Project 2

CMVPepVAX to protect stem cell transplant recipients from CMV infection
Pending success of the pilot trial, the National Cancer Institute will provide a fresh lot of CMVPepVax to support a multicenter phase II study jointly with the University of Minnesota Comprehensive Cancer Center. We propose two phase II trials in hematopoietic cell transplantation (HCT) with the goal of preventing cytomegalovirus (CMV) reactivation. We will conduct a randomized placebo-controlled phase II study (Trial 1) powered to test the primary endpoint of reduced CMV reactivation in  matched related donor (MRD) HCT. Nested within this trial, we will assess immunologic efficacy of vaccinating the MRD to augment levels and function of CMV immunity that are concurrently boosted by the vaccine in HCT recipients. Immunologic 20 endpoints will be quantified by frequency measurements of CMV-specific T cells using HLA tetramers and functional studies with T-box transcription factors, T-bet and Eomes that will assess improved effector function in vaccinated patients. Trial 2 will test protective function of CMVPepVax in higher risk unrelated donor (URD) or umbilical cord blood (UCB) recipients. This randomized trial will address a broad range of URD settings, stratified as low risk 8/8 HLA match, or higher risk 7/8 match URD, or lesser matched UCB grafts, all resulting in a higher risk of CMV reactivation compared to MRD. URD recipients will be immunized at City of Hope while UCB recipients will be exclusively immunized at the University of Minnesota, and results of Trial 2 participants will be combined to assess protective efficacy. The goal is to capitalize on our phase I success with CMVPepVax by conducting phase II studies that will not only establish the therapeutic benefit for HCT recipients at risk for complications of CMV infection, but as well define the immunologic basis for this protection.
Ryotaro Nakamura, M.D.
Principal Investigator of IRB12022

Jeff Longmate, Ph.D.
Director, Department of Biostatistics
Jennifer Drake, C.C.R.P.
Study Coordinator

Cindy Slape, R.N.
Clinical Research Nurse
Corinna La Rosa, Ph.D.
Associate Research Professor
Conducts the laboratory investigations

Staff of the Department of Hematology & Hematopoietic Cell Transplantation
Recruits and consents patients
Michael Verneris, M.D.
Pediatric Leukemia Program Director, University of Minnesota
Principal Investigator at the University of Minnesota site

Jeff Miller, M.D.
Director, Cancer Experimental Therapeutics Initiative (CETI), University of Minnesota
Co-investigator at the University of Minnesota site