A Study of the Drug Letermovir as Prevention of Cytomegalovirus Infection After Stem Cell Transplant in Pediatric Patients
Start Date
7/11/2024
Completion Date
6/30/2029
Summary
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.
Detailed Description
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.
Eligibility Criteria
Age Range: 2 years to 18 years
Interventions
Biospecimen Collection
Letermovir
Conditions
Locations
Children's Hospital of Alabama
Birmingham, Alabama 35233
United States
UCSF Benioff Children's Hospital Oakland
Oakland, California 94609
United States
UCSF Medical Center-Mission Bay
San Francisco, California 94158
United States
Children's Hospital Colorado
Aurora, Colorado 80045
United States
Alfred I duPont Hospital for Children
Wilmington, Delaware 19803
United States
Nemours Children's Clinic-Jacksonville
Jacksonville, Florida 32207
United States
Nicklaus Children's Hospital
Miami, Florida 33155
United States
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii 96826
United States
Riley Hospital for Children
Indianapolis, Indiana 46202
United States
University of Iowa/Holden Comprehensive Cancer Center
Iowa City, Iowa 52242
United States
Norton Children's Hospital
Louisville, Kentucky 40202
United States
Children's Hospital New Orleans
New Orleans, Louisiana 70118
United States
Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland 21287
United States
Children's Hospital of Michigan
Detroit, Michigan 48201
United States
Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital
Grand Rapids, Michigan 49503
United States
Children's Mercy Hospitals and Clinics
Kansas City, Missouri 64108
United States
Washington University School of Medicine
St Louis, Missouri 63110
United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma 73104
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United States
Saint Jude Children's Research Hospital
Memphis, Tennessee 38105
United States
The Children's Hospital at TriStar Centennial
Nashville, Tennessee 37203
United States
Vanderbilt University/Ingram Cancer Center
Nashville, Tennessee 37232
United States
Medical City Dallas Hospital
Dallas, Texas 75230
United States
UT Southwestern/Simmons Cancer Center-Dallas
Dallas, Texas 75390
United States
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
Houston, Texas 77030
United States
Methodist Children's Hospital of South Texas
San Antonio, Texas 78229
United States
Primary Children's Hospital
Salt Lake City, Utah 84113
United States
VCU Massey Comprehensive Cancer Center
Richmond, Virginia 23298
United States
University of Wisconsin Carbone Cancer Center - University Hospital
Madison, Wisconsin 53792
United States