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
- Phase
- Phase 3
- Intervention
- ASP0113
- Conditions
- Cytomegalovirus (CMV)-Positive Recipients
- Sponsor
- Astellas Pharma Global Development, Inc.
- Enrollment
- 514
- Locations
- 83
- Primary Endpoint
- Percentage of Participants With Composite of All-Cause Mortality and Adjudicated Cytomegalovirus End Organ Disease (CMV EOD) Through 1 Year Post Transplant
- Status
- Completed
- Last Updated
- last year
Overview
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.
Detailed Description
Participants will be followed for 5.5 years post-transplant for long-term safety via an annual telephone contact.
Investigators
Eligibility 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)
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
Arms & Interventions
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).
Intervention: ASP0113
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).
Intervention: Placebo
Outcomes
Primary Outcomes
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 Outcomes
- 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))