ClinicalTrials.gov
ClinicalTrials.gov Menu

Phase II Study of Azacitidine and Sargramostim as Maintenance Treatment for Poor-Risk AML or MDS

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01700673
Recruitment Status : Active, not recruiting
First Posted : October 4, 2012
Last Update Posted : August 17, 2018
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

October 2, 2012
October 4, 2012
August 17, 2018
June 2013
November 2019   (Final data collection date for primary outcome measure)
To evaluate the 2 year relapse free survival of patients [ Time Frame: 2 year ]
to evaluate the two-year relapse-free survival (RFS) of patients with poor-risk Acute Myeloid Leukemia (AML) or Myelodysplasia (MDS), who receive maintenance treatment with 5-Azacytidine(5AC) in combination with GM-CSF during remission, following definitive therapy with either a stem cell transplant (SCT) or cytarabine-based consolidation chemotherapy.
Same as current
Complete list of historical versions of study NCT01700673 on ClinicalTrials.gov Archive Site
  • 1. Describe and quantify the toxicity profile of the combination of 5AC and GM-CSF [ Time Frame: 1 year ]
    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 [ Time Frame: 1 year ]
    2. Determine the impact on one-year RFS and overall survival for poor-risk myeloid disorders following maintenance therapy with 5AC and GM-CSF
Same as current
Not Provided
Not Provided
 
Phase II Study of Azacitidine and Sargramostim as Maintenance Treatment for Poor-Risk AML or MDS
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)
To determine the impact of maintenance therapy in patients with MDS/AML in remission.

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.

Interventional
Phase 2
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
  • Acute Myeloid Leukemia
  • Myelodysplastic Syndrome
  • Drug: Azacitidine
    Azacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
    Other Names:
    • Vidaza
    • 5-azacytidine
    • Azacytidine
  • Biological: Sargramostim
    Sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
    Other Name: GM-CSF
  • Experimental: Myeloablative BMT
    Azacitidine and sargramostim after myeloablative stem cell transplant
    Interventions:
    • Drug: Azacitidine
    • Biological: Sargramostim
  • Experimental: Non-myeloablative BMT
    Azacitidine and sargramostim after non-myeloablative stem cell transplant
    Interventions:
    • Drug: Azacitidine
    • Biological: Sargramostim
  • Experimental: Standard consolidation
    Azacitidine and sargramostim after standard consolidation
    Interventions:
    • Drug: Azacitidine
    • Biological: Sargramostim
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
26
75
November 2019
November 2019   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
6 Months and older   (Child, Adult, Older Adult)
No
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT01700673
J1240
P01CA015396 ( U.S. NIH Grant/Contract )
NA_00072223 ( Other Identifier: JHMIRB )
No
Not Provided
Plan to Share IPD: No
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
National Cancer Institute (NCI)
Study Chair: Margaret Showel, MD JHU
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
August 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP