Vaccine Therapy and GM-CSF in Treating Patients With Myeloid Cancer - Full Text View

Sponsored by:	National Heart, Lung, and Blood Institute (NHLBI)
Information provided by:	National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:	NCT00499772

Purpose

RATIONALE: Vaccines made from peptides may help the body build an effective immune response to kill cancer cells. Colony-stimulating factors, such as GM-CSF, may increase the number of immune cells found in bone marrow or peripheral blood. Giving vaccine therapy together with GM-CSF may kill more cancer cells.

PURPOSE: This phase II trial is studying the side effects and how well giving vaccine therapy together with GM-CSF works in treating patients with myeloid cancer.

Condition	Intervention	Phase
Leukemia Myelodysplastic Syndromes	Drug: PR1 leukemia peptide vaccine Drug: WT1 126-134 peptide vaccine Drug: incomplete Freund's adjuvant Drug: sargramostim	Phase II

MedlinePlus related topics: Cancer Leukemia, Adult Acute Leukemia, Adult Chronic Wilms' Tumor

Drug Information available for: Sargramostim Granulocyte-macrophage colony-stimulating factor Freund's adjuvant Montanide ISA 51

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Active Control
Official Title:	Efficacy of WT1 and PR1 Peptide Vaccination for Patients With Low Risk Myeloid Malignancies

Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:

Immune response as assessed by tetramer and intracellular staining for interferon-gamma production at baseline, during vaccination, and after completion of study therapy [ Designated as safety issue: No ]

Secondary Outcome Measures:

Reduction in bone marrow blast cells [ Designated as safety issue: No ]
Changes in blood counts [ Designated as safety issue: No ]
Transfusion dependence [ Designated as safety issue: No ]
Time to disease progression [ Designated as safety issue: No ]
Survival [ Designated as safety issue: No ]

Estimated Enrollment:	24
Study Start Date:	June 2007
Estimated Primary Completion Date:	December 2009 (Final data collection date for primary outcome measure)

Detailed Description:

OBJECTIVES:

Primary

Evaluate the efficacy and toxicity associated with 6 doses of a combination of WT1:126-134 and PR1:169-177 peptide vaccines in incomplete Freund's adjuvant administered concurrently with sargramostim (GM-CSF) in selected patients with myeloid malignancies (i.e., MDS, AML, or CML).

Secondary

Clinically evaluate disease response using hematological measurements (i.e., reduction in marrow blast cells, changes in blood counts), transfusion dependence, time to disease progression, and survival.

OUTLINE: Patients receive WT1:126-134 and PR1:169-177 peptide vaccines emulsified in incomplete Freund's adjuvant subcutaneously (SC) administered concurrently with sargramostim (GM-CSF) SC once every 2 weeks. In order to monitor the local effects of each vaccine, injections are administered to separate sites that are rotated every 2 weeks. Treatment continues for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients with immunological response to one or both peptide vaccines will have the option of receiving an additional 6 boosters of the peptide vaccines at 3 monthly intervals.

After completion of study therapy, patients are followed at weeks 12 and 16.

Eligibility

Ages Eligible for Study:	18 Years to 85 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

DISEASE CHARACTERISTICS:

Diagnosis of 1 of the following:
- Myelodysplastic syndromes (MDS), including 1 of the following FAB histologic subtypes:
  - Refractory anemia
  - Refractory anemia with ringed sideroblasts (low-risk disease)
  - AND meets the following criteria:
    - No hypoplastic MDS
    - No relapsed MDS after hematopoietic stem cell transplantation
- Acute myeloid leukemia (AML) meeting the following criteria:
  - In complete remission within 5 years of prior treatment
  - Less than 5% marrow blasts
  - No relapsed AML
- Chronic myelogenous leukemia (CML) meeting the following criteria:
  - In chronic phase
  - No relapsed CML following hematopoietic stem cell transplantation
  - No CML in accelerated phase or blast crisis
Meets 1 of the following criteria:
- Not suitable for stem cell transplantation due to age over 60 years OR a fully-matched donor is not available
- Made an informed decision not to undergo the transplantation procedure
- At least 6 months-3 years since prior allogeneic stem cell transplantation AND demonstrates the following clinical features:
  - 100% donor engraftment
  - Less than 5% blasts in marrow
  - Normal marrow cellularity
No hypocellular bone marrow (i.e., < 20%)
HLA-A0201 positive at one allele
No history of Wegener's granulomatosis or vasculitis

PATIENT CHARACTERISTICS:

See Disease Characteristics
Predicted survival ≥ 28 days
Not pregnant or nursing
Negative pregnancy test
Fertile patients must use effective contraception
Hemoglobin ≥ 9 g/dL
Absolute neutrophil count ≥ 1,000/mm³
Platelet count ≥ 75,000/mm³
No serologic antibody against proteinase-3 (i.e., ANCA positive)
No prior allergic reaction to incomplete Freund's adjuvant
HIV-negative
No comorbidity of such severity that it would preclude the patient's ability to tolerate protocol therapy

PRIOR CONCURRENT THERAPY:

More than 14 days since prior systemic corticosteroids
No concurrent enrollment in another drug or vaccine clinical study
Concurrent nonlymphoablative chemotherapeutic agents (e.g., tyrosine kinase inhibitors or hydroxyurea) allowed for control of disease burden at the discretion of the principal investigator

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00499772

Locations

United States, Maryland
NIH - Warren Grant Magnuson Clinical Center	Recruiting
Bethesda, Maryland, United States, 20892-1182
Contact: Clinical Trials Office - NIH - Warren Grant Magnuson Clinical 800-411-1222

Sponsors and Collaborators

National Heart, Lung, and Blood Institute (NHLBI)

Investigators

Principal Investigator:

Quan Le, PhD

National Heart, Lung, and Blood Institute (NHLBI)

More Information

Clinical trial summary from the National Cancer Institute's PDQ® database This link exits the ClinicalTrials.gov site

Responsible Party:	NHLBI - Hematology Branch ( Quan Le )
Study ID Numbers:	CDR0000558028, NHLBI-07-H-0159
Study First Received:	July 10, 2007
Last Updated:	December 25, 2008
ClinicalTrials.gov Identifier:	NCT00499772
Health Authority:	Unspecified

Keywords provided by National Cancer Institute (NCI):

refractory anemia with ringed sideroblasts
refractory anemia
de novo myelodysplastic syndromes
secondary myelodysplastic syndromes
previously treated myelodysplastic syndromes
adult acute myeloid leukemia in remission
adult acute myeloid leukemia with 11q23 (MLL) abnormalities

adult acute myeloid leukemia with inv(16)(p13;q22)
adult acute myeloid leukemia with t(15;17)(q22;q12)
adult acute myeloid leukemia with t(16;16)(p13;q22)
adult acute myeloid leukemia with t(8;21)(q22;q22)
chronic phase chronic myelogenous leukemia
relapsing chronic myelogenous leukemia

Study placed in the following topic categories:

Precancerous Conditions
Chronic myelogenous leukemia
Refractory anemia
Leukemia, Myeloid, Chronic-Phase
Leukemia, Myeloid, Acute
Leukemia
Preleukemia
Anemia, Refractory
Neoplasm Metastasis
Wilms Tumor
Acute myeloid leukemia, adult
Congenital Abnormalities
Acute myelocytic leukemia

Myelodysplastic syndromes
Hematologic Diseases
Myelodysplastic Syndromes
Myelodysplasia
Wilms' tumor
Anemia
Myeloproliferative Disorders
Acute myelogenous leukemia
Leukemia, Myeloid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Freund's Adjuvant
Bone Marrow Diseases

Additional relevant MeSH terms:

Neoplasms
Pathologic Processes
Disease
Neoplasms by Histologic Type
Immunologic Factors

Syndrome
Physiological Effects of Drugs
Adjuvants, Immunologic
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 16, 2009