Clinical trial Recruiting at 53 sites Phase 3 Interventional Ages Any age to 17

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

Official title: 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)

Run by Takeda · NCT05319353 · Data updated 2026-11-12

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.

More Cytomegalovirus (CMV) trials

Quick facts

PhasePhase 3
Study typeInterventional
AgesAny age to 17 Years
SexAll
Enrollment80 (estimated)
SponsorTakeda
Industry-sponsored
Locations53 sites (Wilmington, Delaware and 52 other locations)
StatusRecruiting
StartedNovember 13, 2023
Main results expectedJanuary 18, 2027
Study endsJanuary 18, 2027
Registry record updatedMay 22, 2026
Trial IDNCT05319353 on ClinicalTrials.gov

Is this trial a fit for you?

Likely a 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.

Probably 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.

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

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 gone: 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.

Who can join: the full rules

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 it's running

  • Nemours Children's Health - Wilmington - PIN
    Wilmington, Delaware, United States
    Recruiting
  • Johns Hopkins All Children's Hospital - Main - PIN
    St. Petersburg, Florida, United States
    Recruiting
  • Ann and Robert H Lurie Childrens Hospital of Chicago - PIN
    Chicago, Illinois, United States
    Recruiting
  • University of Nebraska Medical Center -985400 Nebraska Medical Center
    Omaha, Nebraska, United States
    Recruiting
  • Cincinnati Children's Hospital Medical Center - PIN
    Cincinnati, Ohio, United States
    Recruiting
  • Cook Children's Health Care System
    Fort Worth, Texas, United States
    Recruiting
Show 44 more locations
  • University of Texas MD Anderson Cancer Center - 1515 Holcombe Blvd
    Houston, Texas, United States
    Recruiting
  • Texas Children's Hospital - Wallace Tower - PIN
    Houston, Texas, United States
    Recruiting
  • Minderoo Children's Comprehensive Cancer Centre
    Randwick, New South Wales, Australia
    Recruiting
  • Queensland Children's Hospital
    Woollangabba, Queensland, Australia
    Recruiting
  • Royal Children's Hospital Melbourne - PIN
    Parkville, Victoria, Australia
    Recruiting
  • Perth Children's Hospital
    Nedlands, Western Australia, Australia
    Recruiting
  • Hôpital Universitaire des Enfants Reine Fabiola (HUDERF)
    Brussels, Brussels Capital, Belgium
    Recruiting
  • Cliniques Universitaires Saint-Luc
    Woluwe-Saint-Lambert, Brussels Capital, Belgium
    Recruiting
  • UZ Gent
    Ghent, Oost-Vlaanderen, Belgium
    Recruiting
  • Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
    Porto Alegre, Rio Grande do Sul, Brazil
    Recruiting
  • Irmandade Da Santa Casa de Misericordia de Porto Alegre
    Porto Alegre, Rio Grande do Sul, Brazil
    Recruiting
  • Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
    Porto Alegre, Rio Grande do Sul, Brazil
    Recruiting
  • Hospital Do Rim E Hipertensão
    São Paulo, Brazil
    Recruiting
  • Capital Center For Children's Healthy, Capital Medical University
    Beijing, Beijing Municipality, China
    Recruiting
  • Beijing Children's Hospital, Capital Medical University - PIN
    Beijing, Beijing Municipality, China
    Recruiting
  • The First Affiliated Hospital, Sun Yat-sen University - Main
    Guangzhou, Guangdong, China
    Recruiting
  • Shenzhen Children's Hospital
    Shenzhen, Guangdong, China
    Recruiting
  • Shanghai Children's Medical Center - Zhangjiang Campus
    Shanghai, Shanghai Municipality, China
    Recruiting
  • Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences - PPDS
    Tianjin, Tianjin Municipality, China
    Recruiting
  • CHU de Rennes - Hôpital Pontchaillou
    Rennes, Ille-et-Vilaine, France
    Recruiting
  • CHU de Grenoble Alpes - Hôpital Michallon
    La Tronche, Isère, France
    Recruiting
  • CHRU Nantes
    Nantes, Loire-Atlantique, France
    Recruiting
  • Hopital Necker
    Paris, France
    Recruiting
  • Universitätsklinikum Würzburg
    Würzburg, Bavaria, Germany
    Recruiting
  • Universitätsklinikum Münster
    Münster, North Rhine-Westphalia, Germany
    Recruiting
  • Universitatsklinikum Jena - Am Klinikum 1-Erlanger Allee 101
    Jena, Thuringia, Germany
    Recruiting
  • Universitätsklinikum Hamburg Eppendorf
    Hamburg, Germany
    Recruiting
  • Medizinische Hochschule Hannover
    Hanover, Germany
    Recruiting
  • The Chaim Sheba Medical Center - PPDS
    Ramat Gan, Tel Aviv, Israel
    Recruiting
  • Tel Aviv Sourasky Medical Center Ichilov - PPDS
    Tel Aviv, Tel Aviv, Israel
    Recruiting
  • Rambam Health Care Campus - PPDS
    Haifa, Israel
    Recruiting
  • Hadassah Medical Center- Ein Kerem - PPDS
    Petah Tikva, Israel
    Recruiting
  • Saitama Prefectural Children's Medical Center
    Saitama-Shi Chuo-Ku, Saitama, Japan
    Recruiting
  • Shizuoka Children's Hospital
    Aoi-ku, Shizuoka, Japan
    Recruiting
  • National Center for Child Health and Development
    Setagaya-Ku, Tokyo, Japan
    Recruiting
  • Hyogo Prefectural Kobe Children's Hospital
    Chiba, Japan
    Recruiting
  • Osaka Women's and Children's Hospital
    Izumi-Shi, Ôsaka, Japan
    Recruiting
  • Hospital Sant Joan de Deu - PIN
    Espluges de Llobregat, Barcelona, Spain
    Recruiting
  • Hospital Universitario La Paz - PPDS
    Horcajo de la Sierra, Madrid, Spain
    Recruiting
  • Hospital Regional Universitario de Malaga - Hospital Materno-Infantil
    Málaga, Málaga, Spain
    Recruiting
  • Hospital Universitario Vall d´Hebron- PPDS
    Barcelona, Spain
    Recruiting
  • Hospital Infantil Universitario Niño Jesus - PIN
    Madrid, Spain
    Recruiting
  • King's College Hospital
    London, Lambeth, United Kingdom
    Recruiting
  • Royal Manchester Children's Hospital - PIN
    Manchester, Lancashire, United Kingdom
    Recruiting

+3 more sites. See ClinicalTrials.gov for the full list.

Study contacts

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

How to take part

  1. Review the eligibility criteria above with your treating physician.
  2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
  3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.
Last reviewed 2026-11-14 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.