Allogeneic, Hematopoietic Cell Transplant (HCT) Clinical Trial
— HELIOSOfficial 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)
| Verified date | December 2022 |
| Source | Astellas Pharma Inc |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Completed |
| Enrollment | 514 |
| Est. completion date | March 1, 2022 |
| Est. primary completion date | September 28, 2017 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years and older |
| Eligibility | 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 |
| Country | Name | City | State |
|---|---|---|---|
| Australia | Site AU43004 | Adelaide | South Australia |
| Australia | Site AU43001 | Herston | Queensland |
| Belgium | Site BE32001 | Brugge | |
| Belgium | Site BE32005 | Leuven | |
| Belgium | Site BE32007 | Roeselare | |
| Canada | Site CA15004 | Montreal | Quebec |
| Canada | Site CA15003 | Quebec | |
| Canada | Site CA15002 | Toronto | Ontario |
| Canada | Site CA15001 | Vancouver | British Columbia |
| France | Site FR33011 | Besancon | |
| France | Site FR33005 | Creteil | |
| France | Site FR33009 | Nantes | |
| France | Site FR33010 | Nice | |
| Germany | Site DE49010 | Bonn | |
| Germany | Site DE49012 | Dusseldorf | |
| Germany | Site DE49013 | Gottingen | |
| Germany | Site DE49014 | Koln | |
| Germany | Site DE49002 | Leipzig | |
| Germany | Site DE49015 | Mainz | |
| Germany | Site DE49007 | Muenster | |
| Germany | Site DE49005 | Stuttgart | |
| Germany | Site DE49008 | Tuebingen | |
| Germany | Site DE49006 | Ulm | |
| Germany | Site DE49001 | Wurzburg | |
| Japan | Site JP81010 | Bunkyo-ku | Tokyo |
| Japan | Site JP81008 | Chuo-ku | Tokyo |
| Japan | Site JP81001 | Fukuoka | |
| Japan | Site JP81005 | Fukuoka | |
| Japan | Site JP81011 | Maebashi | Gunma |
| Japan | Site JP81009 | Minato-ku | Tokyo |
| Japan | Site JP81003 | Nagoya | Aichi |
| Japan | Site JP81006 | Osaka | |
| Japan | Site JP81007 | Sapporo | Hokkaido |
| Japan | Site JP81004 | Shinjuku-ku | Tokyo |
| Korea, Republic of | Site KR82001 | Seoul | |
| Korea, Republic of | Site KR82002 | Seoul | |
| Korea, Republic of | Site KR82003 | Seoul | |
| Korea, Republic of | Site KR82004 | Seoul | Seoul Teugbyeolsi |
| Spain | Site ES34001 | Badalona | |
| Spain | Site ES34004 | Barcelona | |
| Spain | Site ES34006 | Barcelona | |
| Spain | Site ES34005 | Cordoba | |
| Spain | Site ES34003 | Granada | |
| Spain | Site ES34011 | Madrid | |
| Spain | Site ES34007 | Murcia | |
| Spain | Site ES34010 | Salamanca | |
| Spain | Site ES34002 | Santander | |
| Spain | Site ES34009 | Valencia | |
| Sweden | Site SE46001 | Gothenburg | |
| Sweden | Site SE46006 | Linkoping | |
| Sweden | Site SE46004 | Lund | |
| Sweden | Site SE46003 | Stockholm | |
| Sweden | Site SE46005 | Umea | |
| Taiwan | Site TW88601 | Taipei City | |
| Taiwan | Site TW88602 | Taipei City | |
| Taiwan | Site TW88603 | Taoyuan County | |
| United States | Site US10012 | Atlanta | Georgia |
| United States | Site US10011 | Baltimore | Maryland |
| United States | Site US10043 | Baltimore | Maryland |
| United States | Site US10028 | Birmingham | Alabama |
| United States | Site US10021 | Boston | Massachusetts |
| United States | Site US10025 | Chapel Hill | North Carolina |
| United States | Site US10013 | Chicago | Illinois |
| United States | Site US10002 | Dallas | Texas |
| United States | Site US10023 | Hackensack | New Jersey |
| United States | Site US10045 | Houston | Texas |
| United States | Site US10007 | Indianapolis | Indiana |
| United States | Site US10010 | Louisville | Kentucky |
| United States | Site US10019 | Milwaukee | Wisconsin |
| United States | Site US10016 | Nashville | Tennessee |
| United States | Site US10047 | New York | New York |
| United States | Site US10031 | Richmond | Virginia |
| United States | Site US10027 | Rochester | New York |
| United States | Site US10036 | Rochester | Minnesota |
| United States | Site US10042 | Saint Louis | Missouri |
| United States | Site US10046 | Salt Lake City | Utah |
| United States | Site US10035 | San Francisco | California |
| United States | Site US10024 | Seattle | Washington |
| United States | Site US10039 | Seattle | Washington |
| United States | Site US10026 | Stanford | California |
| United States | Site US10030 | Tampa | Florida |
| United States | Site US10044 | Tucson | Arizona |
| United States | Site US10020 | Westwood | Kansas |
| Lead Sponsor | Collaborator |
|---|---|
| Astellas Pharma Global Development, Inc. |
United States, Australia, Belgium, Canada, France, Germany, Japan, Korea, Republic of, Spain, Sweden, Taiwan,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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 | Percentage of Participants With Protocol-Defined CMV Viremia Through 1 Year Posttransplant | 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. | From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365) | |
| Secondary | Percentage of Participants With Adjudicated CMV-Specific Antiviral Therapy (AVT) Through 1 Year Posttransplant | 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. | From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365) | |
| Secondary | Percentage of Participants With a Composite Endpoint of Protocol-defined CMV Viremia and Adjudicated CMV-Specific AVT Use | 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. | From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365) | |
| Secondary | 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 | 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. | From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365) | |
| Secondary | All-Cause Mortality at 1 Year Posttransplant | 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. | From first study dose injection (Day -14 to -3 prior to transplant) up to one year post study drug injection (Day 365) |