This trial is active, not recruiting.

Condition leukemia
Treatments pr1 leukemia peptide vaccine, sargramostim, placebo
Phase phase 3
Sponsor The Vaccine Company
Start date May 2005
End date April 2009
Trial size 244 participants
Trial identifier NCT00454168, CDR0000510853, UCCRC-14613B, VACCINE-PR1-104


RATIONALE: Vaccines made from a peptide may help the body build an effective immune response to kill cancer cells. Colony-stimulating factors, such as GM-CSF, increase the number of white blood cells and platelets found in bone marrow or peripheral blood. Giving vaccine therapy together with GM-CSF may be an effective treatment for acute myeloid leukemia. It is not yet known whether giving vaccine therapy together with GM-CSF is more effective than giving placebo together with GM-CSF in treating acute myeloid leukemia.

PURPOSE: This randomized phase III trial is studying vaccine therapy and GM-CSF to see how well they work compared with a placebo and GM-CSF in treating patients with acute myeloid leukemia in remission.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation randomized
Primary purpose treatment
Patients receive PR1 leukemia peptide vaccine and sargramostim (GM-CSF) subcutaneously.
pr1 leukemia peptide vaccine
Given subcutaneously
Given subcutaneously
(Active Comparator)
Patients receive placebo vaccine and GM-CSF subcutaneously.
Given subcutaneously
Given subcutaneously

Primary Outcomes

Overall survival
time frame:

Secondary Outcomes

Relapse-free survival
time frame:
Remission duration
time frame:
Immune response as measured by PR1-HLA-A2 tetramer assay
time frame:

Eligibility Criteria

Male or female participants at least 18 years old.

DISEASE CHARACTERISTICS: - Diagnosis of acute myeloid leukemia (AML), defined by the presence of > 20% blasts in marrow or blood, including the following subtypes: - De novo AML, defined as AML with no clinical history of prior myelodysplastic syndromes (MDS) or myeloproliferative disorder (MPD) or exposure to potentially leukemogenic therapies or agents - Secondary AML, defined as the following: - AML secondary to prior existing MDS or MPD or development of AML secondary to proven leukemogenic exposure - History of fatigue, bleeding, or recurrent infections preceding diagnosis of AML by ≥ 1 month with confirmation of existing peripheral blood film that demonstrates morphologic dysplasia - In first complete remission (CR) (patients ≥ 55 years of age) OR second CR (patients ≥ 18 years of age) within the past month - FAB stages M0-M2 and M4-M7 allowed if in first CR - No acute promyelocytic leukemia in first CR - FAB stages M0-M7 allowed if in second CR - Marrow blast count < 5% (≤ 200 nucleated cell count) - No blasts in blood - HLA-A2 positive at 1 allele - No extramedullary disease - No Auer rods - No active meningeal or CNS leukemia PATIENT CHARACTERISTICS: - ECOG performance status 0-1 - Life expectancy must not be severely limited by other diseases - Absolute neutrophil count > 1,000/mm^3 - Platelet count > 100,000/mm^3 - Bilirubin < 2 mg/mL - ALT < 2 times upper limit of normal - Creatinine ≤ 1.6 mg/mL - Not pregnant or nursing - Negative pregnancy test - Fertile patients must use effective contraception - Antineutrophil cytoplasmic antibody negative - No serious medical condition, laboratory abnormality, or psychiatric illness that would preclude study compliance or increase risk to patient - No other malignancy within the past 5 years except basal cell or squamous cell carcinoma or carcinoma in situ of the cervix or breast - No known allergy to incomplete Freund's adjuvant - No hypercalcemia - No progressive viral or bacterial infection - Must be afebrile for 7 days without antibiotics - No symptomatic cardiac disease - LVEF ≥ 40% - No symptomatic pulmonary disease - FEV_1, FVC, and DLCO ≥ 50% of predicated (without bronchodilator) - No history of HIV positivity or AIDS - No known hypersensitivity to sargramostim (GM-CSF), yeast-derived products, or any component of this product - No history of Wegener's granulomatosis or vasculitis PRIOR CONCURRENT THERAPY: - Recovered from prior surgery and/or radiotherapy - No prior allogeneic or syngeneic stem cell transplantation - No prior solid organ transplantation - No prior vaccine therapy for AML - More than 28 days since prior chronic use (> 2 weeks) of corticosteroids > 10 mg/day (prednisone [or equivalent]) - Concurrent topical or inhaled corticosteroids allowed - More than 3 months since prior experimental therapy, cyclosporine, or tacrolimus - No concurrent radiotherapy

Additional Information

Official title A Phase 3, Randomized, Double-Blind, Multicenter Study of Proteinase 3 PR1 Peptide Mixed With Montanide ISA-51 VG Adjuvant and Administered With GM-CSF in Elderly Patients With AML in First Complete Remission or Adults in Second Complete Remission: A Pivotal Study
Description OBJECTIVES: Primary - Compare improvement of overall survival of patients with acute myeloid leukemia treated with PR1 leukemia peptide vaccine and sargramostim (GM-CSF) vs placebo vaccine and GM-CSF. Secondary - Compare improvement of relapse-free survival of patients treated with these regimens. - Compare remission duration in patients treated with these regimens. - Compare immune response, as measured by PR1-HLA-A2 tetramer assay, in patients treated with these regimens. OUTLINE: This is a randomized, placebo-controlled, multicenter study. Patients are stratified according to age and complete remission (CR) (≥ 18 years of age and in second CR vs ≥ 55 years of age and in first CR), type of acute myeloid leukemia (de novo vs secondary), and cytogenetics (unfavorable vs favorable and intermediate). Patients are randomized to 1 of 2 treatment arms. - Arm I: Patients receive PR1 leukemia peptide vaccine and sargramostim (GM-CSF) subcutaneously (SC). - Arm II: Patients receive placebo vaccine and GM-CSF SC. PROJECTED ACCRUAL: A total of 244 patients will be accrued for this study.
Trial information was received from ClinicalTrials.gov and was last updated in January 2014.
Information provided to ClinicalTrials.gov by National Cancer Institute (NCI).