This phase III single arm trial determines whether taking prophylactic letermovir will reduce the likelihood of infection with cytomegalovirus (CMV) in children and adolescents after stem cell transplant compared to estimated rate of infection without prophylaxis. The treatments used to prepare for HCT reduce the body's natural infection-fighting ability and increase the likelihood of an infection with a virus called cytomegalovirus. "Prophylaxis" means to take a drug to prevent a disease or side effect. Letermovir is an antiviral drug that stops cytomegalovirus from multiplying and may prevent cytomegalovirus infection and make the disease less severe.
PRIMARY OBJECTIVE: I. To evaluate the efficacy of letermovir prophylaxis in the prevention of clinically significant CMV infection through Week 14 (\~100 days) post-transplant in children and adolescents receiving allogeneic hematopoietic cell transplant (allo-HCT). SECONDARY OBJECTIVE: I. To evaluate the efficacy of letermovir prophylaxis as assessed by CMV-free survival through 24 weeks (\~6 months) post-transplant in pediatric patients. EXPLORATORY OBJECTIVES: I. To evaluate the incidence of clinically significant CMV infection through 24 and 52 weeks post-transplant in patients who receive letermovir prophylaxis. II. To evaluate overall survival post-transplant in patients who receive letermovir prophylaxis. III. To evaluate time to engraftment and describe the cumulative incidence of non-engraftment among patients who receive letermovir. IV. To examine the following clinically significant adverse events among patients exposed to letermovir: the total duration of neutropenia through week 14 (\~100 days) post-transplant, the cumulative incidence of acute kidney injury and chronic kidney disease by 52 weeks post-transplant, and total inpatient hospital days by 14 weeks (\~100 days) and 52 weeks post-transplant. V. Describe patterns of anti-viral resistance at the onset of CMV DNAemia after allo-HCT among patients who receive letermovir prophylaxis. VI. To describe immune reconstitution and CMV-specific immunity among patients who receive letermovir prophylaxis. OUTLINE: Enrolled patients will be added to the single arm of the study and receive letermovir prophylaxis. ARM A: Patients receive letermovir orally (PO) or intravenously (IV) over 60 minutes once daily (QD) starting on day +1 post-transplant for 14 weeks. Patients undergo collection of blood samples for CMV polymerase chain reaction (PCR) analysis weekly for 14 weeks, every 2 weeks until week 24, week 32, week 40 and week 52. ARM B (CLOSED TO ACCRUAL 09/29/2025): Patients undergo collection of blood samples for CMV PCR analysis weekly for 14 weeks, every 2 weeks until week 24, week 32, week 40 and week 52.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
PREVENTION
Masking
SINGLE
Enrollment
105
Undergo collection of blood samples
Given PO or IV
Children's Hospital of Alabama
Birmingham, Alabama, United States
RECRUITINGUCSF Benioff Children's Hospital Oakland
Oakland, California, United States
RECRUITINGUCSF Medical Center-Mission Bay
San Francisco, California, United States
RECRUITINGAlfred I duPont Hospital for Children
Wilmington, Delaware, United States
RECRUITINGNemours Children's Clinic-Jacksonville
Jacksonville, Florida, United States
RECRUITINGUniversity of Iowa/Holden Comprehensive Cancer Center
Iowa City, Iowa, United States
RECRUITINGNorton Children's Hospital
Louisville, Kentucky, United States
RECRUITINGChildren's Hospital New Orleans
New Orleans, Louisiana, United States
RECRUITINGJohns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland, United States
RECRUITINGChildren's Hospital of Michigan
Detroit, Michigan, United States
RECRUITING...and 12 more locations
Clinically significant cytomegalovirus (CMV) infection
Clinically significant CMV is defined as the first of (1) initiation of anti-CMV preemptive therapy for documented CMV DNAemia or (2) onset of CMV end-organ disease. Will estimate the cumulative incidence of clinically significant CMV at 14-weeks post-transplant and will report the corresponding 95% confidence interval.
Time frame: Up to week 14 post-transplant
Detection of CMV DNAemia
Will estimate the cumulative incidence of CMV DNAemia at 14-weeks post-transplant by study arm and will report the corresponding 95% confidence intervals.
Time frame: Up to week 14 post-transplant
CMV-free survival
Will estimate the cumulative incidence of the occurrence of CMV DNAemia or death by week 24 post-transplant by study arm and will report the corresponding 95% confidence intervals.
Time frame: Up to 24 weeks post-transplant
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