A Study to Evaluate a Therapeutic Vaccine, ASP0113, in Cytomegalovirus (CMV)-Seropositive Recipients Undergoing Allogeneic, Hematopoietic Cell Transplant (HCT)
HELIOS
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)
2 other identifiers
interventional
514
11 countries
83
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_3
Started Sep 2013
Longer than P75 for phase_3
83 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 12, 2013
CompletedFirst Posted
Study publicly available on registry
June 14, 2013
CompletedStudy Start
First participant enrolled
September 11, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 28, 2017
CompletedResults Posted
Study results publicly available
November 28, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2022
CompletedOctober 24, 2024
October 1, 2024
4 years
June 12, 2013
September 17, 2018
October 15, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Percentage of Participants With Composite of All-Cause Mortality and Adjudicated Cytomegalovirus End Organ Disease (CMV EOD) Through 1 Year Post Transplant
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 Outcomes (5)
Percentage of Participants With Protocol-Defined CMV Viremia Through 1 Year Posttransplant
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
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
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
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
From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
Study Arms (2)
ASP0113
EXPERIMENTALParticipants 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).
Placebo
PLACEBO COMPARATORParticipants 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).
Interventions
Eligibility Criteria
You may qualify if:
- 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)
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (83)
Site US10028
Birmingham, Alabama, 35294, United States
Site US10044
Tucson, Arizona, 85724, United States
Site US10035
San Francisco, California, 94143, United States
Site US10026
Stanford, California, 94305, United States
Site US10030
Tampa, Florida, 33612, United States
Site US10012
Atlanta, Georgia, 30322, United States
Site US10013
Chicago, Illinois, 60612, United States
Site US10007
Indianapolis, Indiana, 46237, United States
Site US10020
Westwood, Kansas, 66205, United States
Site US10010
Louisville, Kentucky, 40202, United States
Site US10043
Baltimore, Maryland, 21201, United States
Site US10011
Baltimore, Maryland, 21205, United States
Site US10021
Boston, Massachusetts, 02114, United States
Site US10036
Rochester, Minnesota, 55905, United States
Site US10042
St Louis, Missouri, 63110, United States
Site US10023
Hackensack, New Jersey, 07601-2105, United States
Site US10047
New York, New York, 10032, United States
Site US10027
Rochester, New York, 14642, United States
Site US10025
Chapel Hill, North Carolina, 27514, United States
Site US10016
Nashville, Tennessee, 37232, United States
Site US10002
Dallas, Texas, 75246, United States
Site US10045
Houston, Texas, 77030, United States
Site US10046
Salt Lake City, Utah, 84143, United States
Site US10031
Richmond, Virginia, 23298, United States
Site US10039
Seattle, Washington, 98108, United States
Site US10024
Seattle, Washington, 98109, United States
Site US10019
Milwaukee, Wisconsin, 53226, United States
Site AU43001
Herston, Queensland, QLD 4029, Australia
Site AU43004
Adelaide, South Australia, SA 5000, Australia
Site BE32001
Bruges, 8000, Belgium
Site BE32005
Leuven, 3000, Belgium
Site BE32007
Roeselare, 8800, Belgium
Site CA15001
Vancouver, British Columbia, V5Z 1M9, Canada
Site CA15002
Toronto, Ontario, M5G 2M9, Canada
Site CA15004
Montreal, Quebec, H2W 1S6, Canada
Site CA15003
Québec, Quebec, G1R 2J6, Canada
Site FR33011
Besançon, 25030, France
Site FR33005
Créteil, 94000, France
Site FR33009
Nantes, 44093, France
Site FR33010
Nice, 06200, France
Site DE49010
Bonn, 53127, Germany
Site DE49014
Cologne, 50937, Germany
Site DE49012
Düsseldorf, 40225, Germany
Site DE49013
Göttingen, 37075, Germany
Site DE49002
Leipzig, 04103, Germany
Site DE49015
Mainz, 55131, Germany
Site DE49007
Münster, 48149, Germany
Site DE49005
Stuttgart, 70376, Germany
Site DE49008
Tübingen, 72076, Germany
Site DE49006
Ulm, 89081, Germany
Site DE49001
Würzburg, 97080, Germany
Site JP81003
Nagoya, Aichi-ken, Japan
Site JP81011
Maebashi, Gunma, Japan
Site JP81007
Sapporo, Hokkaido, Japan
Site JP81010
Bunkyo-ku, Tokyo, Japan
Site JP81008
Chuo-ku, Tokyo, Japan
Site JP81009
Minato-ku, Tokyo, Japan
Site JP81004
Shinjuku-ku, Tokyo, Japan
Site JP81001
Fukuoka, Japan
Site JP81005
Fukuoka, Japan
Site JP81006
Osaka, Japan
Site KR82004
Seoul, Seoul Teugbyeolsi, 06351, South Korea
Site KR82002
Seoul, 05505, South Korea
Site KR82003
Seoul, 110744, South Korea
Site KR82001
Seoul, 137-701, South Korea
Site ES34001
Badalona, 08615, Spain
Site ES34006
Barcelona, 08041, Spain
Site ES34004
Barcelona, 08908, Spain
Site ES34005
Córdoba, 14004, Spain
Site ES34003
Granada, 18012, Spain
Site ES34011
Madrid, 28028, Spain
Site ES34007
Murcia, 30008, Spain
Site ES34010
Salamanca, 37007, Spain
Site ES34002
Santander, 39008, Spain
Site ES34009
Valencia, 46010, Spain
Site SE46001
Gothenburg, 413 45, Sweden
Site SE46006
Linköping, 581 85, Sweden
Site SE46004
Lund, 221 85, Sweden
Site SE46003
Stockholm, 14186, Sweden
Site SE46005
Umeå, 901 85, Sweden
Site TW88601
Taipei, 10002, Taiwan
Site TW88602
Taipei, 11217, Taiwan
Site TW88603
Taoyuan, 33305, Taiwan
Related Publications (1)
Ljungman P, Bermudez A, Logan AC, Kharfan-Dabaja MA, Chevallier P, Martino R, Wulf G, Selleslag D, Kakihana K, Langston A, Lee DG, Solano C, Okamoto S, Smith LR, Boeckh M, Wingard JR, Cywin B, Fredericks C, Lademacher C, Wang X, Young J, Maertens J. A randomised, placebo-controlled phase 3 study to evaluate the efficacy and safety of ASP0113, a DNA-based CMV vaccine, in seropositive allogeneic haematopoietic cell transplant recipients. EClinicalMedicine. 2021 Mar 19;33:100787. doi: 10.1016/j.eclinm.2021.100787. eCollection 2021 Mar.
PMID: 33842870DERIVED
Related Links
Limitations and Caveats
In Oct 2014 data monitoring committee informed Astellas of the results of the first futility analysis. The FDA considered data for 68 participants included in futility analysis compromised and asked that it is not included in final efficacy analysis.
Results Point of Contact
- Title
- Clinical Trial Disclosure
- Organization
- Astellas Pharma Global Development, Inc.
Study Officials
- STUDY DIRECTOR
Medical Director
Astellas Pharma Global Development, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- PREVENTION
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 12, 2013
First Posted
June 14, 2013
Study Start
September 11, 2013
Primary Completion
September 28, 2017
Study Completion
March 1, 2022
Last Updated
October 24, 2024
Results First Posted
November 28, 2018
Record last verified: 2024-10
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, CSR
- 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.
- 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.
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/.