Trial Outcomes & Findings for 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 (NCT NCT00548847)

Last Updated: 2016-10-26

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

15 participants

Primary outcome timeframe

3 months after cytokine treatment

Results posted on

2016-10-26

Participant Flow

All patients were enrolled at the Winship Cancer Institute of Emory University. The study closed to accrual in May 2011.

Participant milestones

Participant milestones
Measure	GM-CSF, Interferon-α-2b Granulocyte-macrophage colony-stimulating factor (GM-CSF), Interferon-α-2b: Dosing schedule: GM-CSF, 250 mcg/m2 Mon-Wed-Fri; Pegylated Interferon-α-2b 1.5 mcg/kg Monday weekly. Response assessed between 2 and 4 weeks. Duration on study is 3 months.
Overall Study STARTED	15
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	7

Reasons for withdrawal

Reasons for withdrawal
Measure	GM-CSF, Interferon-α-2b Granulocyte-macrophage colony-stimulating factor (GM-CSF), Interferon-α-2b: Dosing schedule: GM-CSF, 250 mcg/m2 Mon-Wed-Fri; Pegylated Interferon-α-2b 1.5 mcg/kg Monday weekly. Response assessed between 2 and 4 weeks. Duration on study is 3 months.
Overall Study Disease progression	7

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	GM-CSF, Interferon-α-2b n=15 Participants GM-CSF, Interferon-α-2b: Dosing schedule: GM-CSF, 250 mcg/m2 Mon-Wed-Fri; Pegylated Interferon-α-2b 1.5 mcg/kg Monday weekly. Response assessed between 2 and 4 weeks. Duration on study is 3 months.
Age, Categorical <=18 years	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	13 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	13 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	13 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	15 participants n=5 Participants

PRIMARY outcome

Timeframe: 3 months after cytokine treatment

Outcome measures

Outcome measures
Measure	GM-CSF, Interferon-α-2b n=15 Participants GM-CSF, Interferon-α-2b: Dosing schedule: GM-CSF, 250 mcg/m² Mon-Wed-Fri; Pegylated Interferon-α-2b 1.5 mcg/kg Monday weekly. Response assessed between 2 and 4 weeks. Duration on study is 3 months.
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 percentage of progression-free patients

SECONDARY outcome

Timeframe: 6 months after cytokine treatment

Outcome measures

Outcome measures
Measure	GM-CSF, Interferon-α-2b n=15 Participants GM-CSF, Interferon-α-2b: Dosing schedule: GM-CSF, 250 mcg/m² Mon-Wed-Fri; Pegylated Interferon-α-2b 1.5 mcg/kg Monday weekly. Response assessed between 2 and 4 weeks. Duration on study is 3 months.
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 participants

Adverse Events

GM-CSF, Interferon-α-2b

Serious events: 8 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	GM-CSF, Interferon-α-2b n=15 participants at risk GM-CSF, Interferon-α-2b: Dosing schedule: GM-CSF, 250 mcg/m2 Mon-Wed-Fri; Pegylated Interferon-α-2b 1.5 mcg/kg Monday weekly. Response assessed between 2 and 4 weeks. Duration on study is 3 months.
General disorders Death	33.3% 5/15
Respiratory, thoracic and mediastinal disorders Dyspnea	6.7% 1/15
Infections and infestations Infection	13.3% 2/15
Blood and lymphatic system disorders Hemorrhage	6.7% 1/15
Respiratory, thoracic and mediastinal disorders Adult Respiratory Distress Syndrome	6.7% 1/15

Other adverse events

Other adverse events
Measure	GM-CSF, Interferon-α-2b n=15 participants at risk GM-CSF, Interferon-α-2b: Dosing schedule: GM-CSF, 250 mcg/m2 Mon-Wed-Fri; Pegylated Interferon-α-2b 1.5 mcg/kg Monday weekly. Response assessed between 2 and 4 weeks. Duration on study is 3 months.
General disorders Fever	6.7% 1/15
Musculoskeletal and connective tissue disorders Myalgia	6.7% 1/15
Musculoskeletal and connective tissue disorders Arthralgia	6.7% 1/15
General disorders Malaise	6.7% 1/15
Musculoskeletal and connective tissue disorders Bone Pain	6.7% 1/15
General disorders Nausea	6.7% 1/15

Additional Information

Martha Arellano, MD

Emory University

Phone: 404-778-1900

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place