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

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ClinicalTrials.gov Identifier: NCT01877655
Recruitment Status : Active, not recruiting
First Posted : June 14, 2013
Results First Posted : November 28, 2018
Last Update Posted : November 28, 2018
Sponsor:
Collaborator:
Vical
Information provided by (Responsible Party):
Astellas Pharma Inc ( Astellas Pharma Global Development, Inc. )

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.

Condition or disease Intervention/treatment Phase
Cytomegalovirus (CMV)-Positive Recipients Allogeneic, Hematopoietic Cell Transplant (HCT) Biological: ASP0113 Drug: Placebo Phase 3

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

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 514 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Prevention
Official Title: 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)
Actual Study Start Date : September 11, 2013
Actual Primary Completion Date : September 28, 2017
Estimated Study Completion Date : March 2020

Arm Intervention/treatment
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




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01877655


  Show 83 Study Locations
Sponsors and Collaborators
Astellas Pharma Global Development, Inc.
Vical
Investigators
Study Director: Medical Director Astellas Pharma Global Development, Inc.
  Study Documents (Full-Text)

Documents provided by Astellas Pharma Inc ( Astellas Pharma Global Development, Inc. ):
Study Protocol  [PDF] October 28, 2016
Statistical Analysis Plan  [PDF] May 19, 2017


Responsible Party: Astellas Pharma Global Development, Inc.
ClinicalTrials.gov Identifier: NCT01877655     History of Changes
Other Study ID Numbers: 0113-CL-1004
2013-000903-18 ( EudraCT Number )
First Posted: June 14, 2013    Key Record Dates
Results First Posted: November 28, 2018
Last Update Posted: November 28, 2018
Last Verified: September 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Access to anonymized individual participant level data collected during the trial, in addition to study-related supporting documentation, is planned for trials conducted with approved product indications and formulations, as well as compounds terminated during development. Conditions and exceptions are described under the Sponsor Specific Details for Astellas on www.clinicalstudydatarequest.com.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Clinical Study Report (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.
URL: https://www.clinicalstudydatarequest.com/

Keywords provided by Astellas Pharma Inc ( Astellas Pharma Global Development, Inc. ):
ASP0113
Cytomegalovirus (CMV)
Hematopoietic Cell Transplant (HCT)

Additional relevant MeSH terms:
Vaccines
Immunologic Factors
Physiological Effects of Drugs