Publication
Presented on: October 22, 2025
Published in: Gene Therapy
October 22: In the article “Characteristics of long-term follow-up studies for gene therapies registered on ClinicalTrials.gov,” published in Gene Therapy, co-authors Carolyn Chapman, Emina Berbic, Ava Glazier, and Barbara Bierer report a descriptive study of key characteristics of LTFU gene therapy study protocols registered in ClinicalTrials.gov. The analysis enabled a better understanding of how registered LTFU studies are currently designed and stimulated ideas for improvement. Most notably, the results suggest a lack of harmonization in how safety outcomes are monitored and reported across LTFU studies. Standardization and/or harmonization of data and outcome reporting may increase the scientific value of these studies.
Chapman, C.R., Glazier, A., Berbić, E. et al. Characteristics of long-term follow-up studies for gene therapies registered on ClinicalTrials.gov. Gene Ther (2025). https://doi.org/10.1038/s41434-025-00571-4