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.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Ages | Any age to 17 Years |
| Sex | All |
| Enrollment | 80 (estimated) |
| Sponsor | Takeda Industry-sponsored |
| Locations | 53 sites (Wilmington, Delaware and 52 other locations) |
| Status | Recruiting |
| Started | November 13, 2023 |
| Main results expected | January 18, 2027 |
| Study ends | January 18, 2027 |
| Registry record updated | May 22, 2026 |
| Trial ID | NCT05319353 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
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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
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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
- Study coordinator: Takeda Contact
- Email: medinfoUS@takeda.com
- Phone: +1-877-825-3327
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.