TY - JOUR
T1 - BEAT CF pulmonary exacerbations core protocol for evaluating the management of pulmonary exacerbations in people with cystic fibrosis
AU - Schultz, Andre
AU - McLeod, Charlie
AU - Berry, Scott
AU - Marsh, Julie
AU - McKenzie, Anne
AU - Messer, Mitch
AU - Wood, Jamie
AU - Saville, Ben
AU - Jaffe, Adam
AU - Ranganathan, Sarath
AU - Stick, Steve
AU - Wark, Peter
AU - Webb, Steve
AU - Snelling, Tom
N1 - Funding Information:
The authors acknowledge the BEAT-CF consumer representative group for their contributions to the design and conduct of BEAT-CF and thank the people living with CF (and their guardians, where relevant) who choose to participate in this study.
Funding Information:
Funding is provided by a Medical Research Future Fund (MRFF) Lifting Clinical Trials and Registries Capacity Grant (GNT1152376). The current funding period ends in May 2024. The funding body will have no role in the design of the study or the collection, analysis, or interpretation or reporting or data. Funding allocation to parties contributing to the study will be administered by the University of Sydney and will be in accordance with a research collaboration agreement. Fellowship support was provided to Schultz by the NHMRC (GNT1168022) and MRFF grants (MRF1193796). Fellowship funding was provided to Snelling by an MRFF Investigator Award (MRF1195153).
Publisher Copyright:
© 2023, Crown.
PY - 2023/12
Y1 - 2023/12
N2 - Background: Cystic fibrosis (CF) is a rare, inherited, life-limiting condition predominantly affecting the lungs, for which there is no cure. The disease is characterized by recurrent pulmonary exacerbations (PEx), which are thought to drive progressive lung damage. Management of these episodes is complex and generally involves multiple interventions targeting different aspects of disease. The emergence of innovative trials and use of Bayesian statistical methods has created renewed opportunities for studying heterogeneous populations in rare diseases. Here, we present the protocol for the BEAT CF PEx cohort, a prospective, multi-site, perpetual, platform enrolling adults and children with CF. The BEAT CF PEx cohort will be used to evaluate the comparative effectiveness of interventions for the treatment of PEx requiring intensive therapy (PERITs), with a primary focus on short-term improvements in lung function. This will be achieved through the conduct of cohort-nested studies, including adaptive clinical trials, within the BEAT CF PEx cohort. This protocol will outline key features of the BEAT CF PEx cohort, including the design, implementation, data collection and management, governance and analysis, and dissemination of results. Methods: This platform will be conducted across multiple sites, commencing with CF treatment centers in Australia. People of all ages with a clinical diagnosis of CF will be eligible to participate, except those who have previously received a lung transplant. Data including demographic and clinical information, treatment details, and outcomes (including safety, microbiology, and patient-reported outcome measures including quality of life scores) will be systematically collected and securely stored via a digital centralized trial management system (CTMS). The primary endpoint is the absolute change in the percentage predicted forced expiratory volume in 1 s (ppFEV1) from the commencement of intensive therapy to 7 to 10 days afterwards. Discussion: The BEAT CF PEx cohort will report clinical, treatment, and outcome data for PEx among people with CF and is intended to serve as a core (master) protocol for future nested, interventional trials evaluating treatment(s) for these episodes. The protocols for nested sub-studies are beyond the scope of this document and will be reported separately. Trial registration: ANZCTR BEAT CF Platform – ACTRN12621000638831. Registration date: Sept. 26, 2022.
AB - Background: Cystic fibrosis (CF) is a rare, inherited, life-limiting condition predominantly affecting the lungs, for which there is no cure. The disease is characterized by recurrent pulmonary exacerbations (PEx), which are thought to drive progressive lung damage. Management of these episodes is complex and generally involves multiple interventions targeting different aspects of disease. The emergence of innovative trials and use of Bayesian statistical methods has created renewed opportunities for studying heterogeneous populations in rare diseases. Here, we present the protocol for the BEAT CF PEx cohort, a prospective, multi-site, perpetual, platform enrolling adults and children with CF. The BEAT CF PEx cohort will be used to evaluate the comparative effectiveness of interventions for the treatment of PEx requiring intensive therapy (PERITs), with a primary focus on short-term improvements in lung function. This will be achieved through the conduct of cohort-nested studies, including adaptive clinical trials, within the BEAT CF PEx cohort. This protocol will outline key features of the BEAT CF PEx cohort, including the design, implementation, data collection and management, governance and analysis, and dissemination of results. Methods: This platform will be conducted across multiple sites, commencing with CF treatment centers in Australia. People of all ages with a clinical diagnosis of CF will be eligible to participate, except those who have previously received a lung transplant. Data including demographic and clinical information, treatment details, and outcomes (including safety, microbiology, and patient-reported outcome measures including quality of life scores) will be systematically collected and securely stored via a digital centralized trial management system (CTMS). The primary endpoint is the absolute change in the percentage predicted forced expiratory volume in 1 s (ppFEV1) from the commencement of intensive therapy to 7 to 10 days afterwards. Discussion: The BEAT CF PEx cohort will report clinical, treatment, and outcome data for PEx among people with CF and is intended to serve as a core (master) protocol for future nested, interventional trials evaluating treatment(s) for these episodes. The protocols for nested sub-studies are beyond the scope of this document and will be reported separately. Trial registration: ANZCTR BEAT CF Platform – ACTRN12621000638831. Registration date: Sept. 26, 2022.
KW - Clinical trial
KW - Cohort
KW - Cystic fibrosis
KW - Exacerbations
KW - FEV
KW - Lung function
KW - Management
KW - Platform
KW - Treatment
UR - http://www.scopus.com/inward/record.url?scp=85150812259&partnerID=8YFLogxK
U2 - 10.1186/s13063-023-07076-8
DO - 10.1186/s13063-023-07076-8
M3 - Article
C2 - 36949472
AN - SCOPUS:85150812259
SN - 1745-6215
VL - 24
JO - Trials
JF - Trials
IS - 1
M1 - 211
ER -