase II Study of 5-Azacitidine (5AC) in Combination With Sargramostim (GM-CSF) as Maintenance Treatment, After Definitive Therapy With Either Stem Cell Transplant (SCT) or Cytarabine-based Chemotherapy, in Patients With Poor-risk Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

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

Primary outcome: To evaluate the 2 year relapse free survival of patients

Secondary outcome:

1. Describe and quantify the toxicity profile of the combination of 5AC and GM-CSF

2. Determine the impact on one-year RFS and overall survival for poor-risk myeloid disorders following maintenance therapy with 5AC and GM-CSF


Minimum age: 6 Months. Maximum age: N/A. Gender(s): Both.


Inclusion Criteria: 1. Age > 6 months

2. Initial diagnosis of poor - risk AML or MDS (defined in section 3. 2), treated with

either stem cell transplant or cytarabine-based consolidation chemotherapy, within the past 60-185 days 3. ECOG performance status 0-2 4. No morphologic evidence of leukemia or active MDS as determined by JHH Hematopathologist independent review of a bone marrow aspirate and biopsy done following the completion of therapy and within 14 days prior to enrollment 5. Peripheral blood count recovery: Neutrophil count ≥ 1000 /µL, platelet count ≥ 50x 109 /µL without platelet transfusions, and adequate hematocrit independent of red cell transfusions . 6. No evidence of extramedullary leukemia, such as CNS or soft tissue involvement 7. Adequate end organ function as measured by the following: AST and ALT < 4 x normal, total serum bilirubin < 2 x upper limit normal (unless due to hemolysis, Gilbert's syndrome, or ineffective erythropoiesis), creatinine < 2 x upper limit of normal 8. Ability to give informed consent 9. In agreement to use an effective barrier method of birth control to avoid pregnancy during the study and for a minimum of 30 days after study treatment, for all male and female patients who are fertile Exclusion Criteria: 1. Patients with untreated or uncontrolled infections 2. Patients with untreated or uncontrolled grade 3 or 4 GVHD 3. Pregnancy and lactation 4. Concurrent use of any other investigational agents. 5. Known HIV-positive patients. 6. Known hypersensitivity to 5AC or GM-CSF

Locations and Contacts

Margaret Showel, MD, Phone: 410-614-7059

The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland 21287, United States; Recruiting
Margaret Showel, MD, Principal Investigator
Additional Information

Starting date: June 2013
Last updated: January 13, 2015

Page last updated: August 23, 2015

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