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

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Astellas Pharma Inc ( Astellas Pharma Global Development, Inc. ) Identifier:
First received: June 12, 2013
Last updated: October 4, 2016
Last verified: October 2016
The purpose of the study is 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 subjects undergoing allogeneic HCT will also be evaluated.

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

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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)

Further study details as provided by Astellas Pharma Inc:

Primary Outcome Measures:
  • Composite of overall mortality and cytomegalovirus (CMV) End Organ Disease (EOD) through one year post-transplant [ Time Frame: One year post-transplant ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Time to first protocol-defined CMV viremia [ Time Frame: One year post transplant ] [ Designated as safety issue: No ]
    CMV viremia defined as CMV plasma viral load ≥ 1000 IU/mL as assessed by the central laboratory

  • Time to first adjudicated CMV-specific anti-viral therapy (AVT) [ Time Frame: One year post-transplant ] [ Designated as safety issue: No ]
  • Time to first CMV-specific AVT for protocol-defined CMV viremia or CMV EOD [ Time Frame: One year post-transplant ] [ Designated as safety issue: No ]
    CMV viremia defined as CMV plasma viral load ≥ 1000 IU/mL as assessed by the central laboratory

Enrollment: 516
Study Start Date: July 2013
Estimated Study Completion Date: September 2022
Estimated Primary Completion Date: September 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ASP0113
ASP0113 arm
Biological: ASP0113
intramuscular injection
Placebo Comparator: Placebo
Placebo arm
Drug: Placebo
intramuscular injection


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Subject is a CMV-seropositive HCT recipient
  • Subject is planned to undergo either of the following:

    • Sibling Donor Transplant
    • Unrelated Donor Transplant
  • 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).
    • A defined myelodysplastic syndrome(s) (MDS)
    • Primary or secondary myelofibrosis
    • Lymphoma (including Hodgkin's)

Exclusion Criteria:

  • Subject has active CMV disease or infection or has received treatment for active CMV disease or infection within 3 months (90 days) prior to transplant
  • Subject has a modified hematopoietic cell transplant comorbidity index (HCT-CI) score ≥ 4
  • Subject has received a prior HCT and has residual Chronic Graft-versus-host Disease (cGVHD)
  • Subject who is scheduled to have a cord blood transplant or a haploidentical transplant
  • Subject has a platelet count of less than 50,000 mm3 within 3 days prior to randomization (platelet transfusions are allowed)
  • Subject has aplastic anemia or multiple myeloma
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01877655

  Show 84 Study Locations
Sponsors and Collaborators
Astellas Pharma Global Development, Inc.
Study Director: Medical Director Astellas Pharma Global Development, Inc.
  More Information

Responsible Party: Astellas Pharma Global Development, Inc. Identifier: NCT01877655     History of Changes
Other Study ID Numbers: 0113-CL-1004  2013-000903-18 
Study First Received: June 12, 2013
Last Updated: October 4, 2016
Health Authority: Germany: Paul-Ehrlich-Institut
Belgium: Federal Agency for Medicinal Products and Health Products
Canada: Health Canada
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Australia: Department of Health and Ageing Therapeutic Goods Administration
Taiwan : Food and Drug Administration
United States: Food and Drug Administration
Korea: Ministry of Food and Drug Safety
Japan: Pharmaceuticals and Medical Devices Agency
Sweden: Medical Products Agency
Spain: Agencia Española de Medicamentos y Productos Sanitarios
Individual Participant Data  
Plan to Share IPD: Undecided
Plan Description: An investigational test is being used to measure the cytomegalovirus (CMV) viral load in the plasma.

Keywords provided by Astellas Pharma Inc:
Cytomegalovirus (CMV)
Hematopoietic Cell Transplant (HCT)

Additional relevant MeSH terms:
Immunologic Factors
Physiological Effects of Drugs processed this record on December 02, 2016