Clinical trial NCT01877655: A Study to Evaluate a Therapeutic Vaccine, ASP0113, in Cytomegalovirus (CMV)-Seropositive Recipients Undergoing Allogeneic, Hematopoietic Cell Transplant (HCT)
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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cytomegalovirus (CMV)-Positive Recipients
Allogeneic, Hematopoietic Cell Transplant (HCT)
Keywords: ASP0113
Cytomegalovirus (CMV)
Hematopoietic Cell Transplant (HCT)
Lead sponsor: Astellas Pharma Inc
Sponsor class: INDUSTRY
First posted: 2013-06-14
Enrollment: 514
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

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.

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)
  * 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

Primary outcomes:
measure: Percentage of Participants With Composite of All-Cause Mortality and Adjudicated Cytomegalovirus End Organ Disease (CMV EOD) Through 1 Year Post Transplant
timeFrame: From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
Secondary outcomes:
measure: Percentage of Participants With Protocol-Defined CMV Viremia Through 1 Year Posttransplant
timeFrame: From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
measure: Percentage of Participants With Adjudicated CMV-Specific Antiviral Therapy (AVT) Through 1 Year Posttransplant
timeFrame: From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
measure: Percentage of Participants With a Composite Endpoint of Protocol-defined CMV Viremia and Adjudicated CMV-Specific AVT Use
timeFrame: From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
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
timeFrame: From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
measure: All-Cause Mortality at 1 Year Posttransplant
timeFrame: From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365)
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Site US10028
state: Alabama
city: Tucson
country: United States
facility: Site US10044
state: Arizona
city: San Francisco
country: United States
facility: Site US10035
state: California
city: Stanford
country: United States
facility: Site US10026
state: California
city: Tampa
country: United States
facility: Site US10030
state: Florida
city: Atlanta
country: United States
facility: Site US10012
state: Georgia
city: Chicago
country: United States
facility: Site US10013
state: Illinois
city: Indianapolis
country: United States
facility: Site US10007
state: Indiana
city: Westwood
country: United States
facility: Site US10020
state: Kansas
city: Louisville
country: United States
facility: Site US10010
state: Kentucky
city: Baltimore
country: United States
facility: Site US10043
state: Maryland
city: Baltimore
country: United States
facility: Site US10011
state: Maryland
city: Boston
country: United States
facility: Site US10021
state: Massachusetts
city: Rochester
country: United States
facility: Site US10036
state: Minnesota
city: Saint Louis
country: United States
facility: Site US10042
state: Missouri
city: Hackensack
country: United States
facility: Site US10023
state: New Jersey
city: New York
country: United States
facility: Site US10047
state: New York
city: Rochester
country: United States
facility: Site US10027
state: New York
city: Chapel Hill
country: United States
facility: Site US10025
state: North Carolina
city: Nashville
country: United States
facility: Site US10016
state: Tennessee
city: Dallas
country: United States
facility: Site US10002
state: Texas
city: Houston
country: United States
facility: Site US10045
state: Texas
city: Salt Lake City
country: United States
facility: Site US10046
state: Utah
city: Richmond
country: United States
facility: Site US10031
state: Virginia
city: Seattle
country: United States
facility: Site US10039
state: Washington
city: Seattle
country: United States
facility: Site US10024
state: Washington
city: Milwaukee
country: United States
facility: Site US10019
state: Wisconsin
city: Herston
country: Australia
facility: Site AU43001
state: Queensland
city: Adelaide
country: Australia
facility: Site AU43004
state: South Australia
city: Brugge
country: Belgium
facility: Site BE32001
city: Leuven
country: Belgium
facility: Site BE32005
city: Roeselare
country: Belgium
facility: Site BE32007
city: Vancouver
country: Canada
facility: Site CA15001
state: British Columbia
city: Toronto
country: Canada
facility: Site CA15002
state: Ontario
city: Montreal
country: Canada
facility: Site CA15004
state: Quebec
city: Quebec
country: Canada
facility: Site CA15003
city: Besancon
country: France
facility: Site FR33011
city: Creteil
country: France
facility: Site FR33005
city: Nantes
country: France
facility: Site FR33009
city: Nice
country: France
facility: Site FR33010
city: Bonn
country: Germany
facility: Site DE49010
city: Dusseldorf
country: Germany
facility: Site DE49012
city: Gottingen
country: Germany
facility: Site DE49013
city: Koln
country: Germany
facility: Site DE49014
city: Leipzig
country: Germany
facility: Site DE49002
city: Mainz
country: Germany
facility: Site DE49015
city: Muenster
country: Germany
facility: Site DE49007
city: Stuttgart
country: Germany
facility: Site DE49005
city: Tuebingen
country: Germany
facility: Site DE49008
city: Ulm
country: Germany
facility: Site DE49006
city: Wurzburg
country: Germany
facility: Site DE49001
city: Nagoya
country: Japan
facility: Site JP81003
state: Aichi
city: Maebashi
country: Japan
facility: Site JP81011
state: Gunma
city: Sapporo
country: Japan
facility: Site JP81007
state: Hokkaido
city: Bunkyo-ku
country: Japan
facility: Site JP81010
state: Tokyo
city: Chuo-ku
country: Japan
facility: Site JP81008
state: Tokyo
city: Minato-ku
country: Japan
facility: Site JP81009
state: Tokyo
city: Shinjuku-ku
country: Japan
facility: Site JP81004
state: Tokyo
city: Fukuoka
country: Japan
facility: Site JP81001
city: Fukuoka
country: Japan
facility: Site JP81005
city: Osaka
country: Japan
facility: Site JP81006
city: Seoul
country: Korea, Republic of
facility: Site KR82004
state: Seoul Teugbyeolsi
city: Seoul
country: Korea, Republic of
facility: Site KR82002
city: Seoul
country: Korea, Republic of
facility: Site KR82003
city: Seoul
country: Korea, Republic of
facility: Site KR82001
city: Badalona
country: Spain
facility: Site ES34001
city: Barcelona
country: Spain
facility: Site ES34006
city: Barcelona
country: Spain
facility: Site ES34004
city: Cordoba
country: Spain
facility: Site ES34005
city: Granada
country: Spain
facility: Site ES34003
city: Madrid
country: Spain
facility: Site ES34011
city: Murcia
country: Spain
facility: Site ES34007
city: Salamanca
country: Spain
facility: Site ES34010
city: Santander
country: Spain
facility: Site ES34002
city: Valencia
country: Spain
facility: Site ES34009
city: Gothenburg
country: Sweden
facility: Site SE46001
city: Linkoping
country: Sweden
facility: Site SE46006
city: Lund
country: Sweden
facility: Site SE46004
city: Stockholm
country: Sweden
facility: Site SE46003
city: Umea
country: Sweden
facility: Site SE46005
city: Taipei City
country: Taiwan
facility: Site TW88601
city: Taipei City
country: Taiwan
facility: Site TW88602
city: Taoyuan County
country: Taiwan
facility: Site TW88603
Study officials:
affiliation: Astellas Pharma Global Development, Inc.
name: Medical Director
role: STUDY_DIRECTOR
References:
citation: 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.