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Maribavir treatment for CMV infection in children and adolescents after stem cell or organ transplant

A Phase 3, Open-label, Single-arm, Repeated-dose Study to Evaluate the Safety and Tolerability, Pharmacokinetics, and Antiviral Activity of Maribavir for the Treatment of Cytomegalovirus (CMV) Infection in Children and Adolescents Who Have Received a Hematopoietic Stem Cell Transplant (HSCT) or a Solid Organ Transplant (SOT)

Phase 3 Interventional Takeda · NCT05319353

This study tests whether maribavir is safe and works to treat CMV infection in children and teenagers who have had a stem cell or solid organ transplant.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment80 (estimated)
AgesN/A to 17 Years
SexAll
SponsorTakeda Industry-sponsored
Locations47 sites (Omaha, Nebraska and 46 other locations)
Trial IDNCT05319353 on ClinicalTrials.gov

What this trial studies

Children and adolescents under 18 who have CMV infection after a hematopoietic stem cell transplant (HSCT) or a solid organ transplant (SOT) will receive maribavir using either a 200 mg tablet or a powder for oral suspension for 8 weeks. The trial will collect safety and tolerability data, measure drug levels (pharmacokinetics), and monitor antiviral activity by measuring CMV DNA using local quantitative PCR. Participants are enrolled in age-based cohorts, including a cohort for 0 to <6 years with specific gestational age and weight requirements, to identify optimal pediatric dosing. All participants will have a 12-week follow-up period with clinic visits to track sustained response and any adverse events.

Who should consider this trial

Good fit: Ideal candidates are children and adolescents under 18 with a functioning HSCT or SOT and documented CMV viremia at or above the study's required viral load thresholds.

Not a fit: Patients without transplant-related CMV viremia, with viral loads below the entry criteria, or with contraindications to maribavir are unlikely to receive benefit from this study.

Why it matters

Potential benefit: If successful, maribavir could offer a well-tolerated, effective antiviral option and an evidence-based pediatric dosing regimen for post-transplant CMV.

How similar studies have performed: Maribavir has demonstrated efficacy in adults with refractory or resistant CMV in prior trials and is approved for adult use, but pediatric experience is limited.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Parent/both parents or legally authorized representative (LAR) must provide signature of informed consent and there must be documentation of assent by the participant, as age appropriate, before completing any study-related procedures.
* Be a male or female child or adolescent \< 18 years of age at the time of consent. For participants in Cohort 3 only (0 to \<6 years) must have a gestational age of at least 39 weeks and a minimum weight of 5 kg.
* Be a recipient of an SOT or an HSCT that is functioning at the time of screening.
* Have a documented CMV infection which may be a first episode of post-transplant CMV viremia (primary or reactivation) or refractory to other anti-CMV treatments, with a CMV DNA screening value of \>= 1365 International Units per milliliter (IU/mL) in whole blood or \>= 455 IU/mL in plasma in 2 consecutive assessments separated by at least 1 day, as determined by local laboratory quantitative polymerase chain reaction (qPCR) or comparable quantitative nucleic acid amplification test (qNAAT) results. Quantitative assays must be standardized to the World Health Organization (WHO) CMV International Standard. Both samples must be taken within 14 days of first dose of study drug, with the second sample obtained within 5 days prior to first dose of study drug. The same laboratory and same sample type (whole blood or plasma) must be used for both assessments. If documented and verified values are available in medical history that fulfill this criterion entirely, they may be used instead.
* Have all the following results as part of screening laboratory assessments:
* Absolute neutrophil count \>= 500 per cubic millimeter (/mm\^3) (0.5 × 10\^9 per liter \[/L\])
* Platelet count \>= 15,000/mm\^3 (15 × 10\^9/L)
* Hemoglobin \>= 8 grams per deciliter (g/dL) (\>=80 grams per liter \[g/L\]).
* Have an estimated glomerular filtration rate (creatinine-based Bedside Schwartz equation) \>= 30 milliliters per minute (mL/min) /1.73 meter square (m\^2).
* Be a female of nonchildbearing potential. If a female of childbearing potential, have a negative serum human chorionic gonadotropin (hCG) or beta-human chorionic gonadotropin (β-hCG) pregnancy test at screening. Males, or nonpregnant, nonlactating females who are sexually active must agree to comply with the applicable contraceptive requirements of this protocol during the study treatment administration period and for 90 days after the last dose of study treatment.
* Have life expectancy of \>= 8 weeks.
* Be willing and have an understanding and ability to fully comply with the study procedures and restrictions defined in the protocol. For younger children, the parent/both parents or LAR must meet this criterion.
* Participants must have a confirmed negative human immunodeficiency virus (HIV) test result within 3 months of first dose of study drug or, if unavailable, be tested by a local laboratory during the screening period.

Exclusion Criteria:

* Have CMV tissue invasive disease involving the central nervous system (CNS) or retina as assessed by the investigator at the time of screening.
* Have uncontrolled other type of infection as assessed by the investigator on the date of enrollment.
* Have a history of clinically relevant alcohol or drug abuse that may interfere with treatment compliance or assessments with the protocol as determined by the investigator.
* Be receiving valganciclovir, ganciclovir, cidofovir, foscarnet, leflunomide, letermovir, or artesunate when study treatment is initiated, or anticipated to require one of these agents during the 8-week treatment period.
* Have a known hypersensitivity to maribavir or to any excipients.
* Have severe vomiting, diarrhea, or other severe gastrointestinal (GI) illness within 24 hours prior to the first dose of study treatment or a GI absorption abnormality that would preclude administration of oral medication.
* Require mechanical ventilation or vasopressors for hemodynamic support at baseline (Visit 2/Day 1/Week 0).
* Be pregnant (or expecting to conceive) or nursing.
* Have previously completed, discontinued, or have been withdrawn from this study.
* Have received any investigational agent or device within 30 days before initiation of study treatment (includes CMV specific T-cells) or plan to receive an investigational agent or device during the study.

Previously approved agents under investigation for additional indications are not exclusionary.

* Have previously received maribavir or CMV vaccine at any time.
* Have any clinically significant medical or surgical condition that, in the investigator's opinion, could interfere with interpretation of study results, contraindicate the administration of the study treatment, or compromise the safety or well-being of the participant.
* Have severe liver disease (Child-Pugh score of \>= 10).
* Have serum aspartate aminotransferase greater than (\>) 5 times upper limit of normal (ULN) at screening, or serum alanine aminotransferase \> 5 times ULN at screening, or total bilirubin \>= 3.0 times ULN at screening (except for documented Gilbert's syndrome), as analyzed by local laboratory.
* Have positive results for HIV.
* Have active malignancy with the exception of nonmelanoma skin cancer, as determined by the investigator. Participants who experience relapse or progression of their underlying malignancy (for which HSCT or SOT was performed), as determined by the investigator, are not to be enrolled.
* Be undergoing treatment for acute or chronic hepatitis B or hepatitis C.
* Requiring ongoing treatment with or an anticipated need for treatment with a strong cytochrome P450 3A (CYP3A) inducer.
* Have a low body weight where total blood volume (TBV) required during study participation will exceed 1 percent (%) TBV per study visit or 3% TBV over a 4-week period.

Where this trial is running

Omaha, Nebraska and 46 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Cytomegalovirus
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.