Protocol Number: 07-H-0159

Title:

Efficacy of WT1 and PRI Peptide Vaccination for Patients with Low Risk Myeloid Malignancies

Number:

07-H-0159

Summary:

This study will test the safety and effectiveness of two vaccines on slowing disease progression, improving blood counts, reducing the need for transfusions of blood and platelets, or achieving remission in patients with myelodysplastic syndrome (MDS, also known as myelodysplasia), acute myeloid leukemia (AML) or chronic myeloid leukemia (CML). The vaccines consist of peptides (parts of proteins) found in MDS, AML and CML stem cells, combined with a substance called MontanideTM. They are administered with GM-CSF. The Montanide and the GM-CSF help the immune system respond to the vaccines.

People 18 years of age or older with MDS, AML or CML may be eligible for this study.

Participants receive six injections of the vaccines, one dose every other week for a total of 10 weeks. The injections are given in the upper arm, upper leg, or abdomen. A separate injection of GM-CSF is given in the same area as the vaccine injections. Subjects are observed for 2 hours after the first vaccination and at least 30 minutes after each subsequent vaccination for allergic reactions. In addition to the vaccination, subjects undergo the following:

-History and physical exam, chest x-ray, blood tests and bone marrow aspirate and biopsy before starting the vaccinations.

-Safety monitoring during vaccine administration (every other week for 10 weeks) with blood tests and check of vital signs.

-Follow-up safety monitoring (weeks 12 and 16) with blood tests every visit, chest x-ray at week 12 and bone marrow biopsy visit 16.

Sponsoring Institute:

National Heart, Lung and Blood Institute (NHLBI)

Recruitment Detail

Type: Participants currently recruited/enrolled

Gender: Male & Female

Referral Letter Required: No

Population Exclusion(s): Children

Eligibility Criteria:

INCLUSION CRITERIA:

Diagnosed with MDS (B subtypes RA, RARS -Low Risk) (MDS with 5q- must have failed lenalidomide or been ineligible to receive it)

OR

Diagnosed with AML and in complete remission within 5 years of treatment with less than 5% marrow blasts

OR

Diagnosed with CML in chronic phase

Unsuitable for stem cell transplantation (age over sixty or unavailability of a fully-matched donor)

or

made an informed decision not to undergo the transplant procedure

or

are between 6 months 3 years following allogeneic SCT and fulfill the following criteria:

100% donor engraftment,

Less than 5% blasts in marrow

normal marrow cellularity

HLA-A020 1 positive at one allele

Ages 18-85 years old

Off all lymphoablative chemotherapeutic agents

EXCLUSION CRITERIA:

- Hypoplastic MDS

- Relapsed AML

- CML in accelerated phase or blast crisis

- Hypocellular bone marrow (less than 20%)

- History of Wegener's granulomatosis

- Serologic antibody against proteinase-3 (ANCA positive)

- Previous allergic reaction to Montanide Adjuvant

- Positive test for HIV

- Treatment with systemic corticosteroids or immunosuppresants within 14 days prior to study entry

- Comorbidity of such severity that it would preclude the patient's ability to tolerate protocol therapy

- Predicted survival less than 28 days

- Pregnant or breast feeding (All female patients must have a urine pregnancy test within 1 week prior to vaccine administration)

- Unwilling to practice abstinence or effective contraception (men and women) during the study period.

- Enrolled in another drug or vaccine clinical trial during the study period

- Inability to comprehend the investigational nature of the study and provide informed consent

Special Instructions:

Currently Not Provided

Keywords:

Myelodyplastic Syndrome (MDS)

Acute Myeloid Leukemia (AML)

Chronic Myeloid Leukemia (CML)

Wilm's Tumor-1

Proteinase-3

Recruitment Keyword(s):

Myelodysplastic Syndrome

MDS

Acute Myeloid Leukemia

AML

Chronic Myeloid Leukemia

CML

Condition(s):

Myelodysplastic Syndrome (MDS)

Acute Myeloid Leukemia (AML)

Chronic Myeloid Leukemia (CML)

Investigational Drug(s):

WT1 and PR1 Peptide Vaccines

Investigational Device(s):

None

Intervention(s):

Biological/Vaccine: WT1:126-134 Peptide

Biological/Vaccine: PR1:169-177 Peptide

Drug: GM-CSF (Sargramostim)

Drug: WT1 and PR1 Peptide Vaccines

Drug: GM-CSF (Sargramostim)

Biological/Vaccine: WT1 and PR1 Peptide Vaccines

Supporting Site:

National Heart, Lung and Blood Institute

Contact(s):

Patient Recruitment and Public Liaison Office
Building 61
10 Cloister Court
Bethesda, Maryland 20892-4754
Toll Free: 1-800-411-1222
TTY: 301-594-9774 (local),1-866-411-1010 (toll free)
Fax: 301-480-9793

Electronic Mail:prpl@mail.cc.nih.gov

Citation(s):

Hofmann WK, LŸbbert M, Hoelzer D, Phillip Koeffler H. Myelodysplastic syndromes. Hematol J. 2004;5(1):1-8..

Estey EH.Prognosis and therapy of secondary myelodysplastic syndromes. Haematologica. 1998 Jun;83(6):543-9

Hellstršm-Lindberg E, Ahlgren T, Beguin Y, Carlsson M, Carneskog J, Dahl IM, Dybedal I, Grimfors G, Kanter-Lewensohn L, Linder O, Luthman M, Lšfvenberg E, Nilsson-Ehle H, Samuelsson J, Tangen JM, Winqvist I, Oberg G, Osterborg A, Ost A. Treatment of anemia in myelodysplastic syndromes with granulocyte colony-stimulating factor plus erythropoietin: results from a randomized phase II study and long-term follow-up of 71 patients. Blood. 2001 Sep 15;98(6):1985 Blood. 1998 Jul 1;92(1):68-75.

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