Disease-Specific Clinical Trial Networks: the Example of Cystic Fibrosis

Cystic Fibrosis (CF) is an autosomal recessive, life-shortening multiorgan disorder. It is the most frequent rare disease affecting about 42.000 patients in Europe and more than 80.000 patients worldwide. CF is caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene leading to absent or defective chloride channels (CFTR).

• References

• 1 Zolin A, McKone EF, van Rens J. et al. ECFS Patient Registry Annual Data Report 2010. Karup: European Cystic Fibrosis Society; 2014
• 2 De Boeck K, Bulteel V, Tiddens H. et al. on behalf of all ECFS-CTN network partners. Guideline on the design and conduct of cystic fibrosis clinical trials: The European Cystic Fibrosis Society – Clinical Trials Network (ECFS-CTN). JCF 2011; 10 (Suppl. 02) S67-S74
• 3 Boeck K, Bulteel V, Fajac I. Disease-specific clinical trials networks: the example of cystic fibrosis. Eur J Pediatr 2016; 175: 817-824