Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2016 Jun;175(6):817-24.
doi: 10.1007/s00431-016-2712-z. Epub 2016 Mar 15.

Disease-specific clinical trials networks: the example of cystic fibrosis

Kris De Boeck  1 , Veerle Bulteel  2 , Isabelle Fajac  3   4
Affiliations

Disease-specific clinical trials networks: the example of cystic fibrosis

Kris De Boeck et al. Eur J Pediatr. 2016 Jun.

Abstract

This article describes the steps of the development and the structure of a disease-specific clinical trials network for cystic fibrosis in Europe. Activities such as reviewing study protocols, feasibility assessments, training and standardizing of procedures, and outcome measurements help to bring high-quality clinical trials to the patients. Cooperation with the pharmaceutical industry, other research networks, patient organizations, and regulatory agencies is very important throughout all activities.

Conclusion: The European Cystic Fibrosis Society-Clinical Trials Network facilitates the development of new treatments for a rare disease and could be a prototype for other diseases.

What is known: • Clinical research has led to the first approved treatments targeting the basic Cystic Fibrosis defect. • For a rare disease like Cystic Fibrosis, multicenter international collaboration is needed to obtain solid evidence when testing possible new treatments. What is New: • The Clinical Trials Network established by the European Cystic Fibrosis Society has grown to a fully operational network with well-defined structures, procedures and partnerships. • Standardization of outcome parameters, protocol review, feasibility assessment and other activities help to develop high quality, efficient, relevant and feasible clinical trials, with the aim to bring new treatments to the patients.

Keywords: Clinical trials; Cystic fibrosis; Rare diseases; Research network.

PubMed Disclaimer

Figures

Fig. 1
Fig. 1
Structure of the European Cystic Fibrosis Society—Clinical Trials Network (ECFS-CTN)
See this image and copyright information in PMC

References

    1. Bell SC, De Boeck K, Amaral MD. New pharmacological approaches for cystic fibrosis: promises, progress, pitfalls. Pharmacol Ther. 2015;145C:19–34. doi: 10.1016/j.pharmthera.2014.06.005. - DOI - PubMed
    1. Boyle MP, De Boeck K. A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect. Lancet Respir Med. 2013;1(2):158–63. doi: 10.1016/S2213-2600(12)70057-7. - DOI - PubMed
    1. Briggs TA, Bryant M, Smyth RL. Controlled clinical trials in cystic fibrosis—are we doing better? J Cyst Fibros. 2006;5(1):3–8. doi: 10.1016/j.jcf.2005.09.005. - DOI - PubMed
    1. Carbone PP, Tormey DC. Organizing multicenter trials: lessons from the cooperative oncology groups. Prev Med. 1991;20:162–169. doi: 10.1016/0091-7435(91)90017-X. - DOI - PubMed
    1. De Boeck K, Fajac I, Ratjen F. ERS monograph Cystic Fibrosis, European Respiratory Society. 2014. End-points and biomarkers for clinical trials in cystic fibrosis; pp. 104–115.