A Study to Evaluate Safety and Tolerability of a Therapeutic Vaccine, ASP0113, in Subjects Undergoing Allogeneic Hematopoietic Cell Transplant

An Open Phase 2 Trial to Evaluate Safety of a Therapeutic Vaccine, ASP0113, in Recipients Undergoing Allogeneic Hematopoietic Cell Transplant (HCT)

This study is to evaluate safety and tolerability of a therapeutic vaccine, ASP0113, in subjects undergoing allogeneic HCT. The occurrence of CMV viremia and immunogenicity are also assessed.

Inclusion Criteria:

• Subject is planned to undergo either of the following:

  • Sibling Donor Transplant - 7/8 Human Leukocyte Antigen (HLA)-A, -B, -C, -DRß1 match utilizing high resolution typing or 8/8 (HLA)-A, -B, -C, -DRß1 match utilizing low or high resolution typing.
  • Unrelated Donor Transplant - 7/8 or 8/8 HLA-A, -B, -C, -DRß1 match utilizing high resolution typing.

• Subject has one of the following underlying diseases: Acute myeloid leukemia (AML) /Acute lymphoblastic leukemia (ALL) / Acute undifferentiated leukemia (AUL) /Acute biphenotypic leukemia / Chronic myelogenous leukemia (CML) / Chronic lymphocytic leukemia (CLL) / myelodysplastic syndrome(s) (MDS)

• Subject is scheduled to receive an allogeneic peripheral blood stem cell (PBSC) or bone marrow transplant (BMT) for the treatment of hematologic disorders

Exclusion Criteria:

• Subject has active CMV disease or infection or has received treatment for active CMV disease or infection within 90 days prior to transplant

• Subject has planned CMV prophylactic therapy with antiviral drugs or CMV-specific immunoglobulins

• Subject has a modified hematopoietic cell transplant comorbidity index (HCT-CI) score > 3

• Subject is known to be positive for human immunodeficiency virus (HIV), hepatitis B surface antigen or hepatitis C ribonucleic acid (RNA)

• Subject has received any of the following substances or treatments:

  • T-cell depletion of donor cell product.
  • Alemtuzumab within 60 days prior to transplant, including conditioning regimen. Subjects for whom treatment with alemtuzumab is planned at any time from 60 days prior to through one year post-transplant should not be enrolled in the trial.
  • Administration of a CMV vaccine, including any prior exposure to ASP0113.

• Subject has received an allogeneic stem cell transplant within one year prior to transplant

• Subject has a current malignancy in addition to the malignancy being treated for the study or the subject has a history of any other malignancy

• Subject has an unstable medical or psychiatric condition, including a history of illicit drug(s) or alcohol abuse that the Investigator believes will interfere with protocol requirements.