PR1 Vaccination in Myelodysplastic Syndrome (MDS)

NCT00893997 | Terminated Phase 2 Results

• 1 other identifier: 2005-0913
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

Primary aim:

1. 1.To determine the immunologic response, using a PR1-HLA-A2 tetramer assay, to 4 subcutaneous (SQ) injections of TVC-PR1 vaccine formulated in Montanide ISA 51 VG followed by granulocyte macrophage colony-stimulating factor (GM-CSF) in low risk and intermediate-1 myelodysplastic syndrome (MDS) patients.
2. 2.To determine if non-immunologic responders to 4 subcutaneous (SQ) injections of TVCPR1 vaccine formulated in Montanide ISA 51 VG followed by GM-CSF can be converted to immunologic responders by administering 4 additional doses of TVC-PR1 vaccine formulated in Montanide ISA 51 VG followed by GM-CSF.
3. 3.To determine the clinical response to 4 or 8 subcutaneous (SQ) injections of TVC-PR1 vaccine formulated in Montanide ISA 51 VG followed by GM-CSF in patients low risk and intermediate-1 MDS.

Conditions

Leukemia; Myelodysplastic Syndrome

Keywords

myelodysplastic syndrome; MDS; PR1 Peptide Vaccine

Outcome Measures

Primary Outcomes (2)

• Patient Immunologic Response

  Patients assessed after 4th vaccination for immunologic response categorized as 'Immunologic-Responders' or 'Non-Responders.' Immune response defined as an increase of ≥ 0.5 PR1-HLA-A2 tetramer cells/μl compared to the pre study absolute PR1-HLA-A2 tetramer cells/μl. Time period 29 weeks after study entry, with week 0 corresponding to 1st injection, and 8th injection thus being given at week 25, 29 weeks corresponds to 13 weeks after receipt of a 4th injection.

  29 weeks

• Number of Patients With Clinical Response

  Clinical response based on the International Working Group (IWG) Response Criteria in myelodysplastic syndromes (MDS): 'Complete Response' or Hematologic Improvement' and 'No Clinical Response'. Clinical responses as assessed by standard criteria with bone marrow biopsy, cytogenetic studies (standard chromosome banding) and molecular studies 3 weeks after the last vaccination.

  At 29 weeks

Study Arms (1)

PR-1 vaccine

EXPERIMENTAL

4 injections of 0.5 mg PR1 peptide vaccine every 3 weeks.

Biological: PR-1 vaccine

Interventions

PR-1 vaccine BIOLOGICAL

0.5 mg injections under the skin once every 3 weeks for a total of 4 vaccinations.

Also known as: PR1 Peptide Vaccine

PR-1 vaccine

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Must understand and voluntarily sign an informed consent form
• Age \>/= 18 years at the time of signing the informed consent form
• Must be able to adhere to the study visit schedule and other protocol requirements
• HLA-A2 positive at one allele
• Diagnosis of myelodysplastic syndrome (MDS) and must meet all the following criteria
• French-American-British (FAB) Class Refractory anemia (RA), Refractory Anemia with Excess Blasts (RAEB), refractory anemia with ringed sideroblasts (RARS)
• World Health Organization(WHO) Classification refractory anemia (RA), refractory anemia with ringed sideroblasts(RARS), refractory cytopenia with multilineage dysplasia (RCMD), refractory cytopenia with ringed sideroblasts (RCMD-RS) , refractory anemia with excess blasts type 1 (RAEB-1)
• Less than 20% blasts on marrow aspirate
• International Prognostic Scoring System (IPSS) risk groups Intermediate 1 or transfusion dependent low risk.
• Both de novo and therapy related MDS are eligible
• Eastern Cooperative Oncology Group (ECOG) performance status = 0 or 1
• Women of childbearing potential must have a negative serum pregnancy test within 30 days of starting study drug. A woman of child-bearing potential is a sexually mature woman who has not undergone a hysterectomy or who has not been naturally postmenopausal for at least 24 consecutive months (i.e., who has had menses at any time in the preceding 24 consecutive months)
• Male or female of child-bearing potential must agree to use adequate contraceptive methods
• Serum bilirubin \< 2 mg/ml
• Serum creatinine \</= 1.5 mg/ml

You may not qualify if:

• Marrow blasts on aspirate \>/= 20%
• Blood blasts \> 1%
• Inaspirable bone marrow
• Myelosclerosis occupying \>30% of marrow space
• Iron absence on marrow examination or transferrin saturation \<20% and serum ferritin \<50ng/ml
• B-12 deficiency
• Folate deficiency
• History of immune related hematological disorder \[i.e.,immune thrombopenia purpura(ITP),autoimmune hemolytic anemia ( AIHA)\]
• Other causes of cytopenia not related to MDS (i.e., GI blood loss)
• 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 allogeneic or syngeneic transplant
• Prior solid organ transplant
• Life expectancy severely limited by diseases other than MDS
• Pregnant or lactating females
• Prior vaccine therapy for MDS

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• The Vaccine Company: collaborator

Study Sites (1)

U.T. M.D. Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• UT MD Anderson Cancer Center website

MeSH Terms

Conditions

Leukemia; Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases

Results Point of Contact

• Title: Guillermo Garcia-Manero, MD / Associate Professor
• Organization: UT MD Anderson Cancer Center

eharriso@mdanderson.org

713-792-7305

Study Officials

• Guillermo Garcia-Manero, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 4, 2009
• First Posted: May 6, 2009
• Study Start: July 1, 2006
• Primary Completion: March 1, 2009
• Study Completion: March 1, 2009
• Last Updated: July 16, 2012
• Results First Posted: April 12, 2011

Record last verified: 2012-07

Locations

US (1)