Trial Outcomes & Findings for Vaccine Therapy and GM-CSF in Treating Patients With Acute Myeloid Leukemia, Myelodysplastic Syndromes, Non-Small Cell Lung Cancer, or Mesothelioma (NCT NCT00398138)

Results Overview

Toxicities will be tabulated according to the NCI Common Toxicity (version 3.0).

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

22 participants

Primary outcome timeframe

2 years

Results posted on

2016-03-02

Participant Flow

Participant milestones

Participant milestones
Measure	Vaccine Six vaccinations of the WT-1 peptide (1.0 ml of emulsion) will be administered on weeks 0, 4, 6, 8, 10 \& 12. Vaccinations will be administered subcutaneously with sites rotated among extremities. Injection sites will be pre-stimulated with Sargramostim (GM-CSF) (70 mcg) injected subcutaneously on days 0 \& -2 of each vaccination. Patients may self administer the Sargramostim (GM-CSF) if they have been appropriately instructed on SQ injection administration. Patients will keep a logbook noting the time \& placement of the injection. Note: during each vaccination, the Sargramostim (GM-CSF) \& the vaccine emulsion will be administered to the same anatomical site. This site will be marked by the patient or treating healthcare professional by a permanent marker pen. For patients who have a clinical, molecular, or immunologic response \& have not had disease progression, they may receive up to 6 more vaccinations administered approximately every month. WT-1 analog peptide vaccine
Overall Study STARTED	12
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Vaccine Six vaccinations of the WT-1 peptide (1.0 ml of emulsion) will be administered on weeks 0, 4, 6, 8, 10 \& 12. Vaccinations will be administered subcutaneously with sites rotated among extremities. Injection sites will be pre-stimulated with Sargramostim (GM-CSF) (70 mcg) injected subcutaneously on days 0 \& -2 of each vaccination. Patients may self administer the Sargramostim (GM-CSF) if they have been appropriately instructed on SQ injection administration. Patients will keep a logbook noting the time \& placement of the injection. Note: during each vaccination, the Sargramostim (GM-CSF) \& the vaccine emulsion will be administered to the same anatomical site. This site will be marked by the patient or treating healthcare professional by a permanent marker pen. For patients who have a clinical, molecular, or immunologic response \& have not had disease progression, they may receive up to 6 more vaccinations administered approximately every month. WT-1 analog peptide vaccine
Overall Study Adverse Event	2

Baseline Characteristics

Vaccine Therapy and GM-CSF in Treating Patients With Acute Myeloid Leukemia, Myelodysplastic Syndromes, Non-Small Cell Lung Cancer, or Mesothelioma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Vaccine n=10 Participants Six vaccinations of the WT-1 peptide (1.0 ml of emulsion) will be administered on weeks 0, 4, 6, 8, 10 \& 12. Vaccinations will be administered subcutaneously with sites rotated among extremities. Injection sites will be pre-stimulated with Sargramostim (GM-CSF) (70 mcg) injected subcutaneously on days 0 \& -2 of each vaccination. Patients may self administer the Sargramostim (GM-CSF) if they have been appropriately instructed on SQ injection administration. Patients will keep a logbook noting the time \& placement of the injection. Note: during each vaccination, the Sargramostim (GM-CSF) \& the vaccine emulsion will be administered to the same anatomical site. This site will be marked by the patient or treating healthcare professional by a permanent marker pen. For patients who have a clinical, molecular, or immunologic response \& have not had disease progression, they may receive up to 6 more vaccinations administered approximately every month. WT-1 analog peptide vaccine
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants
Age, Categorical >=65 years	5 Participants n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Region of Enrollment United States	10 participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years

Toxicities will be tabulated according to the NCI Common Toxicity (version 3.0).

Outcome measures

Outcome measures
Measure	Vaccine n=10 Participants Six vaccinations of the WT-1 peptide (1.0 ml of emulsion) will be administered on weeks 0, 4, 6, 8, 10 \& 12. Vaccinations will be administered subcutaneously with sites rotated among extremities. Injection sites will be pre-stimulated with Sargramostim (GM-CSF) (70 mcg) injected subcutaneously on days 0 \& -2 of each vaccination. Patients may self administer the Sargramostim (GM-CSF) if they have been appropriately instructed on SQ injection administration. Patients will keep a logbook noting the time \& placement of the injection. Note: during each vaccination, the Sargramostim (GM-CSF) \& the vaccine emulsion will be administered to the same anatomical site. This site will be marked by the patient or treating healthcare professional by a permanent marker pen. For patients who have a clinical, molecular, or immunologic response \& have not had disease progression, they may receive up to 6 more vaccinations administered approximately every month. WT-1 analog peptide vaccine
Safety	10 participants

PRIMARY outcome

Timeframe: 2 years

Immune reactivity to the peptides will be measured in the same fashion for patients with hematologic or thoracic malignancies. Immune responses will be measured by T cell proliferative response and DTH against WT-1 peptides. In patients with adequate samples, T cell gamma interferon release as measured by ELISPOT will be performed as well.

Outcome measures

Outcome measures
Measure	Vaccine n=10 Participants Six vaccinations of the WT-1 peptide (1.0 ml of emulsion) will be administered on weeks 0, 4, 6, 8, 10 \& 12. Vaccinations will be administered subcutaneously with sites rotated among extremities. Injection sites will be pre-stimulated with Sargramostim (GM-CSF) (70 mcg) injected subcutaneously on days 0 \& -2 of each vaccination. Patients may self administer the Sargramostim (GM-CSF) if they have been appropriately instructed on SQ injection administration. Patients will keep a logbook noting the time \& placement of the injection. Note: during each vaccination, the Sargramostim (GM-CSF) \& the vaccine emulsion will be administered to the same anatomical site. This site will be marked by the patient or treating healthcare professional by a permanent marker pen. For patients who have a clinical, molecular, or immunologic response \& have not had disease progression, they may receive up to 6 more vaccinations administered approximately every month. WT-1 analog peptide vaccine
Immune Response	10 participants

Adverse Events

Vaccine

Serious events: 3 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Vaccine n=10 participants at risk Six vaccinations of the WT-1 peptide (1.0 ml of emulsion) will be administered on weeks 0, 4, 6, 8, 10 \& 12. Vaccinations will be administered subcutaneously with sites rotated among extremities. Injection sites will be pre-stimulated with Sargramostim (GM-CSF) (70 mcg) injected subcutaneously on days 0 \& -2 of each vaccination. Patients may self administer the Sargramostim (GM-CSF) if they have been appropriately instructed on SQ injection administration. Patients will keep a logbook noting the time \& placement of the injection. Note: during each vaccination, the Sargramostim (GM-CSF) \& the vaccine emulsion will be administered to the same anatomical site. This site will be marked by the patient or treating healthcare professional by a permanent marker pen. For patients who have a clinical, molecular, or immunologic response \& have not had disease progression, they may receive up to 6 more vaccinations administered approximately every month. WT-1 analog peptide vaccine
Respiratory, thoracic and mediastinal disorders Dyspnea	20.0% 2/10 • Number of events 2
Vascular disorders Edema: head and neck	10.0% 1/10 • Number of events 1
Nervous system disorders Syncope	10.0% 1/10 • Number of events 1

Other adverse events

Other adverse events
Measure	Vaccine n=10 participants at risk Six vaccinations of the WT-1 peptide (1.0 ml of emulsion) will be administered on weeks 0, 4, 6, 8, 10 \& 12. Vaccinations will be administered subcutaneously with sites rotated among extremities. Injection sites will be pre-stimulated with Sargramostim (GM-CSF) (70 mcg) injected subcutaneously on days 0 \& -2 of each vaccination. Patients may self administer the Sargramostim (GM-CSF) if they have been appropriately instructed on SQ injection administration. Patients will keep a logbook noting the time \& placement of the injection. Note: during each vaccination, the Sargramostim (GM-CSF) \& the vaccine emulsion will be administered to the same anatomical site. This site will be marked by the patient or treating healthcare professional by a permanent marker pen. For patients who have a clinical, molecular, or immunologic response \& have not had disease progression, they may receive up to 6 more vaccinations administered approximately every month. WT-1 analog peptide vaccine
Metabolism and nutrition disorders Albumin, low (hypoalbuminemia)	60.0% 6/10
General disorders Fatigue (asthenia, lethargy, malaise)	40.0% 4/10
Metabolism and nutrition disorders Glucose, high (hyperglycemia)	100.0% 10/10
Metabolism and nutrition disorders Hemoglobin	60.0% 6/10
Infections and infestations Leukocytes (total WBC)	40.0% 4/10
Blood and lymphatic system disorders Lymphopenia	10.0% 1/10
Infections and infestations Neutrophils/granulocytes (ANC/AGC)	20.0% 2/10
Blood and lymphatic system disorders Platelets	40.0% 4/10

Additional Information

Dr. Peter Maslak, Attending

Memorial Sloan Kettering Cancer Center

Phone: +1212-639-5518

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place