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

Acute Myeloid Leukemia Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01700673
• Other study ID #: J1240
• Secondary ID: P01CA015396NA_00
• Status: Completed
• Phase: Phase 2
• Start date: June 2013
• Est. completion date: June 2020

Study information

• Verified date: August 2021
• Source: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

To determine the impact of maintenance therapy in patients with MDS/AML in remission.

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: June 2020
• Est. primary completion date: June 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months and older

Eligibility

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

Related Conditions & MeSH terms

• Acute Myeloid Leukemia
• Leukemia
• Leukemia, Myeloid
• Leukemia, Myeloid, Acute
• Myelodysplastic Syndrome
• Myelodysplastic Syndromes
• Preleukemia

Intervention

Drug:
• Azacitidine
Azacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles.

Biological:
• Sargramostim
Sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.

Locations

Country	Name	City	State
United States	The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins	Baltimore	Maryland

Sponsors (2)

Lead Sponsor	Collaborator
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins	National Cancer Institute (NCI)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Two-year Relapse Free Survival of Patients	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 sargramostim (GM-CSF) during remission, following definitive therapy with either a stem cell transplant (SCT) or cytarabine-based consolidation chemotherapy.	2 year
Secondary	Hematologic Toxicity as Determined by Anemia	Percentage of patients with anemia, the most commonly reported hematologic toxicity, after receiving the combination of Azacitidine and sargramostim	1 year
Secondary	One-year RFS	We will report the number of participants with one year RFS	1 year
Secondary	Overall Survival	Percentage of participants with overall survival at 2 years.	2 years