MedPath

Multi-antigen CMV-Modified Vaccinia Ankara Vaccine in Reducing CMV Related Complications in Patients With Blood Cancer Undergoing Donor Stem Cell Transplant

Phase 2
Withdrawn
Conditions
Acute Lymphoblastic Leukemia in Remission
Hodgkin Lymphoma
Myelofibrosis
Myeloproliferative Neoplasm
Non-Hodgkin Lymphoma
Chronic Lymphocytic Leukemia
Hematopoietic Cell Transplantation Recipient
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
Myelodysplastic Syndrome
Interventions
Biological: Multi-antigen CMV-Modified Vaccinia Ankara Vaccine
Other: Laboratory Biomarker Analysis
Other: Placebo
Registration Number
NCT03438344
Lead Sponsor
City of Hope Medical Center
Brief Summary

This randomized phase II trial studies how well multi-antigen cytomegalovirus (CMV)-modified vaccinia Ankara vaccine works in reducing CMV related complications in patients with blood cancer who are undergoing donor stem cell transplant. Vaccines made from a gene-modified virus may help the body build an effective immune response to kill cancer cells.

Detailed Description

PRIMARY OBJECTIVES:

I. To determine if multi-antigen CMV-modified vaccinia Ankara vaccine (CMV-modified vaccinia Ankara \[MVA\] triplex) reduces the frequency of CMV events, defined as reactivation or CMV disease in CMV+, haploidentical hematopoietic cell transplantation (haploHCT) recipients.

SECONDARY OBJECTIVES:

I. To evaluate the safety and tolerability of CMV-MVA triplex in vaccinated, haploHCT recipients by assessing the following: non-relapse mortality (NRM) at 100 days post hematopoietic cell transplantation (HCT), severe (grade 3-4) acute graft versus host disease (GVHD), and grade 3-4 adverse events (AEs) probably or definitely related to the vaccination within 2 weeks from each vaccination.

II. To characterize CMV related events in recipients of CMV-MVA triplex compared to placebo, by assessing time to viremia (number of days from transplantation to the date of \>= 1250 IU/mL), duration of viremia, recurrence of viremia, incidence of late CMV viremia/disease (\> 100 and =\< 360 days post HCT), use of antiviral drugs (triggered by clinically significant viremia of \>= 1250 IU/mL), cumulative number of CMV specific antiviral treatment days.

III. To evaluate the impact of CMV-MVA triplex on transplant related outcomes by assessing the incidence of acute graft versus host disease (aGVHD), chronic GVHD, relapse, non-relapse mortality, all-cause mortality, infections.

IV. To determine if CMV-MVA triplex increases levels, function and kinetics of CMV-specific T cell immunity in vaccinated compared to placebo treated, CMV seropositive HCT-recipients.

V. To determine whether vaccination induces adaptive natural killer (NK) cell population changes, and increase in the highly cytotoxic memory NKG2C+ NK cell.

VI. To compare GVHD biomarkers between the vaccine and placebo groups.

OUTLINE: Patients are randomized into 1 of 2 arms.

ARM I: Patients receive multi-antigen CMV-modified vaccinia Ankara vaccine intramuscularly (IM) on days 28 and 56 post-HCT.

ARM II: Patients receive placebo IM on days 28 and 56 post-HCT.

After completion of study treatment, patients are followed up at 100, 140, 180, 270, and 365 days, and then periodically for 3 years.

Recruitment & Eligibility

Status
WITHDRAWN
Sex
All
Target Recruitment
Not specified
Inclusion Criteria
  • All subjects must have the ability to understand and the willingness to sign a written informed consent

  • Participant must be willing to comply with study and/or follow-up procedures, including willingness to be followed for one year post-HCT

  • Planned peripheral blood stem cell (PBSC) or bone marrow (BM) HCT for the treatment of the following hematologic malignancies:

    • Lymphoma (Hodgkin and non-Hodgkin)

    • Myelodysplastic syndrome

    • Acute lymphoblastic leukemia in first or second remission (for acute lymphoblastic leukemia/lymphoblastic lymphoma, the disease status must be in hematologic remission by bone marrow and peripheral blood; persistent lymphadenopathy on computed tomography [CT] or CT/positron emission tomography [PET] scan without progression is allowed)

    • Acute myeloid leukemia in first or second remission

    • Chronic myelogenous leukemia in first chronic or accelerated phase, or in second chronic phase

    • Other hematologic malignancies judged appropriate by the clinical principal investigators (PIs), including chronic lymphocytic leukemia, myeloproliferative disorders and myelofibrosis; patients with multiple myeloma and those with non-malignant disease such as aplastic anemia are excluded

      • Adult cases of multiple myeloma (MM) are excluded; adults with aplastic anemia are excluded; patients undergoing a second haploHCT are not eligible (patients who have undergone a previous autologous HCT are eligible)
  • Patients receiving myeloablation (MA) or reduced intensity conditioning (RIC) are allowed

  • CMV seropositive (recipient)

  • Planned related HCT with molecular 3/6 (haploidentical) intermediate/high resolution human leukocyte antigen (HLA) donor allele matching

  • Planned HCT with minimal to no-T cell depletion of graft

  • Conditioning and immunosuppressive regimens according to institutional guidelines are permitted

  • Negative serum or urine beta-human chorionic gonadotropin (HCG) test (female patient of childbearing potential only) within two weeks of registration

  • Seronegative for human immunodeficiency virus (HIV), hepatitis C virus (HCV) and active hepatitis B virus (HBV) (surface antigen negative) within 2 months of registration and no history of disseminated cutaneous human papillomavirus (HPV) related disease

  • Agreement by females of childbearing potential and sexually active males to use an effective method of contraception (hormonal or barrier method of birth control or abstinence) prior to study entry and for up to 90 days post-HCT; should a woman become pregnant or suspect that she is pregnant while participating on the trial, she should inform her treating physician immediately

Exclusion Criteria
  • Any prior investigational CMV vaccine
  • Experimental anti-CMV chemotherapy in the last 6 months
  • Live attenuated vaccines
  • Medically indicated subunit (Engerix-B for HBV, Gardasil for HPV) or killed vaccine (e.g. influenza, pneumococcal, or allergy treatment with antigen injections)
  • Allergy treatment with antigen injections
  • Alemtuzumab or any equivalent in vivo T-cell depleting agent (or CD34+ selection)
  • Antiviral medications with known therapeutic effects on CMV such as ganciclovir/valganciclovir (GCV/VAL), foscarnet (FOS), Cidofovir, CMX-001, maribavir; acyclovir has no known therapeutic efficacy against CMV and is allowable as standard of care to prevent herpes simplex virus (HSV)
  • Prophylactic therapy with CMV immunoglobulin or prophylactic antiviral CMV treatment
  • Conditioning regimens 30 days (d) prior to trial participation and up to d28 post-HCT
  • Disease-based radiation therapy (not total body irradiation)
  • Other investigational product-concurrent enrollment in other clinical trials using any investigational new drug (IND) drugs with unknown effects on CMV or with unknown toxicity profiles is prohibited
  • Other medications that might interfere with the evaluation of the investigational product
  • Patients with active autoimmune conditions requiting systemic immunosuppressive therapy within the previous 5 years at not eligible
  • Patients considered by PIs/protocol team to have a complicated prior therapy or HCT regimen, or who have a low survival probability (e.g., refractory leukemia and/or undergoing 2nd HCT)
  • Poor risk disease/disease status including: chronic myeloid leukemia (CML) in blast crisis, acute myeloid leukemia (AML)/acute lymphoblastic leukemia (ALL) beyond 2nd remission, multiple myeloma, and aplastic anemia
  • Pregnant women and women who are lactating; breastfeeding should be discontinued if the mother is enrolled on this study
  • Any other condition that would, in the investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns or compliance with clinical study procedures, e.g., social/psychological issues, etc
  • Prospective participants who, in the opinion of the investigator, may not be able to comply with all study procedures (including compliance issues related to feasibility/logistics)

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Arm II (placebo)PlaceboPatients receive placebo via injection on days 28 and 56 post-HCT.
Arm I (CMV-MVA triplex vaccine)Multi-antigen CMV-Modified Vaccinia Ankara VaccinePatients receive multi-antigen CMV-modified vaccinia Ankara vaccine via injection on days 28 and 56 post-HCT.
Arm II (placebo)Laboratory Biomarker AnalysisPatients receive placebo via injection on days 28 and 56 post-HCT.
Arm I (CMV-MVA triplex vaccine)Laboratory Biomarker AnalysisPatients receive multi-antigen CMV-modified vaccinia Ankara vaccine via injection on days 28 and 56 post-HCT.
Primary Outcome Measures
NameTimeMethod
Cytomegalovirus (CMV) eventsUp to 3 years

Will include any CMV reactivation (detection of \>= 1250 U/mL plasma), low-level reactivation prompting antiviral therapy, or CMV disease (defined by histology) prior to day 100 post-hematopoietic cell transplantation (HCT).

Secondary Outcome Measures
NameTimeMethod
Non-relapse mortalityUp to 3 years
RelapseUp to 3 years
All-cause mortalityUp to 3 years
Overall survivalUp to 3 years
Non-relapse mortality (NRM)At 100 days post HCT
Duration of viremiaUp to 3 years

The primary statistical analyses will compare vaccine and placebo regarding hazards of CMV events from injection to day 100. Events prior to injection will inform Kaplan Meier estimates for both arms to obtain unbiased estimates of reactivation rates applicable to all transplanted subjects on each arm.

Duration of anti-CMV therapyUp to 3 years
Incidence of late CMV reactivation or diseaseUp to 3 years
Time to engraftmentUp to 3 years
Incidence of chronic GVHDUp to 3 years
Severe (grade 3-4) acute graft versus host disease (GVHD)Up to 3 years
Grade 3-4 adverse events probably/definitely related to the vaccination and modified vaccinia Ankara vector persistenceUp to 3 years
Peak CMV polymerase chain reaction valueUp to 3 years
Recurrence of CMV viremiaUp to 3 years
Incidence of acute GVHDUp to 3 years

Will be scored using Keystone consensus criteria. Whenever possible, acute GVHD will be confirmed histologically with microscopic review.

InfectionsUp to 3 years
Natural killer cell functionUp to 3 years

Will assess cytotoxicity of NK cells against K562 target cell line compared with control (% killing) and cytokine production using flow-cytometry with intra-cellular staining of gamma-interferon and other cytokines (% of NK cells producing these cytokines).

Levels of CMV-specific T cell immunityUp to 3 years

Will also assess immunophenotyping and function.

Trial Locations

Locations (3)

City of Hope Medical Center

🇺🇸

Duarte, California, United States

Northside Hospital

🇺🇸

Atlanta, Georgia, United States

Dana-Farber Cancer Institute

🇺🇸

Boston, Massachusetts, United States

© Copyright 2025. All Rights Reserved by MedPath