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

Study Overview
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Brief Summary
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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.

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

Conditions
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Allogeneic Hematopoietic Cell Transplant

Intervention / Treatment
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* Biological: ASP0113


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 20 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ASP0113 group<br>Participants receive 1 mL of ASP0113 intramuscularly 5 times, on days -14 to -3, 14 to 40, 60 ± 5, 90 ± 10, and 180 ± 10, counting from the transplantation (stem cell transfusion) day (day 0) | Biological: ASP0113<br>* injection<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety assessed by the incidence of adverse events, vital signs, physical exam and labo-tests |  | for 365 days after HCT | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Local reactogenicity | protocol-specified reactogenicity scale | for 14 days following each injection | 
| Incidence of CMV viremia | CMV plasma viral load ≥ 1000 copies | for 365 days after HCT | 
| CMV-specified antiviral therapy | CMV-specific AVT(Anti-virus therapy) initiated for a CMV plasma viral load ≥ 1000 copies | for 365 days after HCT | 
| Incidence of cytomegalovirus end-organ disease (CMV EOD) | CMV pneumonia, CMV gastroenteritis, CMV hepatitis, et al | for 365 days after HCT | 
| Maximum grade of Graft Versus Host Disease (GVHD) |  | for 365 days after HCT | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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HCT, cytomegalovirus, DNA vaccine, transplantation