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NCT05319353PHASE3Recruiting

A 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)

Takeda

Start Date

11/13/2023

Completion Date

1/18/2027

Summary

The main aim of this study is to find out the safety, tolerability and pharmacokinetics (PK) of maribavir for the treatment of CMV infection in children and teenagers after HSCT or SOT and to identify the optimal dose of maribavir using a 200 milligrams (mg) tablet formulation or powder for oral suspension. The participants will be treated with maribavir for 8 weeks. Participants need to visit their doctor during 12-week follow-up period.

Eligibility Criteria

Age Range: No minimum to 17 years

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.

Interventions

DRUG

Maribavir

Interested in This Trial?

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Conditions

Cytomegalovirus (CMV)

Locations

Nemours Children's Health - Wilmington - PIN

Wilmington, Delaware 19803-3607

United States

Johns Hopkins All Children's Hospital - Main - PIN

St. Petersburg, Florida 33701

United States

Ann and Robert H Lurie Childrens Hospital of Chicago - PIN

Chicago, Illinois 60611

United States

University of Nebraska Medical Center -985400 Nebraska Medical Center

Omaha, Nebraska 68114-4113

United States

Cincinnati Children's Hospital Medical Center - PIN

Cincinnati, Ohio 45229-3026

United States

Cook Children's Health Care System

Fort Worth, Texas 76104-2733

United States

University of Texas MD Anderson Cancer Center - 1515 Holcombe Blvd

Houston, Texas 77030-4000

United States

Texas Children's Hospital - Wallace Tower - PIN

Houston, Texas 77030

United States

Minderoo Children's Comprehensive Cancer Centre

Randwick, New South Wales 2031

Australia

Queensland Children's Hospital

Woollangabba, Queensland 4101

Australia

Royal Children's Hospital Melbourne - PIN

Parkville, Victoria 3052

Australia

Perth Children's Hospital

Nedlands, Western Australia 6009

Australia

Hôpital Universitaire des Enfants Reine Fabiola (HUDERF)

Brussels, Brussels Capital 1020

Belgium

Cliniques Universitaires Saint-Luc

Woluwe-Saint-Lambert, Brussels Capital 1200

Belgium

UZ Gent

Ghent, Oost-Vlaanderen 9000

Belgium

Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo

Porto Alegre, Rio Grande do Sul 05403-000

Brazil

Irmandade Da Santa Casa de Misericordia de Porto Alegre

Porto Alegre, Rio Grande do Sul 90020-090

Brazil

Hospital de Clinicas de Porto Alegre (HCPA) - PPDS

Porto Alegre, Rio Grande do Sul 90035-903

Brazil

Hospital Do Rim E Hipertensão

São Paulo, 04038-002

Brazil

Capital Center For Children's Healthy, Capital Medical University

Beijing, Beijing Municipality 100020

China

Beijing Children's Hospital, Capital Medical University - PIN

Beijing, Beijing Municipality 100045

China

The First Affiliated Hospital, Sun Yat-sen University - Main

Guangzhou, Guangdong 510080

China

Shenzhen Children's Hospital

Shenzhen, Guangdong 518038

China

Shanghai Children's Medical Center - Zhangjiang Campus

Shanghai, Shanghai Municipality 200127

China

Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences - PPDS

Tianjin, Tianjin Municipality 300020

China

CHU de Rennes - Hôpital Pontchaillou

Rennes, Ille-et-Vilaine 35200

France

CHU de Grenoble Alpes - Hôpital Michallon

La Tronche, Isère 38700

France

CHRU Nantes

Nantes, Loire-Atlantique 44000

France

Hopital Necker

Paris, 75015

France

Universitätsklinikum Würzburg

Würzburg, Bavaria 97080

Germany

Universitätsklinikum Münster

Münster, North Rhine-Westphalia 48149

Germany

Universitatsklinikum Jena - Am Klinikum 1-Erlanger Allee 101

Jena, Thuringia 07745

Germany

Universitätsklinikum Hamburg Eppendorf

Hamburg, 20246

Germany

Medizinische Hochschule Hannover

Hanover, 30625

Germany

The Chaim Sheba Medical Center - PPDS

Ramat Gan, Tel Aviv 52394

Israel

Tel Aviv Sourasky Medical Center Ichilov - PPDS

Tel Aviv, Tel Aviv 64239

Israel

Rambam Health Care Campus - PPDS

Haifa, 31096

Israel

Hadassah Medical Center- Ein Kerem - PPDS

Petah Tikva, 4920235

Israel

Saitama Prefectural Children's Medical Center

Saitama-Shi Chuo-Ku, Saitama 330-8777

Japan

Shizuoka Children's Hospital

Aoi-ku, Shizuoka 420-8660

Japan

National Center for Child Health and Development

Setagaya-Ku, Tokyo 157-8535

Japan

Hyogo Prefectural Kobe Children's Hospital

Chiba, 650-0047

Japan

Osaka Women's and Children's Hospital

Izumi-Shi, Ôsaka 594-1101

Japan

Hospital Sant Joan de Deu - PIN

Espluges de Llobregat, Barcelona 08950

Spain

Hospital Universitario La Paz - PPDS

Horcajo de la Sierra, Madrid 28755

Spain

Hospital Regional Universitario de Malaga - Hospital Materno-Infantil

Málaga, Málaga 29011

Spain

Hospital Universitario Vall d´Hebron- PPDS

Barcelona, 08035

Spain

Hospital Infantil Universitario Niño Jesus - PIN

Madrid, 28009

Spain

King's College Hospital

London, Lambeth SE5 9RS

United Kingdom

Royal Manchester Children's Hospital - PIN

Manchester, Lancashire M13 9WL

United Kingdom

Nottingham University Hospitals NHS Trust

Nottingham, Nottinghamshire NG5 1PB

United Kingdom

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, West Midlands B4 6NH

United Kingdom

Great Ormond Street Hospital

London, WC1N 3JH

United Kingdom