Alternate Title
Basic Trial Information
Objectives
Entry Criteria
Expected Enrollment
Outcomes
Outline
Trial Contact Information
Registry Information

Alternate Title

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

Basic Trial Information

Phase Type Status Age Sponsor Protocol IDs Phase II Treatment Active 18 to 85 NHLBI NHLBI-07-H-0159 07-H-0159, NCT00499772

Special Category: NIH Clinical Center trial

Objectives

Primary

1. 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

1. 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.

Entry 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

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

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

Expected Enrollment

Outcomes

Immune response as assessed by tetramer and intracellular staining for interferon-gamma production at baseline, during vaccination, and after completion of study therapy

Reduction in bone marrow blast cells
Changes in blood counts
Transfusion dependence
Time to disease progression
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.

Trial Contact Information

Trial Lead Organizations

National Heart, Lung, and Blood Institute

Quan Le, PhD, Principal investigator		Ph: 301-402-3294

U.S.A.
Maryland
	Bethesda
	NIH - Warren Grant Magnuson Clinical Center
		Clinical Trials Office - NIH - Warren Grant Magnuson Clinical Center	Ph:  800-411-1222

Registry Information
Official Title		Efficacy of WT1 and PR1 Peptide Vaccination for Patients with Low Risk Myeloid Malignancies
Trial Start Date		2007-06-01
Trial Completion Date		2009-12-30 (estimated)
Registered in ClinicalTrials.gov		NCT00499772
Date Submitted to PDQ		2007-06-21
Information Last Verified		2008-11-30