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

Astellas

Status and phase: Completed; Phase 3

Conditions: Allogeneic, Hematopoietic Cell Transplant (HCT); Cytomegalovirus (CMV)-Positive Recipients

Treatments: Drug: Placebo; Biological: ASP0113

Study type: Interventional

Funder types: Industry

Identifiers

NCT01877655

0113-CL-1004

2013-000903-18 (EudraCT Number)

Details and patient eligibility

About

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.

Full description

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

Enrollment: 514 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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514 participants in 2 patient groups, including a placebo group

ASP0113

Experimental group

Description:

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).

Treatment:

Biological: ASP0113

Placebo

Placebo Comparator group

Description:

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).

Treatment:

Drug: Placebo