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
Summary
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-CSF2. Determine the impact on one-year RFS and overall survival for poor-risk myeloid disorders following maintenance therapy with 5AC and GM-CSF
Eligibility
Minimum age: 6 Months.
Maximum age: N/A.
Gender(s): Both.
Criteria:
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
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