A Phase 2 interventional study of GM-CSF and Interferon-α-2b in Leukemia, sponsored by Emory University. Completed at 1 site in United States. Open to participants aged 1 Year and older. Per ClinicalTrials.gov, last updated 2016-10-26.
Sponsored by Emory University · Phase 2, Interventional, and Treatment
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.
This is a pilot phase II open label study testing the activity and feasibility of utilizing a regimen to boost the immune system in order to treat AML, ALL, blast phase CML, and MDS relapse after allogeneic transplantation. The regimen is a step-wise use of withdrawal of immunosuppression, cytoreduction if needed, administration of granulocyte-macrophage colony-stimulating factor (GM-CSF) and pegylated interferon (IFN) α-2b to patients who relapsed after an allogeneic transplant and will assess efficacy.
Relapse is the major problem following allogeneic hematopoietic progenitor cell transplants. There is currently no standard way to treat leukemia that relapsed after transplant, and patients have a poor prognosis.
A retrospective analysis of patients treated at Emory showed that administration of GM-CSF and interferon-alpha-2b was well-tolerated and affected long-term remissions in a small number of relapsed patients (after allogeneic transplant). Pre-clinical and clinical data from ours and other centers showed that relapsed leukemic blasts have down-regulation of co-stimulatory molecules and a tendency to evade the immune system. Cytokines can up-regulate co-stimulatory molecules on leukemic blasts and have been shown to increase the cytotoxicity of T-cells. This effect may be beneficial as a graft vs. leukemia effect for patients with relapse after allogeneic transplant.
5,441 studies on the registry are indexed under Leukemia; 636 are open to participants now.
This study's enrollment of 15 is below the median of 38 across 4,247 interventional studies indexed under Leukemia.
Browse Leukemia studies →Emory University is the lead sponsor of 1,386 studies on the registry; 236 are open to participants now.
Of its 229 completed or terminated interventional studies of FDA-regulated products, 174 (76%) have results posted.
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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.
Exclusion Criteria:
Uncompensated heart failure, New York Heart Association (NYHA) class III-IV:
Biological: GM-CSF · Biological: Interferon-α-2b
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
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
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.
Time frame: 3 months after cytokine treatment
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)
Time frame: 6 months after cytokine treatment
All patients were enrolled at the Winship Cancer Institute of Emory University. The study closed to accrual in May 2011.
| Milestone | GM-CSF, Interferon-α-2b |
|---|---|
| Started | 15 |
| Completed | 8 |
| Not completed | 7 |
| Withdrew: Disease progression | 7 |
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.
| percentage of progression-free patients | GM-CSF, Interferon-α-2b |
|---|---|
| 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 | 13 |
| participants | GM-CSF, Interferon-α-2b |
|---|---|
| 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) | 2 |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| GM-CSF, Interferon-α-2b | — | 8/15 (53.3%) | 6/15 (40%) |
| Event | GM-CSF, Interferon-α-2b |
|---|---|
| DeathGeneral disorders | 5/15 |
| InfectionInfections and infestations | 2/15 |
| DyspneaRespiratory, thoracic and mediastinal disorders | 1/15 |
| HemorrhageBlood and lymphatic system disorders | 1/15 |
| Adult Respiratory Distress SyndromeRespiratory, thoracic and mediastinal disorders | 1/15 |
| Event | GM-CSF, Interferon-α-2b |
|---|---|
| FeverGeneral disorders | 1/15 |
| MyalgiaMusculoskeletal and connective tissue disorders | 1/15 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 1/15 |
| MalaiseGeneral disorders | 1/15 |
| Bone PainMusculoskeletal and connective tissue disorders | 1/15 |
| NauseaGeneral disorders | 1/15 |
| Age, Categorical(Participants) | GM-CSF, Interferon-α-2b |
|---|---|
| <=18 years | 1 |
| Between 18 and 65 years | 13 |
| >=65 years | 1 |
| Sex: Female, Male(Participants) | GM-CSF, Interferon-α-2b |
|---|---|
| Female | 9 |
| Male | 6 |
| Ethnicity (NIH/OMB)(Participants) | GM-CSF, Interferon-α-2b |
|---|---|
| Hispanic or Latino | 1 |
| Not Hispanic or Latino | 13 |
| Unknown or Not Reported | 1 |
| Race (NIH/OMB)(Participants) | GM-CSF, Interferon-α-2b |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 1 |
| White | 13 |
| More than one race | 0 |
| Unknown or Not Reported | 1 |
| Region of Enrollment(participants) | GM-CSF, Interferon-α-2b |
|---|---|
| United States | 15 |
This study is completed, as verified in Sep 2016. You cannot join it, but the record below documents what was studied.
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