Vaccine Therapy in Treating Patients With Philadelphia Chromosome-Positive Chronic Myelogenous Leukemia
Phase II Multicenter Study of P210-B3A2 Derived Peptide Vaccine in Chronic Myeloid Leukemia Patients in Complete Cytogenetic Response With Persistent Molecular Residual Disease During Imatinib Treatment
  • Phase

    Phase 2
  • Study Type

  • Study Participants

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 booster vaccinations may make a stronger immune response and prevent or delay the recurrence of cancer.

PURPOSE: This phase II trial is studying how well vaccine therapy works in treating patients with Philadelphia chromosome-positive chronic myelogenous leukemia.


Determine the activity of bcr-abl p210-b3a2 breakpoint-derived pentapeptide vaccine (CMLVAX100), in terms of peripheral blood bcr-abl/abl ratio reduction, in patients with Philadelphia chromosome-positive chronic myelogenous leukemia.


Determine the reduction of molecular residual disease at 3 months in patients treated with this vaccine.
Determine the reduction of molecular residual disease at 12 months in patients treated with maintenance boosts of this vaccine.
Determine the rate of complete molecular response at any time after vaccination.
Determine in vivo and in vitro peptide-specific immune response induced by the vaccine.

OUTLINE: This is a prospective, nonrandomized, open-label, multicenter study.

Patients receive sargramostim (GM-CSF) subcutaneously (SC) on days 1 and 2 and bcr-abl p210-b3a2 breakpoint-derived pentapeptide vaccine (CMLVAX100) SC on day 2. Treatment repeats every 2 weeks for 6 courses. Patients then receive CMLVAX100 SC once monthly for 3 months and then once every 3 months for 6 months (for a total of 1 year). Patients may receive additional CMLVAX100 SC every 6 months for at least 3 years. Treatment continues in the absence of disease progression or unacceptable toxicity.

PROJECTED ACCRUAL: A total of 69 patients will be accrued for this study.
Study Started
Mar 31
Primary Completion
Jul 31
Study Completion
Jul 31
Results Posted
Aug 28
Last Update
Aug 28

Biological bcr-abl p210-b3a2 breakpoint-derived multipeptide vaccine

Biological sargramostim



Diagnosis of chronic myelogenous leukemia (CML) meeting the following criteria:

Philadelphia chromosome positive disease
b3a2 breakpoint mutation

Prior treatment with conventional imatinib mesylate for ≥ 18 months required

Complete cytogenetic response documented on ≥ 2 different examinations

Persistence of molecularly detectable residual disease (any level of bcr-abl transcript)
Patients continue to receive imatinib mesylate at the same dose (conventional treatment) during study treatment


WHO performance status 0-1
Bilirubin ≤ 2 times upper limit of normal (ULN)
AST and ALT ≤ 2.5 times ULN
Creatinine ≤ 1.5 times ULN
Not pregnant or nursing
Negative pregnancy test
Fertile patients must use effective contraception
No severe active infection or other serious medical illness that would preclude study completion
No known immunodeficiency
No autoimmune disorders


No concurrent immunosuppression or systemic immunosuppressive medication
No concurrent dose escalation of imatinib mesylate
No other concurrent investigational products


Chronic Myeloid Leukemia (CML) Patients

All Events

Event Type Organ System Event Term Chronic Myeloid Leukemia (CML) Patients

Number of Patients Showing a Reduction by at Least 50% of Peripheral Blood BCR-ABL/ABL Ratio Compared to the Individual Prevaccine Level

Response rate evaluated after immunization and reinforcement boosts (evaluation after 6 months, ) and persisting at the 9th month (after 10th vaccination)

Chronic Myeloid Leukemia (CML) Patients

50% reduction at 6 months


50% reduction at 9 months


Number of Patients With Undetectable Transcript at Any Time After Immunization

Chronic Myeloid Leukemia (CML) Patients


Number of Patients With Peptide-specific Immune Response Induced by the Vaccinations

A significant in vitro b3a2-peptide-specific CD4+ T cell proliferation

Chronic Myeloid Leukemia (CML) Patients


Age, Continuous

years (Median)
Full Range: 29.0 to 78.0

Region of Enrollment

Sex: Female, Male

Overall Study

Chronic Myeloid Leukemia (CML) Patients

Drop/Withdrawal Reasons

Chronic Myeloid Leukemia (CML) Patients