Statistical analysis plan for the “empirical treatment against cytomegalovirus and tuberculosis in HIV-infected infants with severe pneumonia” clinical trial

on behalf of EMPIRICAL group

doi:10.1186/s13063-025-08841-7

Statistical analysis plan for the “empirical treatment against cytomegalovirus and tuberculosis in HIV-infected infants with severe pneumonia” clinical trial

on behalf of EMPIRICAL group

Faculty of Education

Fundación Biomedica del Hospital Universitario (FIB-H12O)
Universidad Europea
Spanish Clinical Research Network (SCReN)
Complutense University
Hospital Universitario Infanta Sofía
Servicio Madrileño de Salud
London School of Hygiene and Tropical Medicine
Institut national de la santé et de la recherche médicale
CERPOP - Centre d'Epidémiologie et de Recherche en Santé des Populations
Penta-Onlus Foundation (PENTA)
Radboud University Nijmegen
University of Barcelona
ICREA
Centro de investigação de Saúde de Manhiça
Instituto de Salud Carlos III
University of Lincoln
Ivory Coast
Kamuzu University of Health Sciences
University of North Carolina at Chapel Hill
University of California at Los Angeles
Universidade Eduardo Mondlane
Makerere University
Joint Clinical Research Center
University of Zambia
University of Zimbabwe

Research output: Contribution to journal › Article › peer-review

Abstract

Background: The EMPIRICAL trial aims to assess safety and efficacy of an empirical treatment against cytomegalovirus (CMV) and tuberculosis (TB) compared to standard of care (SoC), on adverse events and 15-day and 1-year mortality among infants living with HIV hospitalized with severe pneumonia in Africa.

Methods and design: The EMPIRICAL trial (NCT03915366) is an international multicenter phase II-III, open-label randomized factorial clinical trial conducted in six African countries. The trial has four randomization arms in a 1:1:1:1 fashion with patients allocated to (i) TB-Treatment plus SoC, (ii) valganciclovir plus SoC, (iii) both TB-Treatment and valganciclovir plus SoC, and (iv) SoC only.

Discussion: This paper describes the statistical analysis plan (SAP) for the trial which, per the study publication plan, needs to be published prior to the database lock and final analysis results. The SAP includes details of the analyses to be undertaken and unpopulated tables that will be reported to address primary and secondary endpoints. The database will be locked on 31st January 2025.

Original language	English
Article number	26
Pages (from-to)	144
Journal	Trials
Volume	26
Issue number	1
DOIs	https://doi.org/10.1186/s13063-025-08841-7
Publication status	Published - 30 Apr 2025

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

SDG 3 Good Health and Well-being

Keywords

Child mortality
Cytomegalovirus
Empirical
Factorial
Factorial randomized clinical trial
HIV
Infants
Pneumonia
Statistical analysis plan
Tuberculosis
Valganciclovir

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10.1186/s13063-025-08841-7Licence: CC BY

s13063-025-08841-7Final published version, 1.74 MBLicence: CC BY

Cite this

@article{c9eaf6991af1494a929d49b0d075a0f7,

title = "Statistical analysis plan for the “empirical treatment against cytomegalovirus and tuberculosis in HIV-infected infants with severe pneumonia” clinical trial",

abstract = "Background: The EMPIRICAL trial aims to assess safety and efficacy of an empirical treatment against cytomegalovirus (CMV) and tuberculosis (TB) compared to standard of care (SoC), on adverse events and 15-day and 1-year mortality among infants living with HIV hospitalized with severe pneumonia in Africa. Methods and design: The EMPIRICAL trial (NCT03915366) is an international multicenter phase II-III, open-label randomized factorial clinical trial conducted in six African countries. The trial has four randomization arms in a 1:1:1:1 fashion with patients allocated to (i) TB-Treatment plus SoC, (ii) valganciclovir plus SoC, (iii) both TB-Treatment and valganciclovir plus SoC, and (iv) SoC only. Discussion: This paper describes the statistical analysis plan (SAP) for the trial which, per the study publication plan, needs to be published prior to the database lock and final analysis results. The SAP includes details of the analyses to be undertaken and unpopulated tables that will be reported to address primary and secondary endpoints. The database will be locked on 31st January 2025. ",

keywords = "Child mortality, Cytomegalovirus, Empirical, Factorial, Factorial randomized clinical trial, HIV, Infants, Pneumonia, Statistical analysis plan, Tuberculosis, Valganciclovir",

author = "\{on behalf of EMPIRICAL group\} and Sara Dom{\'i}nguez-Rodr{\'i}guez and David Lora and Alfredo Tagarro and Cinta Moraleda and {\'A}lvaro Ballesteros and Lola Madrid and Lilit Manukyan and Olivier Marcy and Valeriane Leroy and Alessandra Nardone and David Burger and Quique Bassat and Matthew Bates and Raoul Moh and Tam, \{Pui Ying Iroh\} and Tisungane Mvalo and Justina Magallhaes and Buck, \{W. Chris\} and Jahit Sacarlal and Victor Mussime and Chishala Chabala and Mujuru, \{Hilda Angela\} and Pablo Rojo",

note = "Publisher Copyright: {\textcopyright} The Author(s) 2025.",

year = "2025",

month = apr,

day = "30",

doi = "10.1186/s13063-025-08841-7",

language = "English",

volume = "26",

pages = "144",

journal = "Trials",

issn = "1745-6215",

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TY - JOUR

T1 - Statistical analysis plan for the “empirical treatment against cytomegalovirus and tuberculosis in HIV-infected infants with severe pneumonia” clinical trial

AU - on behalf of EMPIRICAL group

AU - Domínguez-Rodríguez, Sara

AU - Lora, David

AU - Tagarro, Alfredo

AU - Moraleda, Cinta

AU - Ballesteros, Álvaro

AU - Madrid, Lola

AU - Manukyan, Lilit

AU - Marcy, Olivier

AU - Leroy, Valeriane

AU - Nardone, Alessandra

AU - Burger, David

AU - Bassat, Quique

AU - Bates, Matthew

AU - Moh, Raoul

AU - Tam, Pui Ying Iroh

AU - Mvalo, Tisungane

AU - Magallhaes, Justina

AU - Buck, W. Chris

AU - Sacarlal, Jahit

AU - Mussime, Victor

AU - Chabala, Chishala

AU - Mujuru, Hilda Angela

AU - Rojo, Pablo

PY - 2025/4/30

Y1 - 2025/4/30

N2 - Background: The EMPIRICAL trial aims to assess safety and efficacy of an empirical treatment against cytomegalovirus (CMV) and tuberculosis (TB) compared to standard of care (SoC), on adverse events and 15-day and 1-year mortality among infants living with HIV hospitalized with severe pneumonia in Africa. Methods and design: The EMPIRICAL trial (NCT03915366) is an international multicenter phase II-III, open-label randomized factorial clinical trial conducted in six African countries. The trial has four randomization arms in a 1:1:1:1 fashion with patients allocated to (i) TB-Treatment plus SoC, (ii) valganciclovir plus SoC, (iii) both TB-Treatment and valganciclovir plus SoC, and (iv) SoC only. Discussion: This paper describes the statistical analysis plan (SAP) for the trial which, per the study publication plan, needs to be published prior to the database lock and final analysis results. The SAP includes details of the analyses to be undertaken and unpopulated tables that will be reported to address primary and secondary endpoints. The database will be locked on 31st January 2025.

AB - Background: The EMPIRICAL trial aims to assess safety and efficacy of an empirical treatment against cytomegalovirus (CMV) and tuberculosis (TB) compared to standard of care (SoC), on adverse events and 15-day and 1-year mortality among infants living with HIV hospitalized with severe pneumonia in Africa. Methods and design: The EMPIRICAL trial (NCT03915366) is an international multicenter phase II-III, open-label randomized factorial clinical trial conducted in six African countries. The trial has four randomization arms in a 1:1:1:1 fashion with patients allocated to (i) TB-Treatment plus SoC, (ii) valganciclovir plus SoC, (iii) both TB-Treatment and valganciclovir plus SoC, and (iv) SoC only. Discussion: This paper describes the statistical analysis plan (SAP) for the trial which, per the study publication plan, needs to be published prior to the database lock and final analysis results. The SAP includes details of the analyses to be undertaken and unpopulated tables that will be reported to address primary and secondary endpoints. The database will be locked on 31st January 2025.

KW - Child mortality

KW - Cytomegalovirus

KW - Empirical

KW - Factorial

KW - Factorial randomized clinical trial

KW - HIV

KW - Infants

KW - Pneumonia

KW - Statistical analysis plan

KW - Tuberculosis

KW - Valganciclovir

U2 - 10.1186/s13063-025-08841-7

DO - 10.1186/s13063-025-08841-7

M3 - Article

C2 - 40307889

AN - SCOPUS:105004361084

SN - 1745-6215

VL - 26

SP - 144

JO - Trials

JF - Trials

IS - 1

M1 - 26

ER -

Statistical analysis plan for the “empirical treatment against cytomegalovirus and tuberculosis in HIV-infected infants with severe pneumonia” clinical trial

Abstract

UN SDGs

Keywords

Access to Document

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Cite this