A Study to Evaluate a Therapeutic Vaccine, ASP0113, in Cytomegalovirus (CMV)-Seropositive Recipients Undergoing Allogeneic, Hematopoietic Cell Transplant (HCT) (HELIOS)

October 15, 2024 updated by: Astellas Pharma Global Development, Inc.

A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial to Evaluate the Protective Efficacy and Safety of a Therapeutic Vaccine, ASP0113, in Cytomegalovirus (CMV)-Seropositive Recipients Undergoing Allogeneic, Hematopoietic Cell Transplant (HCT)

The purpose of the study was to evaluate the efficacy of ASP0113 compared with placebo as measured by a primary composite endpoint of overall mortality and CMV end organ disease (EOD) through 1 year post-transplant. Safety of ASP0113 in participants undergoing allogeneic HCT will also be evaluated.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Participants will be followed for 5.5 years post-transplant for long-term safety via an annual telephone contact.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
514

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • Queensland
      • Herston, Queensland, Australia, QLD 4029
        • Site AU43001
    • South Australia
      • Adelaide, South Australia, Australia, SA 5000
        • Site AU43004
  • Belgium
      • Brugge, Belgium, 8000
        • Site BE32001
      • Leuven, Belgium, 3000
        • Site BE32005
      • Roeselare, Belgium, 8800
        • Site BE32007
  • Canada
      • Quebec, Canada, G1R 2J6
        • Site CA15003
    • British Columbia
      • Vancouver, British Columbia, Canada, V5Z 1M9
        • Site CA15001
    • Ontario
      • Toronto, Ontario, Canada, M5G 2M9
        • Site CA15002
    • Quebec
      • Montreal, Quebec, Canada, H2W 1S6
        • Site CA15004
  • France
      • Besancon, France, 25030
        • Site FR33011
      • Creteil, France, 94000
        • Site FR33005
      • Nantes, France, 44093
        • Site FR33009
      • Nice, France, 06200
        • Site FR33010
  • Germany
      • Bonn, Germany, 53127
        • Site DE49010
      • Dusseldorf, Germany, 40225
        • Site DE49012
      • Gottingen, Germany, 37075
        • Site DE49013
      • Koln, Germany, 50937
        • Site DE49014
      • Leipzig, Germany, 04103
        • Site DE49002
      • Mainz, Germany, 55131
        • Site DE49015
      • Muenster, Germany, 48149
        • Site DE49007
      • Stuttgart, Germany, 70376
        • Site DE49005
      • Tuebingen, Germany, 72076
        • Site DE49008
      • Ulm, Germany, 89081
        • Site DE49006
      • Wurzburg, Germany, 97080
        • Site DE49001
  • Japan
      • Fukuoka, Japan
        • Site JP81005
      • Fukuoka, Japan
        • Site JP81001
      • Osaka, Japan
        • Site JP81006
    • Aichi
      • Nagoya, Aichi, Japan
        • Site JP81003
    • Gunma
      • Maebashi, Gunma, Japan
        • Site JP81011
    • Hokkaido
      • Sapporo, Hokkaido, Japan
        • Site JP81007
    • Tokyo
      • Bunkyo-ku, Tokyo, Japan
        • Site JP81010
      • Chuo-ku, Tokyo, Japan
        • Site JP81008
      • Minato-ku, Tokyo, Japan
        • Site JP81009
      • Shinjuku-ku, Tokyo, Japan
        • Site JP81004
  • Korea, Republic of
      • Seoul, Korea, Republic of, 05505
        • Site KR82002
      • Seoul, Korea, Republic of, 110744
        • Site KR82003
      • Seoul, Korea, Republic of, 137-701
        • Site KR82001
    • Seoul Teugbyeolsi
      • Seoul, Seoul Teugbyeolsi, Korea, Republic of, 06351
        • Site KR82004
  • Spain
      • Badalona, Spain, 08615
        • Site ES34001
      • Barcelona, Spain, 08041
        • Site ES34006
      • Barcelona, Spain, 08908
        • Site ES34004
      • Cordoba, Spain, 14004
        • Site ES34005
      • Granada, Spain, 18012
        • Site ES34003
      • Madrid, Spain, 28028
        • Site ES34011
      • Murcia, Spain, 30008
        • Site ES34007
      • Salamanca, Spain, 37007
        • Site ES34010
      • Santander, Spain, 39008
        • Site ES34002
      • Valencia, Spain, 46010
        • Site ES34009
  • Sweden
      • Gothenburg, Sweden, 413 45
        • Site SE46001
      • Linkoping, Sweden, 581 85
        • Site SE46006
      • Lund, Sweden, 221 85
        • Site SE46004
      • Stockholm, Sweden, 14186
        • Site SE46003
      • Umea, Sweden, 901 85
        • Site SE46005
  • Taiwan
      • Taipei City, Taiwan, 10002
        • Site TW88601
      • Taipei City, Taiwan, 11217
        • Site TW88602
      • Taoyuan County, Taiwan, 33305
        • Site TW88603
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • Site US10028
    • Arizona
      • Tucson, Arizona, United States, 85724
        • Site US10044
    • California
      • San Francisco, California, United States, 94143
        • Site US10035
      • Stanford, California, United States, 94305
        • Site US10026
    • Florida
      • Tampa, Florida, United States, 33612
        • Site US10030
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Site US10012
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Site US10013
    • Indiana
      • Indianapolis, Indiana, United States, 46237
        • Site US10007
    • Kansas
      • Westwood, Kansas, United States, 66205
        • Site US10020
    • Kentucky
      • Louisville, Kentucky, United States, 40202
        • Site US10010
    • Maryland
      • Baltimore, Maryland, United States, 21201
        • Site US10043
      • Baltimore, Maryland, United States, 21205
        • Site US10011
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Site US10021
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Site US10036
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Site US10042
    • New Jersey
      • Hackensack, New Jersey, United States, 07601-2105
        • Site US10023
    • New York
      • New York, New York, United States, 10032
        • Site US10047
      • Rochester, New York, United States, 14642
        • Site US10027
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27514
        • Site US10025
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Site US10016
    • Texas
      • Dallas, Texas, United States, 75246
        • Site US10002
      • Houston, Texas, United States, 77030
        • Site US10045
    • Utah
      • Salt Lake City, Utah, United States, 84143
        • Site US10046
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Site US10031
    • Washington
      • Seattle, Washington, United States, 98108
        • Site US10039
      • Seattle, Washington, United States, 98109
        • Site US10024
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Site US10019

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

14 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Participant is a CMV-seropositive HCT recipient

  • Participant is planned to undergo either of the following:

    • Sibling Donor Transplant
    • Unrelated Donor Transplant

  • Participant 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).
    • A defined myelodysplastic syndrome(s) (MDS)
    • Primary or secondary myelofibrosis
    • Lymphoma (including Hodgkin's)

Exclusion Criteria:

  • Participant has active CMV disease or infection or has received treatment for active CMV disease or infection within 3 months (90 days) prior to transplant
  • Participant has a modified hematopoietic cell transplant comorbidity index (HCT-CI) score ≥ 4
  • Participant has received a prior HCT and has residual Chronic Graft-versus-host Disease (cGVHD)
  • Participant who is scheduled to have a cord blood transplant or a haploidentical transplant
  • Participant has a platelet count of less than 50,000 mm3 within 3 days prior to randomization (platelet transfusions are allowed)
  • Participant has aplastic anemia or multiple myeloma

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: ASP0113
Participants received 1 mL of 5 mg/mL of ASP0113 via intramuscular injection in the deltoid muscle alternating sides with each dose on days -14 to -3 pretransplant, 14 to 40, 60, 90 and 180 in relation to the day of transplant (Day 0).
Biological: ASP0113
Intramuscular injection
Placebo Comparator: Placebo
Participants received 1 mL of 5 mg/mL of matching placebo via intramuscular injection in the deltoid muscle alternating sides with each dose on days -14 to -3 pretransplant, 14 to 40, 60, 90 and 180 in relation to the day of transplant (Day 0).
Drug: Placebo
Intramuscular injection

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Percentage of Participants With Composite of All-Cause Mortality and Adjudicated Cytomegalovirus End Organ Disease (CMV EOD) Through 1 Year Post Transplant
Time Frame: From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
This was the composite of all-cause mortality and adjudicated CMV EOD through 1 year posttransplant, The CMV EOD was assessed by the independent and blinded adjudication committee, which counted events that were observed up to day 380 from transplantation. Deaths that occurred up to day 365 from transplant were also counted.
From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Percentage of Participants With Protocol-Defined CMV Viremia Through 1 Year Posttransplant
Time Frame: From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
Protocol-defined CMV viremia was defined as a CMV plasma viral load ≥1000 IU/mL as assessed by the central laboratory. Rate was based on cumulative incidence function estimated at 1 year. The central laboratory had the lower limit of quantification [LLOQ] for CMV viral load assessment, so when the viral load was below the LLOQ the actual viral load reading was not possible and was denoted as ≤LLOQ. If participant had any CMV viral load assessments greater than the LLOQ it was classified as viremic.
From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
Percentage of Participants With Adjudicated CMV-Specific Antiviral Therapy (AVT) Through 1 Year Posttransplant
Time Frame: From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
The CMV-specific AVT use was adjudicated by the independent and blinded committee. When the CMV-specific AVT was initiated, a central CMV viral load was obtained weekly until it was discontinued. Participants without any CMV-specific AVT events were censored on the last study evaluation.
From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
Percentage of Participants With a Composite Endpoint of Protocol-defined CMV Viremia and Adjudicated CMV-Specific AVT Use
Time Frame: From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
Protocol-defined CMV viremia was as CMV plasma viral load ≥ 1000 IU/mL as assessed by the central laboratory. The CMV-specific AVT was determined by the adjudication committee. Participants with no posttransplant viral load data were excluded from the analysis.
From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
Percentage of Participants With First Occurrence of Adjudicated CMV-specific AVT or Adjudicated Diagnosis of CMV EOD After Study Drug First Injection Through 1 Year Posttransplant
Time Frame: From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
Rate was based on cumulative incidence function estimate at 1 year. Time to first CMV-specific AVT was defined as time to the start of AVT for CMV viremia or CMV EOD. CMV-specific AVT and EOD were determined by the adjudication committee. This endpoint was a composite endpoint based on the independent adjudication committee assessments of CMV-specific AVT and CMV EOD.
From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
All-Cause Mortality at 1 Year Posttransplant
Time Frame: From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
All-cause mortality through 1-year post-transplantation summary included all deaths and unknown survival status. For the known deaths, the adjudication committee assessed results and summarized them according to the following category: Mortality due to the participant's primary disease, and mortality due to causes unrelated to the participant's primary disease. Participants with unknown survival status at 1 year were considered dead for this analysis.
From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Astellas Pharma Global Development, Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Medical Director, Astellas Pharma Global Development, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
September 11, 2013

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 28, 2017

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 1, 2022

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 12, 2013

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 12, 2013

First Posted (Estimated)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 14, 2013

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
October 24, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 15, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
October 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 0113-CL-1004
  • 2013-000903-18 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Access to anonymized individual participant level data collected during the study, in addition to study-related supporting documentation, is planned for studies conducted with approved product indications and formulations, as well as products terminated during development. Studies conducted with product indications or formulations that remain active in development are assessed after study completion to determine if Individual Participant Data can be shared. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.

IPD Sharing Time Frame

Access to participant level data is offered to researchers after publication of the primary manuscript (if applicable) and is available as long as Astellas has legal authority to provide the data.

IPD Sharing Access Criteria

Researchers must submit a proposal to conduct a scientifically relevant analysis of the study data. The research proposal is reviewed by an Independent Research Panel. If the proposal is approved, access to the study data is provided in a secure data sharing environment after receipt of a signed Data Sharing Agreement.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • CSR

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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