A Phase II Study of 5-Azacitidine and Sargramostim as Maintenance Treatment After Definitive Therapy for Poor-risk AML or MDS
This study is not yet open for participant recruitment.
Verified May 2013 by Sidney Kimmel Comprehensive Cancer Center
Sponsor:
Sidney Kimmel Comprehensive Cancer Center
Collaborator:
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center
ClinicalTrials.gov Identifier:
NCT01700673
First received: October 2, 2012
Last updated: May 14, 2013
Last verified: May 2013
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Acute Myeloid Leukemia Myelodysplastic Syndrome |
Drug: 5-azacitidine and sargramostim |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| 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) |
Resource links provided by NLM:
Further study details as provided by Sidney Kimmel Comprehensive Cancer Center:
Primary Outcome Measures:
- To evaluate the 2 year relapse free survival of patients [ Time Frame: 2 year ] [ Designated as safety issue: No ]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.
Secondary Outcome Measures:
- 1. Describe and quantify the toxicity profile of the combination of 5AC and GM-CSF [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]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 ] [ Designated as safety issue: No ]2. Determine the impact on one-year RFS and overall survival for poor-risk myeloid disorders following maintenance therapy with 5AC and GM-CSF
| Estimated Enrollment: | 75 |
| Study Start Date: | July 2013 |
| Estimated Study Completion Date: | October 2017 |
| Estimated Primary Completion Date: | October 2015 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: ARM 1
Myeloablative
|
Drug: 5-azacitidine and sargramostim
5-azacitidine will be administered days 1-5 of a 28 day cycle. sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
Other Name: Vidaza, GM-CSF
|
|
Experimental: Arm 2
non-myeloablative
|
Drug: 5-azacitidine and sargramostim
5-azacitidine will be administered days 1-5 of a 28 day cycle. sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
Other Name: Vidaza, GM-CSF
|
|
Experimental: Arm 3
standard consolidation
|
Drug: 5-azacitidine and sargramostim
5-azacitidine will be administered days 1-5 of a 28 day cycle. sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
Other Name: Vidaza, GM-CSF
|
Eligibility| Ages Eligible for Study: | 6 Months and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Age > 6 months
- 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
- ECOG performance status 0-2
- 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
- Peripheral blood count recovery: Neutrophil count ≥ 1000 /µL, platelet count ≥ 50x 109 /µL without platelet transfusions, and adequate hematocrit independent of red cell transfusions .
- No evidence of extramedullary leukemia, such as CNS or soft tissue involvement
- 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
- Ability to give informed consent
- 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:
- Patients with untreated or uncontrolled infections
- Patients with untreated or uncontrolled grade 3 or 4 GVHD
- Pregnancy and lactation
- Concurrent use of any other investigational agents.
- Known HIV-positive patients.
- Known hypersensitivity to 5AC or GM-CSF
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01700673
Contacts
| Contact: Margaret Showel, MD | 410-614-3803 | |
| Contact: Jackie Greer, RN | 410-614-1329 | jgreer6@jhmi.edu |
Locations
| United States, Maryland | |
| The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | Not yet recruiting |
| Baltimore, Maryland, United States, 21287 | |
| Principal Investigator: Margaret Showel, MD | |
Sponsors and Collaborators
Sidney Kimmel Comprehensive Cancer Center
More Information
No publications provided
| Responsible Party: | Sidney Kimmel Comprehensive Cancer Center |
| ClinicalTrials.gov Identifier: | NCT01700673 History of Changes |
| Other Study ID Numbers: | J1240, P01CA015396, NA_00072223 |
| Study First Received: | October 2, 2012 |
| Last Updated: | May 14, 2013 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Sidney Kimmel Comprehensive Cancer Center:
|
maintenance treatment stem cell transplant cytarabine-based chemotherapy |
Additional relevant MeSH terms:
|
Leukemia Leukemia, Myeloid, Acute Leukemia, Myeloid Myelodysplastic Syndromes Preleukemia Neoplasms by Histologic Type Neoplasms Bone Marrow Diseases Hematologic Diseases Precancerous Conditions Azacitidine Cytarabine |
Antimetabolites, Antineoplastic Antimetabolites Molecular Mechanisms of Pharmacological Action Pharmacologic Actions Antineoplastic Agents Therapeutic Uses Enzyme Inhibitors Antiviral Agents Anti-Infective Agents Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs |
ClinicalTrials.gov processed this record on May 22, 2013