Remote endpoints for clinical trials in cystic fibrosis: Report from the U.S. CF foundation remote endpoints task force

Jordana E Hoppe; Jacquelyn Sjoberg; Gina Hong; Katie Poch; Edith T Zemanick; Stephanie Thee; Claire Edmondson; Dhiren Patel; Meghana Sathe; Drucy Borowitz; Melissa S Putman; Noah Lechtzin; Kristin A Riekert; Melissa Basile; Christopher H Goss; Mary Elizabeth Jarosz; Margaret Rosenfeld

doi:10.1016/j.jcf.2024.02.011

Remote endpoints for clinical trials in cystic fibrosis: Report from the U.S. CF foundation remote endpoints task force

J Cyst Fibros. 2024 Feb 29:S1569-1993(24)00023-7. doi: 10.1016/j.jcf.2024.02.011. Online ahead of print.

Authors

Jordana E Hoppe¹, Jacquelyn Sjoberg², Gina Hong³, Katie Poch⁴, Edith T Zemanick⁵, Stephanie Thee⁶, Claire Edmondson⁷, Dhiren Patel⁸, Meghana Sathe⁹, Drucy Borowitz¹⁰, Melissa S Putman¹¹, Noah Lechtzin¹², Kristin A Riekert¹², Melissa Basile¹³, Christopher H Goss¹⁴, Mary Elizabeth Jarosz², Margaret Rosenfeld¹⁵

Affiliations

¹ Department of Pediatrics, University of Colorado School of Medicine and Children's Hospital Colorado, Aurora CO, USA. Electronic address: jordana.hoppe@childrenscolorado.org.
² Cystic Fibrosis Foundation, Bethesda MD, USA.
³ Department of Medicine, Division of Pulmonary, Allergy and Critical Care, Perelman School of Medicine at the University of Pennsylvania, Philadelphia Pennsylvania, USA.
⁴ Department of Medicine, National Jewish Health, Denver CO, USA.
⁵ Department of Pediatrics, University of Colorado School of Medicine and Children's Hospital Colorado, Aurora CO, USA.
⁶ Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center, Charité - Universitätsmedizin Berlin, Corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany.
⁷ Department of Paediatric Respiratory Medicine, Great Ormond Street Hospital, London WC1N3JH, USA.
⁸ Division of Pediatric Gastroenterology, Hepatology and Nutrition, Cardinal Glennon Children's Medical Center, Saint Louis University School of Medicine, St. Louis MO, USA.
⁹ Pediatric Gastroenterology and Nutrition, University of Texas Southwestern/Children's Health, Dallas Texas, USA.
¹⁰ Department of Pediatrics, Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, Buffalo New York, USA.
¹¹ Division of Pediatric Endocrinology, Boston Children's Hospital, Boston MA, USA.
¹² Division of Pulmonary and Critical Care, Johns Hopkins University School of Medicine, Baltimore Maryland, USA.
¹³ Feinstein Institutes for Medical Research, Northwell Health, Manhasset NY, USA.
¹⁴ Division of Pulmonary, Critical Care and Sleep Medicine, Department of Medicine, University of Washington Medical Center, Seattle Washington, USA; Department of Pediatrics, University of Washington School of Medicine and Seattle Children's Research Institute, Seattle Washington, USA.
¹⁵ Department of Pediatrics, University of Washington School of Medicine and Seattle Children's Research Institute, Seattle Washington, USA.

PMID: 38429150
DOI: 10.1016/j.jcf.2024.02.011

Abstract

The COVID-19 pandemic necessitated a rapid shift in clinical research to perform virtual visits and remote endpoint assessments, providing a key opportunity to optimize the use of remote endpoints for clinical trials in cystic fibrosis. The use of remote endpoints could allow more diverse participation in clinical trials while minimizing participant burden but must be robustly evaluated to ensure adequate performance and feasibility. In response, the Cystic Fibrosis Foundation convened the Remote Endpoint Task Force (Supplemental Table 1), a multidisciplinary group of CF researchers with remote endpoint expertise and community members tasked to better understand the current and future use of remote endpoints for clinical research. Here, we describe the current use of remote endpoints in CF clinical research, address key unanswered questions regarding their use and feasibility, and discuss the next steps to determine clinical trial readiness.

Keywords: Clinical trials; Cystic fibrosis; Home; Remote monitoring.

Publication types

Review