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)

Status: Recruiting
Location: See all (47) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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
Participation Requirements
Sex: All
Maximum Age: 17
Healthy Volunteers: f
View:

• 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.

Locations
United States
Nebraska
University of Nebraska Medical Center -985400 Nebraska Medical Center
RECRUITING
Omaha
Ohio
Cincinnati Children's Hospital Medical Center - PIN
RECRUITING
Cincinnati
Texas
Cook Children's Health Care System
RECRUITING
Fort Worth
University of Texas MD Anderson Cancer Center - 1515 Holcombe Blvd
RECRUITING
Houston
Other Locations
Australia
Perth Children's Hospital
RECRUITING
Nedlands
Royal Children's Hospital Melbourne - PIN
RECRUITING
Parkville
Sydney Children's Hospital
RECRUITING
Randwick
Queensland Children's Hospital
RECRUITING
South Brisbane
Belgium
Hôpital Universitaire des Enfants Reine Fabiola (HUDERF)
RECRUITING
Brussels
UZ Gent
RECRUITING
Ghent
Cliniques Universitaires Saint-Luc
RECRUITING
Woluwe-saint-lambert
Brazil
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
RECRUITING
Porto Alegre
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
RECRUITING
Porto Alegre
Irmandade Da Santa Casa de Misericordia de Porto Alegre
RECRUITING
Porto Alegre
Hospital Do Rim E Hipertensão
RECRUITING
São Paulo
China
Beijing Children's Hospital, Capital Medical University - PIN
RECRUITING
Beijing
Children's Hospital Capital Institute of Pediatrics
RECRUITING
Beijing
Shanghai Children's Medical Center
RECRUITING
Shanghai
Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences - PPDS
RECRUITING
Tianjin
France
CHU de Grenoble Alpes - Hôpital Michallon
RECRUITING
La Tronche
CHRU Nantes
RECRUITING
Nantes
Hopital Necker
RECRUITING
Paris
CHU de Rennes - Hôpital Pontchaillou
RECRUITING
Rennes
Germany
Universitätsklinikum Hamburg Eppendorf
RECRUITING
Hamburg
Medizinische Hochschule Hannover
RECRUITING
Hanover
Universitatsklinikum Jena - Am Klinikum 1-Erlanger Allee 101
RECRUITING
Jena
Universitätsklinikum Münster
RECRUITING
Münster
Universitätsklinikum Würzburg
RECRUITING
Würzburg
Israel
Rambam Health Care Campus - PPDS
RECRUITING
Haifa
Hadassah Medical Center- Ein Kerem - PPDS
RECRUITING
Petah Tikva
The Chaim Sheba Medical Center - PPDS
RECRUITING
Ramat Gan
Tel Aviv Sourasky Medical Center Ichilov - PPDS
RECRUITING
Tel Aviv
Japan
Shizuoka Children's Hospital
RECRUITING
Aoi-ku
Hyogo Prefectural Kobe Children's Hospital
RECRUITING
Chiba
Saitama Children's Medical Center
RECRUITING
Isehara-shi
Osaka Women's and Children's Hospital
RECRUITING
Izumi-shi
National Center for Child Health and Development
RECRUITING
Nagoya
Spain
Hospital Universitario Vall d´Hebron- PPDS
RECRUITING
Barcelona
Hospital Sant Joan de Deu - PIN
RECRUITING
Espluges De Llobregat
Hospital Universitario La Paz - PPDS
RECRUITING
Horcajo De La Sierra
Hospital Infantil Universitario Niño Jesus - PIN
RECRUITING
Madrid
Hospital Regional Universitario de Malaga - Hospital Materno-Infantil
RECRUITING
Málaga
United Kingdom
Birmingham Women's and Children's NHS Foundation Trust
RECRUITING
Birmingham
Great Ormond Street Hospital
RECRUITING
London
King's College Hospital
RECRUITING
London
Royal Manchester Children's Hospital - PIN
RECRUITING
Manchester
Nottingham University Hospitals NHS Trust
RECRUITING
Nottingham
Contact Information
Primary
Takeda Contact
medinfoUS@takeda.com
+1-877-825-3327
Time Frame
Start Date: 2023-11-13
Estimated Completion Date: 2027-01-18
Participants
Target number of participants: 80
Treatments
Experimental: Cohort 1: Maribavir 400, 200 or 100 mg
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).
Experimental: Cohort 2: Maribavir 400, 200 or 100 mg
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).
Experimental: Cohort 3: Maribavir 400, 200, 100 or 50 mg
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).
Sponsors
Collaborators: Takeda Development Center Americas, Inc.
Leads: Takeda

This content was sourced from clinicaltrials.gov