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

NCT01700673 | Completed Phase 2 Results

• 3 other identifiers: J1240P01CA015396NA_00072223
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Acute Myeloid Leukemia; Myelodysplastic Syndrome

Keywords

maintenance treatment; stem cell transplant; cytarabine-based chemotherapy

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Hematologic Toxicity as Determined by Anemia

  1 year

• One-year RFS

  1 year

• Overall Survival

  2 years

Study Arms (3)

Myeloablative BMT

EXPERIMENTAL

Azacitidine and sargramostim after myeloablative stem cell transplant

Drug: Azacitidine; Biological: Sargramostim

Non-myeloablative BMT

EXPERIMENTAL

Azacitidine and sargramostim after non-myeloablative stem cell transplant

Drug: Azacitidine; Biological: Sargramostim

Standard consolidation

EXPERIMENTAL

Azacitidine and sargramostim after standard consolidation

Drug: Azacitidine; Biological: Sargramostim

Interventions

Azacitidine DRUG

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

Also known as: Vidaza, 5-azacytidine, Azacytidine

Myeloablative BMT; Non-myeloablative BMT; Standard consolidation

Sargramostim BIOLOGICAL

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

Also known as: GM-CSF

Myeloablative BMT; Non-myeloablative BMT; Standard consolidation

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Baltimore, Maryland, 21287, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Myelodysplastic Syndromes

Interventions

Azacitidine; sargramostim; Granulocyte-Macrophage Colony-Stimulating Factor

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Results Point of Contact

• Title: Jonathan Webster, MD
• Organization: Johns Hopkins University School of Medicine, Division of Hematologic Malignancies

jwebst17@jhmi.edu

4106149106

Study Officials

• Margaret Showel, MD

  JHU

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 2, 2012
• First Posted: October 4, 2012
• Study Start: June 1, 2013
• Primary Completion: June 1, 2020
• Study Completion: June 1, 2020
• Last Updated: October 18, 2022
• Results First Posted: September 2, 2021

Record last verified: 2021-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)