Ignite Creation Date:
2024-05-05 @ 5:19 PM
Last Modification Date:
2024-10-26 @ 9:30 AM
Study NCT ID:
NCT00433745
Status:
COMPLETED
Last Update Posted:
2014-07-08
First Post:
2007-02-09
Brief Title:
Wilms Tumor 1 WT1 Peptide Vaccine for High Risk Hematologic Malignancy
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Wilms Tumor 1 WT1 Peptide Vaccination for Patients With High Risk Hematological Malignancies
Status:
COMPLETED
Status Verified Date:
2014-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study will determine the safety and effectiveness of an experimental vaccine in controlling the abnormal growth of cells in patients with myelodysplastic syndrome MDS also known as myelodysplasia acute myeloid leukemia AML acute lymphoblastic leukemia ALL and chronic myeloid leukemia CML It will test whether the vaccine can increase the number of immune cells responding to the cancer and thereby slow progression of the illness improve blood counts reduce the need for transfusions of blood and platelets or even achieve a disease remission The vaccine contains part of a protein that is produced in large amounts by cells of patients with these cancers and an added substance called Montanide that helps the immune system respond to the vaccine Sargramostim another substances that boosts the immune response is also given
Patients 18 to 85 years of age with MDS AML ALL or CML may be eligible for this study Candidates are screened with a medical history physical examination blood tests chest x-ray and bone marrow biopsy Women of childbearing age also have a pregnancy test
Participants undergo the following
Chemotherapy entering the study
Leukapheresis to collect large amounts of white blood cells for infusion before vaccine administration
Participants may need placement of a central line plastic tube or catheter in the upper part of the chest to be used for giving chemotherapy blood or platelet transfusions antibiotics and white blood cells and for collecting blood samples
Weekly vaccine injections for nine weeks given in the upper arm upper leg or abdomen
Sargramostim injections following each vaccination
Standard of care treatment for MDS AML ALL or CML which may include blood or platelet transfusions growth factors and drugs to control underlying disease and potential side effects of the vaccine
Weekly safety monitoring including vital signs check brief health assessment blood tests and observation after the vaccination on the day of each vaccination
Follow-up evaluations with blood tests and chest x-ray 3 weeks after the last vaccine dose and with blood tests and bone marrow biopsy 7 weeks after the last vaccine dose
Patients 18 to 85 years of age with MDS AML ALL or CML may be eligible for this study Candidates are screened with a medical history physical examination blood tests chest x-ray and bone marrow biopsy Women of childbearing age also have a pregnancy test
Participants undergo the following
Chemotherapy entering the study
Leukapheresis to collect large amounts of white blood cells for infusion before vaccine administration
Participants may need placement of a central line plastic tube or catheter in the upper part of the chest to be used for giving chemotherapy blood or platelet transfusions antibiotics and white blood cells and for collecting blood samples
Weekly vaccine injections for nine weeks given in the upper arm upper leg or abdomen
Sargramostim injections following each vaccination
Standard of care treatment for MDS AML ALL or CML which may include blood or platelet transfusions growth factors and drugs to control underlying disease and potential side effects of the vaccine
Weekly safety monitoring including vital signs check brief health assessment blood tests and observation after the vaccination on the day of each vaccination
Follow-up evaluations with blood tests and chest x-ray 3 weeks after the last vaccine dose and with blood tests and bone marrow biopsy 7 weeks after the last vaccine dose
Detailed Description:
Leukemias and the related disorders myelodysplastic syndrome and myeloproliferative diseases represent a wide group of bone marrow stem cell malignancies Some patients can be cured with chemotherapy or by allogeneic stem cell transplantation However standard treatment approaches are not effective for patients who become refractory to chemotherapy those who relapse after transplantation and those with progressive disease The management of such patients remains unsatisfactory and requires new treatment approaches other than chemotherapy
The immunological graft-versus-leukemia GVL effect seen after allogeneic stem cell transplantation suggests that stimulating the patients own T cell responses to hematological malignancies might also retard disease progression and even achieve disease remissions Wilms Tumor 1 WT1 was identified as a target vaccine antigen because this antigen is over-expressed by cluster of differentiation 34 CD34 plus stem cells of most patients with myeloid and lymphoid malignancies but not by normal marrow cells A human leukocyte antigen HLA-A0201 restricted peptide derived from the Wilms Tumor WT protein is anticipated to induce T cell response against MDS and leukemic cells while sparing normal cells Of note about 40 of the population is HLA-A0201 positive
Therefore we propose this Phase II trial the second in a series of planned peptide vaccine research which will evaluate the safety associated with an immunotherapy approach of lymphodepletion lymphocyte infusion and WT1 vaccination in select patients diagnosed with MDS AML ALL and CML The WT1 vaccination will comprise of 9 doses of WT-1 peptide vaccines in Montanide adjuvant administered concomitantly with granulocyte macrophage- colony stimulating factor GM-CSF Sargramostim
The primary objectives will be to evaluate the efficacy and toxicity associated with the immunotherapy approach of lymphodepletion lymphocyte infusion and WT1 vaccination in selected patients with hematological malignancies
Secondary objectives will include evaluation of disease response by following the numbers of WT1 expressing cells in blood hematological measurements reduction in marrow blast cells changes in blood counts transfusion dependence and time to disease progression and survival
The primary endpoint will be the side effects of treatment toxicity and number of circulating WT1 specific T cells efficacy measured through week 16 of the study 7 weeks after the last dose of vaccine
The immunological graft-versus-leukemia GVL effect seen after allogeneic stem cell transplantation suggests that stimulating the patients own T cell responses to hematological malignancies might also retard disease progression and even achieve disease remissions Wilms Tumor 1 WT1 was identified as a target vaccine antigen because this antigen is over-expressed by cluster of differentiation 34 CD34 plus stem cells of most patients with myeloid and lymphoid malignancies but not by normal marrow cells A human leukocyte antigen HLA-A0201 restricted peptide derived from the Wilms Tumor WT protein is anticipated to induce T cell response against MDS and leukemic cells while sparing normal cells Of note about 40 of the population is HLA-A0201 positive
Therefore we propose this Phase II trial the second in a series of planned peptide vaccine research which will evaluate the safety associated with an immunotherapy approach of lymphodepletion lymphocyte infusion and WT1 vaccination in select patients diagnosed with MDS AML ALL and CML The WT1 vaccination will comprise of 9 doses of WT-1 peptide vaccines in Montanide adjuvant administered concomitantly with granulocyte macrophage- colony stimulating factor GM-CSF Sargramostim
The primary objectives will be to evaluate the efficacy and toxicity associated with the immunotherapy approach of lymphodepletion lymphocyte infusion and WT1 vaccination in selected patients with hematological malignancies
Secondary objectives will include evaluation of disease response by following the numbers of WT1 expressing cells in blood hematological measurements reduction in marrow blast cells changes in blood counts transfusion dependence and time to disease progression and survival
The primary endpoint will be the side effects of treatment toxicity and number of circulating WT1 specific T cells efficacy measured through week 16 of the study 7 weeks after the last dose of vaccine
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 07-H-0091 | OTHER | National Heart Lung and Blood Institute | None |