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)

Overview
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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.

SEEK ID: https://cgnbonn.fdm.digital-medicine.org/projects/15

Public web page: Not specified

Organisms: No Organisms specified

NFDI4Health PIs: Cristoph Scheid

Trial Project start date: 9th Sep 2013

Trial Project end date: 3rd Jan 2022

Extended Metadata (Nfdi4Health MDS 3.3)

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Id of the project(*) : 29548

Resource classification(*)

Type of the resource : Study

Title(s)/name(s) of the Project

Title/name : 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)
Language of the title/name : English

Acronym(s) of the Project

Acronym : Astellas ASP0113 (HELIOS)
Language of the acronym : English

Description(s) of the Project

Description : 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.
Language of the description : English

Keyword(s) describing the Project

Keyword(*) : ASP0113

Keyword(*) : Cytomegalovirus (CMV)

Keyword(*) : Hematopoietic Cell Transplant (HCT)

Language(s) of the project : English

Web page of the project : https://www.clinicaltrials.astellas.com/study/0113-CL-1004/

Contributor(s) of the Project

Is it a personal or organisational contribution?(*) : Personal
Details about the contributing organisation(s)/institution(s)/group(s)
Contributor type(*) : Not specified
Funding identifier(s) : Not specified
Name of the organisation/institution/group(*) : Not specified
Details about the contributing person(s)
Contributor type(*) : Principal investigator
Given name(*) : Cristoph
Family name(s)(*) : Scheid
Digital identifier(s)
Identifier(*) : 0009-0007-6539-226X
Scheme(*) : ORCID
Email address : christoph.scheid@uk-koeln.de
Phone number : Not specified
Organisation(s) associated with the contributor
Name(*) : Klinik I für Innere Medizin, University Hopsital Cologne
Address : Kerpener Straße 62, 50937 Cologne, Germany
Web page : https://innere1.uk-koeln.de/
Digital identifier(s)
Identifier(*) : 05mxhda18
Scheme(*) : ROR

Is it a personal or organisational contribution?(*) : Personal
Details about the contributing organisation(s)/institution(s)/group(s)
Contributor type(*) : Not specified
Funding identifier(s) : Not specified
Name of the organisation/institution/group(*) : Not specified
Details about the contributing person(s)
Contributor type(*) : Contact
Given name(*) : Cristoph
Family name(s)(*) : Scheid
Digital identifier(s)
Identifier(*) : 0009-0007-6539-226X
Scheme(*) : ORCID
Email address : Not specified
Phone number : Not specified
Organisation(s) associated with the contributor
Name(*) : Klinik I für Innere Medizin, University Hopsital Cologne
Address : Kerpener Straße 62, 50937 Cologne, Germany
Web page : https://innere1.uk-koeln.de/
Digital identifier(s)
Identifier(*) : 05mxhda18
Scheme(*) : ROR
Identifier(*) : Not specified
Scheme(*) : Not specified
Name(*) : Not specified
Address : Not specified
Web page : Not specified
Digital identifier(s)
Identifier(*) : Not specified
Scheme(*) : Not specified
Identifier(*) : Not specified
Scheme(*) : Not specified

Is it a personal or organisational contribution?(*) : Organisational
Details about the contributing organisation(s)/institution(s)/group(s)
Contributor type(*) : Sponsor (primary)
Funding identifier(s) : Not specified
Name of the organisation/institution/group(*) : Astellas Pharma Inc
Details about the contributing person(s)
Contributor type(*) : Not specified
Given name(*) : Not specified
Family name(s)(*) : Not specified
Digital identifier(s)
Identifier(*) : Not specified
Scheme(*) : Not specified
Identifier(*) : Not specified
Scheme(*) : Not specified
Email address : Not specified
Phone number : Not specified
Organisation(s) associated with the contributor
Name(*) : Astellas Pharma Inc
Address : Not specified
Web page : https://www.astellas.com/en/
Digital identifier(s)
Identifier(*) : 01cjash87
Scheme(*) : ROR
Identifier(*) : Not specified
Scheme(*) : Not specified
Name(*) : Not specified
Address : Not specified
Web page : Not specified
Digital identifier(s)
Identifier(*) : Not specified
Scheme(*) : Not specified
Identifier(*) : Not specified
Scheme(*) : Not specified

Is it a personal or organisational contribution?(*) : Organisational
Details about the contributing organisation(s)/institution(s)/group(s)
Contributor type(*) : Contact
Funding identifier(s) : Not specified
Name of the organisation/institution/group(*) : Astellas Pharma Service Desk, Astellas Pharma Europe B.V.
Details about the contributing person(s)
Contributor type(*) : Not specified
Given name(*) : Not specified
Family name(s)(*) : Not specified
Digital identifier(s)
Identifier(*) : Not specified
Scheme(*) : Not specified
Identifier(*) : Not specified
Scheme(*) : Not specified
Email address : contact@nl.astellas.com
Phone number : 0031715455878
Organisation(s) associated with the contributor
Name(*) : Astellas Pharma Europe B.V.
Address : Sylviusweg 62, Leiden, 2333 BE, Netherlands
Web page : Not specified
Digital identifier(s)
Identifier(*) : 04kyfd050
Scheme(*) : ROR
Identifier(*) : Not specified
Scheme(*) : Not specified
Name(*) : Not specified
Address : Not specified
Web page : Not specified
Digital identifier(s)
Identifier(*) : Not specified
Scheme(*) : Not specified
Identifier(*) : Not specified
Scheme(*) : Not specified

Alternative identifiers

Type of the registry(*) : NCT (ClinicalTrials.gov)
Identifier(*) : NCT01877655

Type of the registry(*) : EudraCT
Identifier(*) : 2013-000903-18

Characteristics of the Project

Is it an interventional or non-interventional Project? : Interventional
Specification of the type of the Project
Interventional Project type : Parallel
Non-interventional Project type : []
Primary health condition(s) or disease(s) considered in the Project
Primary health condition or disease name(*) : Allogeneic, Hematopoietic Cell Transplant (HCT)
Terminology/classification(*) : Free text
Code of the health condition or disease : Not specified
Primary health condition or disease name(*) : Cytomegalovirus (CMV)-Positive Recipients
Terminology/classification(*) : Free text
Code of the health condition or disease : Not specified
Groups of diseases or conditions(*)
Which groups of diseases or conditions were the data collected on? : Neoplasms (II)
On which diseases or conditions were the data collected? : []
Collects mortality data? : Not specified
Administrative information about the Project
Status of the ethics committee approval : Request for approval submitted, approval granted
Overall Project status : Completed: Recruitment, data collection, and data quality management completed normally
Participants enrolled by invitation? : Not specified
Start date : 9 September 2013
End date : 3 January 2022
Mono- or multicentric? : Multicentric
Number of centers : 83
One or more data providers? : Not specified
Number of data providers : Not specified
Primary subject(*) : Person
Eligibility criteria for Project participants
Eligibility criteria: Minimum age
Minimum eligible age(*) : 18
Unit of age(*) : Years
Eligibility criteria: Maximum age
Maximum eligible age(*) : Not specified
Unit of age(*) : Not specified
Eligible gender : Male, Female, Diverse
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
Population of the Project(*)
Coverage : International
Country(ies) : Germany, France, United States
Region(s) and/or city(ies) : Not specified
Interventions of the Project
Name of the intervention(*) : Biological: ASP0113
Type of the intervention : Biological/Vaccine
Additional information about the intervention : 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).
Name(s) of the arm(s) associated with the given intervention : Experimental: ASP0113
Name of the intervention(*) : Drug: Placebo
Type of the intervention : Drug (including placebo)
Additional information about the intervention : 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).
Name(s) of the arm(s) associated with the given intervention : Placebo Comparator: Placebo
Exposures of the Project
Name of the exposure : Not specified
Type of the exposure : Not specified
Additional information about the exposure : Not specified
Name(s) of the group(s) associated with the given exposure : Not specified
Outcome measures in the Project
Name of the outcome measure(*) : Percentage of Participants With Composite of All-Cause Mortality and Adjudicated Cytomegalovirus End Organ Disease (CMV EOD) Through 1 Year Post Transplant
Description of the outcome measure : 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.
Type of the outcome measure : Primary
Time point(s) of assessment : From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
Name of the outcome measure(*) : Percentage of Participants With Protocol-Defined CMV Viremia Through 1 Year Posttransplant
Description of the outcome measure : 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.
Type of the outcome measure : Secondary
Time point(s) of assessment : From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
Name of the outcome measure(*) : Percentage of Participants With Adjudicated CMV-Specific Antiviral Therapy (AVT) Through 1 Year Posttransplant
Description of the outcome measure : 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.
Type of the outcome measure : Secondary
Time point(s) of assessment : From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
Name of the outcome measure(*) : Percentage of Participants With a Composite Endpoint of Protocol-defined CMV Viremia and Adjudicated CMV-Specific AVT Use
Description of the outcome measure : 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.
Type of the outcome measure : Secondary
Time point(s) of assessment : From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
Name of the outcome measure(*) : 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
Description of the outcome measure : 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.
Type of the outcome measure : Secondary
Time point(s) of assessment : From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
Name of the outcome measure(*) : All-Cause Mortality at 1 Year Posttransplant
Description of the outcome measure : 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.
Type of the outcome measure : Secondary
Time point(s) of assessment : From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
Additional information about the Project : Not specified
Additional assessments/measurements in the Project : []
Data sharing strategy of the Project(*)
Is it planned to share the data?(*) : Yes, there is a plan to make data available
Supporting documents available in addition to the data : Study protocol, Statistical analysis plan, Clinical study report
When and for how long will the data be available? : 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.
Criteria for data access : 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.
Additional information about data sharing : Not specified
DataSHIELD/Opal infrastructure available? : Not specified
Link to data request application : https://www.clinicaltrials.astellas.com/transparency/
Web page with additional information about data sharing : https://www.clinicaltrials.astellas.com/transparency/
Record linkage possible? : Not specified
Non-interventional aspects of the Project
Temporal design : []
Target follow-up duration of the Project
Target follow-up duration(*) : Not specified
Unit of time(*) : Not specified
Number of follow-ups conducted : Not specified
Biosamples retained in a biorepository : []
Specific types of retained biosamples : Not specified
Interventional aspects of the Project
Numerical phase : Phase-3
Masking of intervention(s) assignment
Masking implemented? : true
Who is masked? : Participant, Care provider, Investigator, Outcomes assessor
Additional information about masking : Not specified
Type of allocation of participants to an arm : Randomized
Off-label use of a drug product : Not specified

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)

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