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Myelodysplastic SyndromesPhase 2NCT00513578

Vaccine Therapy and GM-CSF in Treating Patients With Low-Risk or Intermediate-Risk Myelodysplastic Syndrome

A Phase 2 study testing PR1 leukemia peptide vaccine, incomplete Freund's adjuvant and sargramostim in people with Myelodysplastic Syndromes, run by The Vaccine Company at 1 site in United States.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

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, increase the number of white blood cells and platelets found in bone marrow or peripheral blood. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myelodysplastic Syndromes
What is being tested?
PR1 leukemia peptide vaccine, incomplete Freund's adjuvant and sargramostim
Type of treatment
Vaccines
How many people
30 participants
Where
1 siteUnited States
Study dates
Jan 2007to Jan 2009
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Diagnosis of myelodysplastic syndromes (MDS) and must meet all of the following criteria:
  • FAB class refractory anemia (RA), RA with excess blasts (RAEB), or RA with ringed sideroblasts (RARS)
  • WHO Classification RA, RARS, refractory cytopenia with multilineage dysplasia (RCMD), RCMD with ringed sideroblasts, or RAEB-1
  • Less than 20% blasts on marrow aspirate
  • IPSS risks groups intermediate-1- OR transfusion dependent low-risk
  • Patients with de novo or therapy-related MDS eligible
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  • HLA-A2 positive at one allele
  • ECOG performance status 0 or 1
  • Women of childbearing potential must have a negative serum pregnancy test within 30 days of starting study drug
  • Male or female of child-bearing potential must agree to use adequate contraceptive methods
  • Serum bilirubin < 2 mg/mL
  • Creatinine ≤ 1.5 mg/mL
  • ALT < 2 times upper normal limit
  • Antineutrophil cytoplasmic antibody (cANCA) negative

Who cannot join

  • RAEB in transformation or RAEB-2
  • Chloroma
  • Marrow blasts on aspirate ≥ 20%
  • Blood blasts > 1%
  • Inaspirable bone marrow
  • History or current myelosclerosis occupying > 30% of marrow space
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  • History of acute myeloid leukemia
  • Other causes of cytopenia not related to MDS (i.e., gastrointestinal blood loss)
  • PATIENT CHARACTERISTICS:
  • Pregnant or lactating
  • Iron absence on marrow examination or transferrin saturation < 20% and serum ferritin < 50ng/mL
  • B12 deficiency
  • Folate deficiency
  • History of immune-related hematological disorder (i.e., idiopathic thrombocytopenic purpura, autoimmune hemolytic anemia)
  • Life expectancy severely limited by diseases other than MDS
  • Prior history of malignancy other than MDS (except basal cell or squamous cell carcinoma or carcinoma in situ of the cervix or breast) unless the subject has been free of disease for ≥ 5 years
  • Known allergy to incomplete Freund's adjuvant
  • Hypercalcemia
  • Progressive viral or bacterial infection
  • All infections must be resolved and the patient has remained afebrile for seven days without antibiotics
  • Cardiac disease of symptomatic nature or cardiac ejection fraction < 40%
  • History of Wegener granulomatosis or vasculitis
  • Symptomatic pulmonary disease or FEV_1, FVC, and DLCO ≤ 50% predicted
  • History of HIV positivity or AIDS
  • Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form or that will place the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret the data
  • PRIOR CONCURRENT THERAPY:
  • Has received specific therapy for MDS within the past 4 weeks
  • Prior allogeneic or syngeneic transplant
  • Prior solid organ transplant
  • Chronic use (> 2 weeks) of greater than physiologic doses of a corticosteroid agent (dose equivalent to > 10 mg/day of prednisone) within 30 days of the first day of study drug treatment
  • Topical and inhaled corticosteroids are permitted
  • Experimental therapy, cyclosporine, antithymocyte globulin, or tacrolimus within 3 months of study entry
  • Treatment with androgenic hormones, danazol, colony-stimulating factors, erythropoietin, thalidomide, arsenic trioxide or other agents used to treat MDS within four weeks of the first day of study treatment
  • Prior vaccine therapy for MDS
  • Prohibited medications during study, including any of the following:
  • Systemic steroids except as required for transfusion reactions
  • Chemotherapy or other investigational drugs
  • Sargramostim (GM-CSF) (except as part of study regimen)
  • Filgrastim (G-CSF)
  • Interleukin-11

Trial locations and contacts

1 site in 1 country.

United States1 site

  • M. D. Anderson Cancer Center at University of Texas

    Houston, Texas, 77030-4009

Other trials in United States

About this study

Official title: Phase 2 Study of Proteinase 3 PR1 Peptide Mixed With Montanide ISA 51 VG Adjuvant and Administered With GM-CSF in Low Risk and Intermediate-1 MDS

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, increase the number of white blood cells and platelets found in bone marrow or peripheral blood. Giving vaccine therapy together with GM-CSF may kill more cancer cells.

PURPOSE: This phase II trial is studying how well giving vaccine therapy together with GM-CSF works in treating patients with low-risk or intermediate-risk myelodysplastic syndrome.

Read the detailed description

OBJECTIVES:

Primary

  • To determine the immunologic response, using a PR1-HLA-A2 tetramer assay, to 4 subcutaneous injections of PR1 leukemia peptide vaccine formulated in incomplete Freund's adjuvant (IFA) followed by sargramostim (GM-CSF) in patients with low- and intermediate-1-risk myelodysplastic syndromes.

Secondary

  • To determine if non-immunologic responders to 4 subcutaneous injections of PR1 leukemia peptide vaccine formulated in IFA followed by GM-CSF can be converted to immunologic responders by administering 4 additional doses of this treatment.
  • To determine the clinical response to 4 or 8 subcutaneous injections of this vaccine.

OUTLINE: This is a multicenter study.

Patients will receive proteinase PR1 leukemia peptide vaccine (TVC-PR1) conjugated with incomplete Freund's adjuvant administered subcutaneously with sargramostim (GM-CSF). Patients will receive a series of four vaccinations at 3-week intervals. Non-immunologic responders after 4 doses of vaccine are eligible to receive 4 additional doses of TVC-PR1 vaccine with the same dose and same dosing intervals. Patients who mount an immunologic response after 4 doses will not receive additional doses of TVC-PR1 vaccine.

After completion of study therapy, patients are followed monthly for up to 6 months.

Being tested
PR1 leukemia peptide vaccine, incomplete Freund's adjuvant, sargramostim
Keywords
refractory anemia with excess blasts, refractory anemia with ringed sideroblasts, refractory anemia, refractory cytopenia with multilineage dysplasia, de novo myelodysplastic syndromes, secondary myelodysplastic syndromes
Medical subject headings
Myelodysplastic Syndromes, Anemia, Refractory, with Excess of Blasts, Anemia, Refractory

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PR1 leukemia peptide vaccineBiological

  • incomplete Freund's adjuvantBiological

  • sargramostimBiological

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Immunologic response after four injections of vaccine formulation as determined by an increase in the absolute PR1-HLA-A2 tetramer count by at least 0.5/μl

Secondary outcomes (2)

  1. Conversion of non-immunologic responders to immunologic responders by administering 4 additional doses of vaccine

  2. Clinical response as determined by modified IWG criteria

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Aug 2007 (estimated)
  2. Study startJan 2007
  3. Primary completionJan 2009 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Masking
None (open label)
Enrolment
30 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00513578
Sponsor's protocol ID
CDR0000558127
Other IDs
VACCINE-PR1-204
Status last verified
Feb 2009
First submitted
6 Aug 2007
Last updated
6 Jan 2014
Results posted
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing PR1 leukemia peptide vaccine, incomplete Freund's adjuvant and sargramostim in people with Myelodysplastic Syndromes, run by The Vaccine Company at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 40 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 6 Jan 2014. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Immunologic response after four injections of vaccine formulation as determined by an increase in the absolute PR1-HLA-A2 tetramer count by at least 0.5/μl. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00513578.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jan 2014.

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