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.

Study Overview

• Status: Completed
• Conditions: Allogeneic Hematopoietic Cell Transplant
• Intervention / Treatment: Biological: ASP0113

• Study Type: Interventional
• Enrollment (Actual): 9
• Phase: Phase 2

Contacts and Locations

Study Locations

• Japan
  • Kantou, Japan
  • Kyushu, Japan

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 20 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ASP0113 group; 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; injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Collaborators and Investigators

• Sponsor: Astellas Pharma Inc
• Investigators: Study Director: Medical Director, Astellas Pharma Inc

Publications and helpful links

General Publications

• Mori T, Kanda Y, Takenaka K, Okamoto S, Kato J, Kanda J, Yoshimoto G, Gondo H, Doi S, Inaba M, Kodera Y. Safety of ASP0113, a cytomegalovirus DNA vaccine, in recipients undergoing allogeneic hematopoietic cell transplantation: an open-label phase 2 trial. Int J Hematol. 2017 Feb;105(2):206-212. doi: 10.1007/s12185-016-2110-3. Epub 2016 Oct 28.

Helpful Links

• Link to results on the Astellas Clinical Study Results website

Study record dates

Study Major Dates

• Study Start (Actual): June 20, 2013
• Primary Completion (Actual): January 19, 2015
• Study Completion (Actual): January 19, 2015

Study Registration Dates

• First Submitted: July 1, 2013
• First Submitted That Met QC Criteria: July 17, 2013
• First Posted (Estimated): July 19, 2013

Study Record Updates

• Last Update Posted (Actual): October 17, 2024
• Last Update Submitted That Met QC Criteria: October 15, 2024
• Last Verified: October 1, 2024

More Information

Terms related to this study

• Keywords: transplantation; cytomegalovirus; HCT; DNA vaccine
• Other Study ID Numbers: 0113-CL-1003

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Access to anonymized individual participant level data will not be provided for this trial. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.