Actively Recruiting
A Phase I Pharmacokinetic and Safety Assessment of Oral Letermovir in Infants With Symptomatic Congenital Cytomegalovirus Disease
Led by National Institute of Allergy and Infectious Diseases (NIAID) ยท Updated on 2026-05-22
12
Participants Needed
10
Research Sites
15 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating oral letermovir in newborns with symptomatic congenital Cytomegalovirus (CMV) disease in a Phase 1, open-label, single-arm study. The study aims to determine how the drug is processed in the body and assess its safety in infants. The trial involves two groups of neonates, focusing on letermovir exposure and viral suppression alongside standard treatment with valganciclovir. In this study, Group 1 of 4 infants will receive a single oral dose of letermovir, followed by detailed blood testing over 24 hours to measure drug levels. Depending on these results, they will start a 14-day course of daily oral letermovir. Group 2 of 8 infants will start the 14-day letermovir course concurrently with valganciclovir treatment. Dosages may be adjusted based on observed drug exposure. Throughout, infants will also receive valganciclovir as standard care. Participants will have blood draws on multiple study days to monitor drug levels, safety labs, and CMV viral loads in blood, saliva, and urine. Pharmacokinetic profiles will be collected on several days, including Day 10. Adverse events will be tracked during treatment and for four weeks after the last dose. After the study, infants will continue valganciclovir therapy for approximately six months as part of routine care. The primary measure is the drug's plasma exposure over 24 hours through Day 14.
CONDITIONS
Brief Title
A Safety Assessment of Oral Letermovir in Infants With Symptomatic Congenital Cytomegalovirus
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent from parent(s) or legal guardian(s)
- Confirmed Cytomegalovirus (CMV) infection by culture, shell vial, or PCR from saliva, blood, or urine obtained within 30 days of life
- Symptomatic congenital CMV disease, shown by at least one symptom such as thrombocytopenia, petechiae, hepatomegaly, splenomegaly, growth restriction, hepatitis, or CNS involvement
- Age at enrollment: up to 83 days for Group 1; up to 90 days for Group 2
- Weight between 2.6 kg and less than 8.0 kg at enrollment
- Born at or after 32 weeks gestation
- Plan by the patient's physician to treat infant with oral valganciclovir for 6 months for symptomatic congenital CMV disease
You will not qualify if you...
- Imminent demise
- Infant born to a woman known to be HIV positive
- Currently receiving other investigational drugs
- Grade 3 or 4 alanine aminotransferase (ALT) elevation per DAIDS Toxicity Table
- Grade 3 or 4 total bilirubin elevation per DAIDS Toxicity Table
- Gastrointestinal abnormalities that prevent oral medication absorption, such as necrotizing enterocolitis
- Expected use of carbamazepine, nafcillin, phenobarbital, or phenytoin during the study drug administration period
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - Up to 83 days for Group 1; up to 90 days for Group 2
Participants are screened for eligibility to participate in the trial.
1 screening and enrollment visit
Duration - 14 days
Participants receive oral letermovir with dosing adjusted based on pharmacokinetic results, alongside standard oral valganciclovir. Safety labs and viral load tests are conducted throughout treatment.
Visits on Study Days 0 (Group 1 only), 1, 5, 10, and 14 for dosing, blood draws, and safety assessments
Duration - 28 days post-treatment
Participants are monitored after treatment ends for adverse events and viral load rebound. They continue oral valganciclovir as routine care.
Visits on Study Days 21 and 42 for safety and viral load assessments
Trial Site Locations
Total: 10 locations
1
Children's of Alabama Child Health Research Unit (CHRU)
Birmingham, Alabama, United States, 35233-0011
Actively Recruiting
2
Children's National Medical Center - Sheikh Zayed Campus - Infectious Disease
Washington D.C., District of Columbia, United States, 20010-2916
Withdrawn
3
Emory University School of Medicine
Atlanta, Georgia, United States, 30322-1014
Actively Recruiting
4
University of Louisville School of Medicine - Norton Children's Hospital - Infectious Diseases
Louisville, Kentucky, United States, 40202
Withdrawn
5
Louisiana State University Health Shreveport - Infectious Diseases
Shreveport, Louisiana, United States, 71101
Withdrawn
6
University of Minnesota - Pediatric Infectious Disease
Minneapolis, Minnesota, United States, 55454
Withdrawn
7
SUNY Upstate Medical University Hospital - Pediatrics
Syracuse, New York, United States, 13210-2342
Withdrawn
8
Nationwide Children's Hosp.-Neonatology-Ctr. for Perinatal Rsrch.
Columbus, Ohio, United States, 43205-2664
Withdrawn
9
University of Texas Southwestern Medical Center - Pediatrics
Dallas, Texas, United States, 75390-9063
Withdrawn
10
Medical College of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
Research Team
D
David W. Kimberlin
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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