Immunotherapy for Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Blast Phase Chronic Myelogenous Leukemia (BP CML), and Myelodysplastic Syndrome (MDS) Relapse After Allogeneic Transplantation

NCT00548847 | Completed Phase 2 Results DMC

• 2 other identifiers: IRB00002219WCI1228-06
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

The relapse of acute leukemia, MDS and blast phase CML after allogeneic transplantation affects approximately 1/3 to 1/2 of all transplant recipients and is the main cause of treatment failure. There is currently no effective standard treatment for this condition. This study will test the activity and feasibility of using a regimen to boost the immune system in order to treat AML, ALL, blast phase CML, and MDS relapse after allogeneic transplantation.

Conditions

Leukemia

Keywords

Leukemia

Outcome Measures

Primary Outcomes (1)

• Efficacy of GM-CSF and Pegylated Interferon-alpha 2b When Administered to Patients With AML, ALL, Blast Phase CML, and MDS Relapse After Allogeneic Transplantation, Defined as Progression-free Survival of > 33% at 3 Months

  Efficacy is defined as progression-free survival of \> 33% at 3 months. This is based on our retrospective data on 10% 3 month survival for relapsed patients. Progression is defined an an increase in blasts in blood or marrow by 50% compared to baseline with at least 20% of all cells being blasts at the time of assessment.

  3 months after cytokine treatment

Secondary Outcomes (1)

• Overall Survival at 6 Months (Evaluate Overall Responses; Perform Lab Experiments to Test Hypothesis That Exposure to Interferon-alpha and GM-CSF Up-regulates Co-stimulatory Molecule Expression on Relapsed Acute Leukemia Cells)

  6 months after cytokine treatment

Study Arms (1)

GM-CSF, Interferon-α-2b

EXPERIMENTAL

Biological: GM-CSF; Biological: Interferon-α-2b

Interventions

GM-CSF BIOLOGICAL

Dosing schedule: 250 mcg/m² subcutaneously Mon-Wed-Fri. Response assessed between 2 and 4 weeks. Duration on study is 3 months.

Also known as: Sargramostim, Leukine, Rhu GM-CSF, NSC #617589

GM-CSF, Interferon-α-2b

Interferon-α-2b BIOLOGICAL

Dosing schedule: 1.5 mcg/kg subcutaneously Monday weekly. Response assessed between 2 and 4 weeks. Duration on study is 3 months.

Also known as: Interferon-alpha-2b, Intron A®, Pegylated Interferon-α-2b, Peg-Intron, Peginterferon alfa-2b

GM-CSF, Interferon-α-2b

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age \> 1 year.
• Patients who have received an allogeneic transplant to treat AML, ALL, MDS, or CML and have relapse or progression of their AML, ALL, MDS, or CML are eligible to participate in the study. Relapse is defined as: reappearance of leukemic blasts as determined by morphologic analysis of the blood or marrow, reappearance of a phenotypic population of leukemia blasts by flow cytometric analysis of the blood or marrow, reappearance of a chromosome abnormality which is associated with the original leukemia as determined by chromosomal or fluorescence in situ hybridization (FISH) testing (ex: a translocation between chromosomes 9 and 22 for CML), reappearance of the molecular marker which is associated with the original leukemia as determined by polymerase chain reaction (PCR) (ex: breakpoint cluster region \[BCR\]-Abelson murine leukemia \[ABL\] for CML or ALL).
• \*Patients who received allogeneic transplantation to treat AML, ALL, MDS, or CML with detectable disease, and did not achieve remission of their leukemia after transplant are eligible.
• Eastern Cooperative Oncology Group (ECOG) performance status \< 2 for adults, and Lansky status 60% for children.
• Liver functions tests (aspartate transaminase \[AST\]/alanine aminotransferase \[ALT\]/bilirubin) \< 5x the upper limit of normal.
• Creatinine \< 3x the upper limit of normal.
• Lack of active grade 2-4 acute graft-versus-host disease (GVHD) 3 weeks after discontinuation of immunosuppression.
• Patients with limited stage and extensive stage chronic GVHD of mild severity (lichenoid changes), or requiring \< prednisone 10 mg/m² daily will be included.
• Recipients of grafts procured from related and unrelated donors with any level of human leukocyte antigen (HLA)-matching.

You may not qualify if:

• Pregnant patients are excluded due to unknown risk to the unborn fetus with cytokines.
• Allergy to components of interferon-alpha-2b or GM-CSF.
• Current uncontrolled infection.
• Current grade 2-4 acute GVHD or chronic extensive GVHD of moderate to severe nature, requiring treatment with more than 10 mg/m² of prednisone daily.
• Uncompensated heart failure, New York Heart Association (NYHA) class III-IV:
• Class I: patients with no limitation of activities; they suffer no symptoms from ordinary activities;
• Class II: patients with slight, mild limitation of activity; they are comfortable with rest or with mild exertion;
• Class III: patients with marked limitation of activity; they are comfortable only at rest;
• Class IV: patients who should be at complete rest, confined to bed or chair; any physical activity brings on discomfort and symptoms occur at rest.
• Breast feeding, due to unknown risk to the infant.
• Inability to give informed consent.
• Children under 1 year of age.

Sponsors & Collaborators

• Emory University: lead
• Bayer: collaborator

Study Sites (1)

Emory University Winship Cancer Institute

Atlanta, Georgia, 30322, United States

Location

MeSH Terms

Conditions

Leukemia

Interventions

Granulocyte-Macrophage Colony-Stimulating Factor; sargramostim; Introns; peginterferon alfa-2b

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

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; DNA, Intergenic; Genome Components; Genome; Genetic Structures; Genetic Phenomena; Gene Components; Genes

Results Point of Contact

• Title: Martha Arellano, MD
• Organization: Emory University

marella@emory.edu

404-778-1900

Study Officials

• Martha Arellano, MD

  Emory University Winship Cancer Institute

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: October 22, 2007
• First Posted: October 24, 2007
• Study Start: January 1, 2007
• Primary Completion: March 1, 2015
• Study Completion: March 1, 2015
• Last Updated: October 26, 2016
• Results First Posted: August 18, 2016

Record last verified: 2016-09

Locations

US (1)