LeukemiaNCT00665002
Pilot Trial of a WT-1 Analog Peptide Vaccine in Patients With Myeloid Neoplasms
A study testing WT-1, Montanide and Sargramostim (GM-CSF) in people with Leukemia, run by H. Lee Moffitt Cancer Center and Research Institute at 1 site in United States.
Trial summary
The purpose of this study is to determine whether the WT-1 vaccine causes an immune response and is safe. The WT-1 vaccine is made up of protein pieces that the patient's immune system can recognize as abnormal. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia
- What is being tested?
- WT-1, Montanide and Sargramostim (GM-CSF)
- Type of treatment
- Vaccines, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2008to Feb 2015
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Cytologic or histologic diagnosis of acute myelogenous leukemia or myelodysplastic syndrome confirmed at Moffitt Cancer Center.
- Patients with acute myelogenous leukemia will have completed induction chemotherapy, achieved first complete remission (CR) 1 or 2, and will have completed any planned postremission therapy (at discretion of treating physician),with no plan for allogenic or autologous transplant.
- Patients with myelodysplastic syndrome who according to the International Prognostic Scoring System (IPSS) are category Int-2 or greater, with disease that relapsed, progressed, or not responded to at least 1 prior course of approved therapy for MDS (i.e. hypomethylating agent or lenalidomide).
- Patients with AML/MDS must have documented WT-1 + disease. For purposes of this study, this may be either the demonstration of WT-1 protein on a pretreatment bone marrow biopsy or detectable disease with RQ-PCR. For patients in whom a bone marrow aspirate is not available or possible (e.g. "dry tap"), a peripheral blood sample may be used for WT-1 screening. In such cases, 10 cc of peripheral blood will be collected in a heparinized tube.
- At least 4 weeks must have elapsed between the patient's last chemotherapy or radiation treatment and the first vaccination.
- Karnofsky performance status ≥ 70%
Show all 13Show fewer
- Hematologic parameters:
- Absolute neutrophil count ≥ 1000/mcL (except for MDS, for which the parameter is ≥ 500/mcL)
- Platelets > 50 K/mcL (except for MDS for which the parameter is > 25 K/mcL and not transfusion dependent)
- Biochemical parameters:
- Total bilirubin ≤ 2.0 mg/dl
- Aspartic transaminase (AST) and Alanine transaminase (ALT) ≤ 2.5 x upper limits of normal
- Creatinine ≤ 2.0 mg/dl
Who cannot join
- Pregnant or lactating women
- Patients with leptomeningeal disease
- Patients with active infection requiring systemic antibiotics, antiviral, or antifungal treatments
- Patients with serious unstable medical illness
- Patients taking systemic corticosteroids
- Patients with central nervous system (CNS) involvement with cancer/leukemia
Trial locations and contacts
1 site in 1 country.
United States1 site
H. Lee Moffitt Cancer Center and Research Institute
Tampa, Florida, 33612
About this study
The purpose of this study is to determine whether the WT-1 vaccine causes an immune response and is safe. The WT-1 vaccine is made up of protein pieces that the patient's immune system can recognize as abnormal.
Read the detailed description
Design:
This will be a pilot trial evaluating the safety and immunogenicity of the WT-1 peptide vaccine in patients with hematologic malignancies. Ten patients with acute myelogenous leukemia (AML) or advanced myelodysplastic syndrome (MDS), will be enrolled. Patients will be vaccinated with a preparation of WT-1-derived native and synthetic peptides plus immunologic adjuvant Montanide ISA 51 VG (Seppic Pharmaceuticals, Fairfield, NJ) and Sargramostim (GM-CSF). One dose level will be tested.
Patients will receive 6 injections of the WT-1 vaccine. Doses will be given every 2 weeks. Each vaccine is given at a different location under the skin in the arm or leg. Patients will be monitored for 30 minutes after vaccination.
WT-1 vaccine is given with another substance, Montanide, which clumps the WT-1 vaccine and improves the immune response. Patients will also receive an injection of Sargramostim (GM-CSF) 2 days before each vaccination and again on the day of the WT-1 injection at the same spot. Sargramostim (GM-CSF) stimulates the body's white blood cells to boost the immune response. Patients may be taught to do the Sargramostim (GM-CSF) injection themselves in which case patients will be given a log sheet to record the injection time and location. If not, they will need to come for an additional 24 study visits.
To monitor their health while receiving the vaccine, patients will need the following tests and procedures as a part of regular cancer care.
- History and physical examination every 2 weeks
- Complete blood count (CBC) and comprehensive panel every 2 weeks
- Bone marrow aspirate at week 14 for patients with acute myelogenous leukemia or myelodysplastic syndrome.
Patients will need these tests and procedures to see whether the vaccine is causing an immune response:
- A skin test will be performed before patients start the study and again in the 8th and 14th weeks in which a small amount of the vaccine will be placed under the skin. Two days later, the site will be checked to see whether a bump or swelling has formed. Another substance which typically does cause a mild reaction (mumps) will also be placed under the skin to measure whether they have a normal immune reaction.
- We will take about 7 tablespoons of blood to measure their immune response. About 1 tablespoon of blood will be taken to measure the levels of WT-1 in their blood. Blood samples will be taken prior to receiving the first vaccination and prior to receiving the vaccination at weeks 6 and 12.
If the vaccine causes the patient to have an immune response, and their cancer does not grow, they may continue to receive the WT-1 vaccinations monthly for 6 more months. If this occurs, the patient will have a computed tomography (CT) scan or bone marrow test and immunology blood tests 2 weeks after the 9th and 12th vaccinations.
- Conditions
- Leukemia
- Being tested
- WT-1, Montanide, Sargramostim (GM-CSF)
- Keywords
- Myeloid, Monocytic, Hematopoietic, Neoplasms, Acute Myelogenous Leukemia, AML, Myelodysplastic Syndrome, MDS
- Medical subject headings
- Leukemia, Neoplasms, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
WT-1Biological
Also called Analog Peptide Vaccine
Immune responses were to be evaluated at weeks 6 and 12 via delayed-type hypersensitivity, CD4 T cell proliferation, CD4 and CD8 T cell interferon release, as well as by bone marrow cytogenetics including polymerase chain reaction (PCR) to look for molecular evidence of disease. Patients who had an immunologic response and had not had disease progression could continue with up to 6 more vaccinations administered approximately every month. In that case, patients were to be reevaluated with bone marrows/immunologic studies after the 9th and 12th vaccination. In addition, patients would undergo evaluations for residual disease including immunohistochemistry and/or quantitative polymerase chain reaction (RQ-PCR) for WT-1 expression (on selected patients), and multiparameter flow cytometry (AML/ MDS).
MontanideDrug
The WT-1 vaccine is given with another substance, called Montanide, which clumps the WT-1 vaccine and thereby improves the immune response.
Sargramostim (GM-CSF)Drug
GM-CSF was administered at a dose 70 mcg (140ul) as a subcutaneous injection at the site of vaccination on day -2 and day 0.
| Study group | Role | What they receive |
|---|---|---|
| WT-1 Analog Peptide Vaccine | Experimental | Participants received 6 bi-weekly vaccinations over 10 weeks. WT-1 vaccine was given with Montanide. Participants also received an injection of Sargramostim (GM-CSF) two days before each vaccination and again on the day of the WT-1 injection at the same spot. Receives: Biological: WT-1; Drug: Montanide; Drug: Sargramostim (GM-CSF) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Adverse Events (AEs)
Toxicities were tabulated according to the NCI Common Toxicity (version 3.0) by grade and category. If more than one patient developed ≥ grade 3 non-hematologic toxicity or grade 4 hematologic toxicity, the study accrual was to be suspended immediately for a careful toxicity data evaluation. Depending upon the findings of such safety/toxicity data assessment and consultation with the supporting pharmaceutical company, the principal investigator of this trial would have the option of terminating this trial permanently, amending the study protocol, or resuming the patient accrual.
Measured: 12 weeks to 6 months
Secondary outcomes (1)
Participants Whose Samples Demonstrated Immunological Response After Vaccination
Immune Response: Immune reactivity was measured for all participants. Immune response was 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 and/or multiparameter intracellular staining by flow cytometry were performed as well.
ELISPOT Assay: CD4+ immune response, CD4+ and CD8+ response. The samples of participants' blood obtained at baseline and week 12 were tested for CD4 T cell proliferation, CD4 and CD8 T cell interferon release.
Tetramer Analysis of WT1-specific Immune responses: subtle WT1 T cell expansion, positive by ELISPOT and T cell expansion.
Delayed-type Hypersensitivity (DTH): measurable DTH response without overlap with ELISPOT or tetramer responders).
Overall: any form of immune response.
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Apr 2008 (estimated)
- Study startJun 2008
- Primary completionJul 2013
- Study completionFeb 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by H. Lee Moffitt Cancer Center and Research InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Innovive Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Jeffrey Lancet, M.D., Principal investigator, H. Lee Moffitt Cancer Center and Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00665002
- Sponsor's protocol ID
- MCC-15025
- Other IDs
- INNO-305 WT-1, 105946
- Status last verified
- Feb 2015
- First submitted
- 18 Apr 2008
- Last updated
- 27 Feb 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Shallis RM, Podoltsev NA. Maintenance therapy for acute myeloid leukemia: sustaining the pursuit for sustained remission. Curr Opin Hematol. 2021 Mar 1;28(2):110-121. doi: 10.1097/MOH.0000000000000637.
PubMed 33394722
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Trial of a WT-1 Analog Peptide Vaccine in Patients With Myeloid Neoplasms
- ClinicalTrials.gov ID
- NCT00665002
- Acronym / sponsor ID
- MCC-15025
- Conditions
- Leukemia
- Keywords
- Myeloid, Monocytic, Hematopoietic, Neoplasms, Acute Myelogenous Leukemia, AML, Myelodysplastic Syndrome, MDS
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research InstituteUniversity, hospital or other
- Collaborators
- Innovive Pharmaceuticals
- Study start
- Jun 2008
- Primary completion
- Jul 2013 (actual)
- Study completion
- Feb 2015 (actual)
- First posted
- 23 Apr 2008 (estimated)
- Results
- Posted 24 Jul 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine whether the WT-1 vaccine causes an immune response and is safe. The WT-1 vaccine is made up of protein pieces that the patient's immune system can recognize as abnormal.
Detailed description
Design:
This will be a pilot trial evaluating the safety and immunogenicity of the WT-1 peptide vaccine in patients with hematologic malignancies. Ten patients with acute myelogenous leukemia (AML) or advanced myelodysplastic syndrome (MDS), will be enrolled. Patients will be vaccinated with a preparation of WT-1-derived native and synthetic peptides plus immunologic adjuvant Montanide ISA 51 VG (Seppic Pharmaceuticals, Fairfield, NJ) and Sargramostim (GM-CSF). One dose level will be tested.
Patients will receive 6 injections of the WT-1 vaccine. Doses will be given every 2 weeks. Each vaccine is given at a different location under the skin in the arm or leg. Patients will be monitored for 30 minutes after vaccination.
WT-1 vaccine is given with another substance, Montanide, which clumps the WT-1 vaccine and improves the immune response. Patients will also receive an injection of Sargramostim (GM-CSF) 2 days before each vaccination and again on the day of the WT-1 injection at the same spot. Sargramostim (GM-CSF) stimulates the body's white blood cells to boost the immune response. Patients may be taught to do the Sargramostim (GM-CSF) injection themselves in which case patients will be given a log sheet to record the injection time and location. If not, they will need to come for an additional 24 study visits.
To monitor their health while receiving the vaccine, patients will need the following tests and procedures as a part of regular cancer care.
- History and physical examination every 2 weeks
- Complete blood count (CBC) and comprehensive panel every 2 weeks
- Bone marrow aspirate at week 14 for patients with acute myelogenous leukemia or myelodysplastic syndrome.
Patients will need these tests and procedures to see whether the vaccine is causing an immune response:
- A skin test will be performed before patients start the study and again in the 8th and 14th weeks in which a small amount of the vaccine will be placed under the skin. Two days later, the site will be checked to see whether a bump or swelling has formed. Another substance which typically does cause a mild reaction (mumps) will also be placed under the skin to measure whether they have a normal immune reaction.
- We will take about 7 tablespoons of blood to measure their immune response. About 1 tablespoon of blood will be taken to measure the levels of WT-1 in their blood. Blood samples will be taken prior to receiving the first vaccination and prior to receiving the vaccination at weeks 6 and 12.
If the vaccine causes the patient to have an immune response, and their cancer does not grow, they may continue to receive the WT-1 vaccinations monthly for 6 more months. If this occurs, the patient will have a computed tomography (CT) scan or bone marrow test and immunology blood tests 2 weeks after the 9th and 12th vaccinations.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| WT-1 Analog Peptide Vaccine | Experimental | Biological: WT-1, Drug: Montanide, Drug: Sargramostim (GM-CSF)Participants received 6 bi-weekly vaccinations over 10 weeks. WT-1 vaccine was given with Montanide. Participants also received an injection of Sargramostim (GM-CSF) two days before each vaccination and again on the day of the WT-1 injection at the same spot. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Cytologic or histologic diagnosis of acute myelogenous leukemia or myelodysplastic syndrome confirmed at Moffitt Cancer Center.
- Patients with acute myelogenous leukemia will have completed induction chemotherapy, achieved first complete remission (CR) 1 or 2, and will have completed any planned postremission therapy (at discretion of treating physician),with no plan for allogenic or autologous transplant.
- Patients with myelodysplastic syndrome who according to the International Prognostic Scoring System (IPSS) are category Int-2 or greater, with disease that relapsed, progressed, or not responded to at least 1 prior course of approved therapy for MDS (i.e. hypomethylating agent or lenalidomide).
- Patients with AML/MDS must have documented WT-1 + disease. For purposes of this study, this may be either the demonstration of WT-1 protein on a pretreatment bone marrow biopsy or detectable disease with RQ-PCR. For patients in whom a bone marrow aspirate is not available or possible (e.g. "dry tap"), a peripheral blood sample may be used for WT-1 screening. In such cases, 10 cc of peripheral blood will be collected in a heparinized tube.
- At least 4 weeks must have elapsed between the patient's last chemotherapy or radiation treatment and the first vaccination.
Key exclusion criteria (6 listed)
- Pregnant or lactating women
- Patients with leptomeningeal disease
- Patients with active infection requiring systemic antibiotics, antiviral, or antifungal treatments
- Patients with serious unstable medical illness
- Patients taking systemic corticosteroids
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| WT-1Also: Analog Peptide VaccineImmune responses were to be evaluated at weeks 6 and 12 via delayed-type hypersensitivity, CD4 T cell proliferation, CD4 and CD8 T cell interferon release, as well as by bone marrow cytogenetics including polymerase chain reaction (PCR) to look for molecular evidence of disease. Patients who had an immunologic response and had not had disease progression could continue with up to 6 more vaccinations administered approximately every month. In that case, patients were to be reevaluated with bone marrows/immunologic studies after the 9th and 12th vaccination. In addition, patients would undergo evaluations for residual disease including immunohistochemistry and/or quantitative polymerase chain reaction (RQ-PCR) for WT-1 expression (on selected patients), and multiparameter flow cytometry (AML/ MDS). | Biological | WT-1 Analog Peptide Vaccine |
| MontanideThe WT-1 vaccine is given with another substance, called Montanide, which clumps the WT-1 vaccine and thereby improves the immune response. | Drug | WT-1 Analog Peptide Vaccine |
| Sargramostim (GM-CSF)GM-CSF was administered at a dose 70 mcg (140ul) as a subcutaneous injection at the site of vaccination on day -2 and day 0. | Drug | WT-1 Analog Peptide Vaccine |
Primary outcome
Number of Participants With Adverse Events (AEs)
Time frame: 12 weeks to 6 months
Toxicities were tabulated according to the NCI Common Toxicity (version 3.0) by grade and category. If more than one patient developed ≥ grade 3 non-hematologic toxicity or grade 4 hematologic toxicity, the study accrual was to be suspended immediately for a careful toxicity data evaluation. Depending upon the findings of such safety/toxicity data assessment and consultation with the supporting pharmaceutical company, the principal investigator of this trial would have the option of terminating this trial permanently, amending the study protocol, or resuming the patient accrual.
1 secondary outcome
Participants Whose Samples Demonstrated Immunological Response After Vaccination
Time frame: 12 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33394722Shallis RM, Podoltsev NA. Maintenance therapy for acute myeloid leukemia: sustaining the pursuit for sustained remission. Curr Opin Hematol. 2021 Mar 1;28(2):110-121. doi: 10.1097/MOH.0000000000000637.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jeffrey Lancet, M.D. principal investigator
H. Lee Moffitt Cancer Center and Research Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing WT-1, Montanide and Sargramostim (GM-CSF) in people with Leukemia, run by H. Lee Moffitt Cancer Center and Research Institute at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Feb 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 24 Jul 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse Events (AEs) (time frame: 12 weeks to 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00665002, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Feb 2015.