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

NCT ID: NCT01700673

Last Updated: 2022-10-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 25 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-06-30
• Study Completion Date: 2020-06-30

Brief Summary

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

Detailed 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.

Conditions

Acute Myeloid Leukemia; Myelodysplastic Syndrome

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Myeloablative BMT

Azacitidine and sargramostim after myeloablative stem cell transplant

Group Type: EXPERIMENTAL

Azacitidine

Intervention Type: DRUG

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

Sargramostim

Intervention Type: BIOLOGICAL

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

Non-myeloablative BMT

Azacitidine and sargramostim after non-myeloablative stem cell transplant

Group Type: EXPERIMENTAL

Azacitidine

Intervention Type: DRUG

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

Sargramostim

Intervention Type: BIOLOGICAL

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

Standard consolidation

Azacitidine and sargramostim after standard consolidation

Group Type: EXPERIMENTAL

Azacitidine

Intervention Type: DRUG

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

Sargramostim

Intervention Type: BIOLOGICAL

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

Interventions

Azacitidine

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

Intervention Type: DRUG

Sargramostim

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

Intervention Type: BIOLOGICAL

Other Intervention Names

Vidaza; 5-azacytidine; Azacytidine; GM-CSF

Eligibility 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

• Minimum Eligible Age: 6 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Cancer Institute (NCI)

NIH

Sponsor Role: collaborator

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Margaret Showel, MD

Role: STUDY_CHAIR

JHU

Locations

The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Baltimore, Maryland, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

P01CA015396

Identifier Type: NIH

Identifier Source: secondary_id

View Link

NA_00072223

Identifier Type: OTHER

Identifier Source: secondary_id

J1240

Identifier Type: -

Identifier Source: org_study_id