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)

NCT05319353 | Recruiting Phase 3 DMC FDA Drug

• 4 other identifiers: TAK-620-20042021-004279-15jRCT20312307532023-508988-73-00
• Study Type: interventional
• Target: 80
• Locations: 11 countries
• Sites: 47

Brief 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.

Conditions

Cytomegalovirus (CMV)

Outcome Measures

Primary Outcomes (9)

• Maximum Observed Plasma Concentration (Cmax) of Maribavir

  Cmax of maribavir will be evaluated.

  Pre-dose; 0.5, 1.5, 3, 4, 6, and 8 hours post-dose on Day 7 (Week 1)

• Time to Maximum Observed Concentration (Tmax) of Maribavir

  Tmax of maribavir will be evaluated.

  Pre-dose; 0.5, 1.5, 3, 4, 6, and 8 hours post-dose on Day 7 (Week 1)

• Minimum Plasma Concentration (Cmin) of Maribavir

  Cmin of maribavir will be evaluated.

  Pre-dose; (0.5, 1.5, 3, 4, 6, and 8 hours post-dose) on Day 7 (Week 1); Pre-dose on Day 28 (Week 4); Pre-dose; (2 to 4 hours post-dose) on Day 56 (Week 8)

• Area Under the Plasma Concentration-Time Curve Over the 1 Dosing Interval of 12 Hours at Steady State (AUC0-tau) of Maribavir

  AUC0-tau of maribavir will be evaluated.

  Pre-dose; 0.5, 1.5, 3, 4, 6, and 8 hours post-dose on Day 7 (Week 1)

• Half-Life (t1/2) of Maribavir

  t1/2 of maribavir will be evaluated.

  Pre-dose; 0.5, 1.5, 3, 4, 6, and 8 hours post-dose on Day 7 (Week 1)

• Terminal Elimination Rate Constant (lambdaz) of Maribavir

  Lambdaz of maribavir will be evaluated.

  Pre-dose; 0.5, 1.5, 3, 4, 6, and 8 hours post-dose on Day 7 (Week 1)

• Apparent Volume of Distribution (Vz/F) of Maribavir

  Vz/F of maribavir will be evaluated.

  Pre-dose; 0.5, 1.5, 3, 4, 6, and 8 hours post-dose on Day 7 (Week 1)

• Apparent Oral Clearance (CL/F) of Maribavir

  CL/F of maribavir will be evaluated.

  Pre-dose; 0.5, 1.5, 3, 4, 6, and 8 hours post-dose on Day 7 (Week 1)

• Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

  An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily be considered related to investigational product. SAE is any untoward medical occurrence (whether considered related to investigational product or not) that at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital abnormality or birth defect, or is an important medical event.

  From start of study drug administration up to follow-up (Week 20)

Secondary Outcomes (8)

• Percentage of Participants With Confirmed CMV viremia Clearance at Week 8

  At Week 8

• Percentage of Participants who Achieve Maintenance of Confirmed CMV Viremia Clearance and Symptom Control at Week 8 Through Weeks 12, 16 and 20

  At Week 8 through Weeks 12, 16, and 20

• Percentage of Participants With Confirmed Recurrence of CMV Viremia on Study Treatment and Off Study Treatment

  Up to Week 20

• Time to First Confirmed Viremia Clearance

  Up to Week 20

• Percentage of Participants With Confirmed Recurrence of CMV Viremia Treated With Alternative Anti-CMV Treatment During 12-Week Follow-up Period in Participants With Confirmed Viremia Clearance at Week 8

  From Week 8 through Week 20

Study Arms (3)

Cohort 1: Maribavir 400, 200 or 100 mg

EXPERIMENTAL

Participants with greater than or equal to (\>=) 12 to less than (\<) 18 years of age will receive maribavir 400 milligrams (mg) (2\*200 mg tablets or powder for oral suspension) twice daily (BID) based on body weight \>= 25 kilogram (kg); or 200 mg tablet or powder for oral suspension BID based on body weight 14 to \< 25 kg; or 100 mg powder for oral suspension BID based on body weight 10 to \< 14 kg for up to 8 weeks treatment period (Day 1/Week 0 to Day 56/Week 8).

Drug: Maribavir

Cohort 2: Maribavir 400, 200 or 100 mg

EXPERIMENTAL

Participants with \>= 6 to \< 12 years of age will receive maribavir 400 mg (2\*200 mg tablets or powder for oral suspension) BID based on body weight \>= 25 kg; or 200 mg tablet or powder for oral suspension BID based on body weight 14 to \< 25 kg; or 100 mg powder for oral suspension BID based on body weight 10 to \< 14 kg orally for up to 8 weeks treatment period (Day 1/Week 0 to Day 56/Week 8).

Drug: Maribavir

Cohort 3: Maribavir 400, 200, 100 or 50 mg

EXPERIMENTAL

Participants with 0 to \< 6 years of age will receive maribavir 400 mg (2\*200 mg tablets or powder for oral suspension) BID based on body weight \>= 25 kg; or 200 mg tablet or powder for oral suspension BID based on body weight 14 to \< 25 kg; or 100 mg powder for oral suspension BID based on body weight 10 to \< 14 kg; or 50 mg powder for oral suspension BID based on body weight 7 to \< 10 kg; or 50 mg powder for oral suspension once daily (QD) based on body weight 5 to \<7 kg for up to 8 weeks treatment period (Day 1/Week 0 to Day 56/Week 8).

Drug: Maribavir

Interventions

Maribavir DRUG

Participants will receive maribavir.

Also known as: TAK-620

Cohort 1: Maribavir 400, 200 or 100 mg; Cohort 2: Maribavir 400, 200 or 100 mg; Cohort 3: Maribavir 400, 200, 100 or 50 mg

Eligibility Criteria

• Age: Up to 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Takeda: lead
• Takeda Development Center Americas, Inc.: collaborator

Study Sites (47)

University of Nebraska Medical Center -985400 Nebraska Medical Center

Omaha, Nebraska, 68114-4113, United States

RECRUITING

Cincinnati Children's Hospital Medical Center - PIN

Cincinnati, Ohio, 45229-3026, United States

RECRUITING

Cook Children's Health Care System

Fort Worth, Texas, 76104-2733, United States

RECRUITING

University of Texas MD Anderson Cancer Center - 1515 Holcombe Blvd

Houston, Texas, 77030-4000, United States

RECRUITING

Sydney Children's Hospital

Randwick, New South Wales, 2031, Australia

RECRUITING

Queensland Children's Hospital

South Brisbane, Queensland, 4101, Australia

RECRUITING

Royal Children's Hospital Melbourne - PIN

Parkville, Victoria, 3052, Australia

RECRUITING

Perth Children's Hospital

Nedlands, Western Australia, 6009, Australia

RECRUITING

Hôpital Universitaire des Enfants Reine Fabiola (HUDERF)

Brussels, Brussels Capital, 1020, Belgium

RECRUITING

Cliniques Universitaires Saint-Luc

Woluwe-Saint-Lambert, Brussels Capital, 1200, Belgium

RECRUITING

UZ Gent

Ghent, Oost-Vlaanderen, 9000, Belgium

RECRUITING

Irmandade Da Santa Casa de Misericordia de Porto Alegre

Porto Alegre, Rio Grande do Sul, 90020-090, Brazil

RECRUITING

Hospital de Clinicas de Porto Alegre (HCPA) - PPDS

Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

RECRUITING

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

Porto Alegre, Rio Grande do Sul, 90160-093, Brazil

RECRUITING

Hospital Do Rim E Hipertensão

São Paulo, 04038-002, Brazil

RECRUITING

Children's Hospital Capital Institute of Pediatrics

Beijing, Beijing Municipality, 100020, China

RECRUITING

Beijing Children's Hospital, Capital Medical University - PIN

Beijing, Beijing Municipality, 100045, China

RECRUITING

Shanghai Children's Medical Center

Shanghai, Shanghai Municipality, 200127, China

RECRUITING

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

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

CHU de Rennes - Hôpital Pontchaillou

Rennes, Ille-et-Vilaine, 35200, France

RECRUITING

CHU de Grenoble Alpes - Hôpital Michallon

La Tronche, Isère, 38700, France

RECRUITING

CHRU Nantes

Nantes, Loire-Atlantique, 44000, France

RECRUITING

Hopital Necker

Paris, 75015, France

RECRUITING

Universitätsklinikum Würzburg

Würzburg, Bavaria, 97080, Germany

RECRUITING

Universitätsklinikum Münster

Münster, North Rhine-Westphalia, 48149, Germany

RECRUITING

Universitatsklinikum Jena - Am Klinikum 1-Erlanger Allee 101

Jena, Thuringia, 07745, Germany

RECRUITING

Universitätsklinikum Hamburg Eppendorf

Hamburg, 20246, Germany

RECRUITING

Medizinische Hochschule Hannover

Hanover, 30625, Germany

RECRUITING

The Chaim Sheba Medical Center - PPDS

Ramat Gan, Tel Aviv, 52394, Israel

RECRUITING

Tel Aviv Sourasky Medical Center Ichilov - PPDS

Tel Aviv, Tel Aviv, 64239, Israel

RECRUITING

Rambam Health Care Campus - PPDS

Haifa, 31096, Israel

RECRUITING

Hadassah Medical Center- Ein Kerem - PPDS

Petah Tikva, 4920235, Israel

RECRUITING

National Center for Child Health and Development

Nagoya, Aiti, 453-0046, Japan

RECRUITING

Saitama Children's Medical Center

Isehara-Shi, Kanagawa, 259-1143, Japan

RECRUITING

Shizuoka Children's Hospital

Aoi-ku, Shizuoka, 420-8660, Japan

RECRUITING

Hyogo Prefectural Kobe Children's Hospital

Chiba, 650-0047, Japan

RECRUITING

Osaka Women's and Children's Hospital

Izumi-Shi, Ôsaka, 594-1101, Japan

RECRUITING

Hospital Sant Joan de Deu - PIN

Espluges de Llobregat, Barcelona, 08950, Spain

RECRUITING

Hospital Universitario La Paz - PPDS

Horcajo de la Sierra, Madrid, 28755, Spain

RECRUITING

Hospital Regional Universitario de Malaga - Hospital Materno-Infantil

Málaga, Málaga, 29011, Spain

RECRUITING

Hospital Universitario Vall d´Hebron- PPDS

Barcelona, 08035, Spain

RECRUITING

Hospital Infantil Universitario Niño Jesus - PIN

Madrid, 28009, Spain

RECRUITING

King's College Hospital

London, Lambeth, SE5 9RS, United Kingdom

RECRUITING

Royal Manchester Children's Hospital - PIN

Manchester, Lancashire, M13 9WL, United Kingdom

RECRUITING

Nottingham University Hospitals NHS Trust

Nottingham, Nottinghamshire, NG5 1PB, United Kingdom

RECRUITING

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, West Midlands, B4 6NH, United Kingdom

RECRUITING

Great Ormond Street Hospital

London, WC1N 3JH, United Kingdom

RECRUITING

Related Publications (1)

• Fisher JE, Mulieri K, Finch E, Ericson JE. Use of Maribavir for Multidrug Resistant Cytomegaloviremia in a Pediatric Oncology Patient. J Pediatr Hematol Oncol. 2024 Apr 1;46(3):e244-e247. doi: 10.1097/MPH.0000000000002841. Epub 2024 Mar 1.

  PMID: 38447094 DERIVED

Related Links

• To obtain more information on the study, click here/on this link

MeSH Terms

Interventions

maribavir

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Central Study Contacts

Takeda Contact

CONTACT

+1-877-825-3327; medinfoUS@takeda.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Three dosing cohorts based on participant's age.

Study Record Dates

• First Submitted: April 1, 2022
• First Posted: April 8, 2022
• Study Start: November 13, 2023
• Primary Completion (Estimated): January 18, 2027
• Study Completion (Estimated): January 18, 2027
• Last Updated: July 17, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/ For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

IL (4); AU (4); BR (4); DE (5); CN (4); JP (5); GB (5); BE (3); ES (5); FR (4); US (4)