Phase 2 N=10 Treatment

Peptide Vaccinations to Treat Patients With Low-Risk Myeloid Cancers

Myelodysplastic Syndrome (MDS) · Acute Myeloid Leukemia (AML) · Chronic Myeloid Leukemia (CML)

Bottom Line

View on ClinicalTrials.gov: NCT00488592 ↗

Enrolled (actual)

Serious AEs

20.0%

Results posted

Jul 2014

Primary outcome: Primary: Number of Participants Who Experienced Inducing or Boosting of a Cellular Immune Response — 5; 2 participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: WT1:126-134 (Biological); PR1:169-177 Peptide (Biological); GM-CSF (Sargramostim) (Drug); Montanide adjuvant (Biological)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
Primary completion: Apr 2010

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants Who Experienced Inducing or Boosting of a Cellular Immune Response	5; 2	—
SECONDARY Number of Participants Who Experienced a Hematological Response	—	—

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 granulocyte- macrophage colony- stimulating factor (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.

Eligibility Criteria

INCLUSION CRITERIA:

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

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

Diagnosed with CML in chronic phase

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

made an informed decision not to undergo the transplant procedure

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

Human leukocyte antigen (HLA-A020 1) positive at one allele

Ages 18-85 years old

Off all lympho-ablative 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 immunosuppressants within 14 days prior to study entry
Co-morbidity 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00488592). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.