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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05319353




Registration number
NCT05319353
Ethics application status
Date submitted
1/04/2022
Date registered
8/04/2022

Titles & IDs
Public title
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)
Scientific 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)
Secondary ID [1] 0 0
2021-004279-15
Secondary ID [2] 0 0
TAK-620-2004
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cytomegalovirus (CMV) 0 0
Condition category
Condition code
Infection 0 0 0 0
Other infectious diseases
Infection 0 0 0 0
Studies of infection and infectious agents

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Maribavir

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).


Treatment: Drugs: Maribavir
Participants will receive maribavir.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Maximum Observed Plasma Concentration (Cmax) of Maribavir
Timepoint [1] 0 0
Pre-dose; 0.5, 1.5, 3, 4, 6, and 8 hours post-dose on Day 7 (Week 1)
Primary outcome [2] 0 0
Time to Maximum Observed Concentration (Tmax) of Maribavir
Timepoint [2] 0 0
Pre-dose; 0.5, 1.5, 3, 4, 6, and 8 hours post-dose on Day 7 (Week 1)
Primary outcome [3] 0 0
Minimum Plasma Concentration (Cmin) of Maribavir
Timepoint [3] 0 0
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)
Primary outcome [4] 0 0
Area Under the Plasma Concentration-Time Curve Over the 1 Dosing Interval of 12 Hours at Steady State (AUC0-tau) of Maribavir
Timepoint [4] 0 0
Pre-dose; 0.5, 1.5, 3, 4, 6, and 8 hours post-dose on Day 7 (Week 1)
Primary outcome [5] 0 0
Half-Life (t1/2) of Maribavir
Timepoint [5] 0 0
Pre-dose; 0.5, 1.5, 3, 4, 6, and 8 hours post-dose on Day 7 (Week 1)
Primary outcome [6] 0 0
Terminal Elimination Rate Constant (lambdaz) of Maribavir
Timepoint [6] 0 0
Pre-dose; 0.5, 1.5, 3, 4, 6, and 8 hours post-dose on Day 7 (Week 1)
Primary outcome [7] 0 0
Apparent Volume of Distribution (Vz/F) of Maribavir
Timepoint [7] 0 0
Pre-dose; 0.5, 1.5, 3, 4, 6, and 8 hours post-dose on Day 7 (Week 1)
Primary outcome [8] 0 0
Apparent Oral Clearance (CL/F) of Maribavir
Timepoint [8] 0 0
Pre-dose; 0.5, 1.5, 3, 4, 6, and 8 hours post-dose on Day 7 (Week 1)
Primary outcome [9] 0 0
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [9] 0 0
From start of study drug administration up to follow-up (Week 20)
Secondary outcome [1] 0 0
Percentage of Participants With Confirmed CMV viremia Clearance at Week 8
Timepoint [1] 0 0
At Week 8
Secondary outcome [2] 0 0
Percentage of Participants who Achieve Maintenance of Confirmed CMV Viremia Clearance and Symptom Control at Week 8 Through Weeks 12, 16 and 20
Timepoint [2] 0 0
At Week 8 through Weeks 12, 16, and 20
Secondary outcome [3] 0 0
Percentage of Participants With Confirmed Recurrence of CMV Viremia on Study Treatment and Off Study Treatment
Timepoint [3] 0 0
Up to Week 20
Secondary outcome [4] 0 0
Time to First Confirmed Viremia Clearance
Timepoint [4] 0 0
Up to Week 20
Secondary outcome [5] 0 0
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
Timepoint [5] 0 0
From Week 8 through Week 20
Secondary outcome [6] 0 0
Change From Baseline in Log10 Plasma CMV Deoxyribonucleic Acid (DNA) Load
Timepoint [6] 0 0
Baseline up to Week 20
Secondary outcome [7] 0 0
Number of Participants who Develop CMV Resistance to Maribavir
Timepoint [7] 0 0
Up to Week 12
Secondary outcome [8] 0 0
Summary Scores for Palatability Assessment of Maribavir
Timepoint [8] 0 0
At Weeks 1, 4, and 8

Eligibility
Key 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 (ß-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.
Minimum age
No limit
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
13/11/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
18/01/2027
Actual
Sample size
Target
80
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
Recruitment hospital [1] 0 0
Sydney Children's Hospital - Randwick
Recruitment hospital [2] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [3] 0 0
Royal Children's Hospital Melbourne - PIN - Parkville
Recruitment hospital [4] 0 0
Perth Children's Hospital - Nedlands
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
3052 - Parkville
Recruitment postcode(s) [4] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Nebraska
Country [2] 0 0
United States of America
State/province [2] 0 0
Ohio
Country [3] 0 0
United States of America
State/province [3] 0 0
Texas
Country [4] 0 0
Belgium
State/province [4] 0 0
Brussels
Country [5] 0 0
Belgium
State/province [5] 0 0
Oost-Vlaanderen
Country [6] 0 0
Brazil
State/province [6] 0 0
Rio Grande Do Sul
Country [7] 0 0
Brazil
State/province [7] 0 0
São Paulo
Country [8] 0 0
China
State/province [8] 0 0
Beijing
Country [9] 0 0
China
State/province [9] 0 0
Shanghai
Country [10] 0 0
China
State/province [10] 0 0
Tianjin
Country [11] 0 0
France
State/province [11] 0 0
Ille-et-Vilaine
Country [12] 0 0
France
State/province [12] 0 0
Isère
Country [13] 0 0
France
State/province [13] 0 0
Loire-Atlantique
Country [14] 0 0
France
State/province [14] 0 0
Paris
Country [15] 0 0
Germany
State/province [15] 0 0
Bayern
Country [16] 0 0
Germany
State/province [16] 0 0
Nordrhein-Westfalen
Country [17] 0 0
Germany
State/province [17] 0 0
Thüringen
Country [18] 0 0
Germany
State/province [18] 0 0
Hamburg
Country [19] 0 0
Germany
State/province [19] 0 0
Hannover
Country [20] 0 0
Israel
State/province [20] 0 0
Tel-Aviv
Country [21] 0 0
Israel
State/province [21] 0 0
Haifa
Country [22] 0 0
Israel
State/province [22] 0 0
Petah Tiqva
Country [23] 0 0
Japan
State/province [23] 0 0
Aiti
Country [24] 0 0
Japan
State/province [24] 0 0
Kanagawa
Country [25] 0 0
Japan
State/province [25] 0 0
Sizuoka
Country [26] 0 0
Japan
State/province [26] 0 0
Chiba-Shi
Country [27] 0 0
Japan
State/province [27] 0 0
Ôsaka
Country [28] 0 0
Spain
State/province [28] 0 0
Barcelona
Country [29] 0 0
Spain
State/province [29] 0 0
Madrid
Country [30] 0 0
Spain
State/province [30] 0 0
Málaga
Country [31] 0 0
United Kingdom
State/province [31] 0 0
Lambeth
Country [32] 0 0
United Kingdom
State/province [32] 0 0
Lancashire
Country [33] 0 0
United Kingdom
State/province [33] 0 0
Nottinghamshire
Country [34] 0 0
United Kingdom
State/province [34] 0 0
West Midlands
Country [35] 0 0
United Kingdom
State/province [35] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Takeda
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Takeda Development Center Americas, Inc.
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Study Director
Address 0 0
Takeda
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Takeda Contact
Address 0 0
Country 0 0
Phone 0 0
+1-877-825-3327
Fax 0 0
Email 0 0
medinfoUS@takeda.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR)
When will data be available (start and end dates)?
Available to whom?
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.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org/ourmember/takeda/


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05319353