Table 1.

Publications evaluating safety and efficacy and post-approval observational studies of CF transmembrane conductance regulator (CFTR) modulators in CF patients (*).

ClinicalTrials.gov ID	Phase	Subjects	Age (years)	Drug(s)	Follow up	Reference(s)
NCT00909532	III	G551D in at least one allele	≥12	Ivacaftor	48 weeks	Ramsey et al., 2011; Quittner et al., 2015; Solem et al., 2016; Flume et al., 2018
NCT01225211	II	F508del-homozygous and -heterozygous	≥18	Lumacaftor/Ivacaftor	56 days	Boyle et al., 2014; Rowe et al., 2017b
NCT01531673	II	F508del-homozygous and -heterozygous	≥12	Tezacaftor/Ivacaftor	56 days	Donaldson et al., 2018a
NCT01705145	III	A gating mutation in at least one allele	2–5	Ivacaftor	24 weeks	Davies et al., 2016
NCT01784419	N-of-1	A gating mutation in at least one allele	≥8	Ivacaftor	Cycles of 14 days	McGarry et al., 2017
NCT01807923 and NCT01807949	III	F508del-homozygous	≥12	Lumacaftor/Ivacaftor	24 weeks	Wainwright et al., 2015; Elborn et al., 2016; Flume et al., 2019; McColley et al., 2019
NCT01897233	III	F508del-homozygous	6–11	Lumacaftor/Ivacaftor	24 weeks	Milla et al., 2017
NCT01931839	III	F508del-homozygous	≥12	Lumacaftor/Ivacaftor	96 weeks	Konstan et al., 2017
NCT01937325	IV	G551D in at least one allele	≥16	Ivacaftor	3 months	Edgeworth et al., 2017
NCT01946412	III	A gating mutation in at least one allele	2–5	Ivacaftor	84 weeks	Rosenfeld et al., 2019
NCT02141464	IV	A gating mutation in at least one allele	≥6	Ivacaftor	3 months	Stallings et al., 2018
NCT02275936	I	F508del-homozygous	≥18	Cavosonstat	28 days	Donaldson et al., 2017
NCT02347657	III	F508del-homozygous	≥12	Tezacaftor/Ivacaftor	24 weeks	Taylor-Cousar et al., 2017
NCT02390219	III	F508del-homozygous	≥12	Lumacaftor/Ivacaftor	24 weeks	Taylor-Cousar et al., 2018
NCT02392234	III	F508del-heterozygous with a residual function mutation in trans	≥12	Tezacaftor/Ivacaftor	8 weeks	Rowe et al., 2017a
NCT02514473	III	F508del-homozygous	6–11	Lumacaftor/Ivacaftor	24 weeks	Ratjen et al., 2017
NCT02707562	II	G551D in at least one allele	≥18	ABBV-974	4 weeks	Davies et al., 2019
NCT02725567	III	A gating mutation in at least one allele	1–2	Ivacaftor	24 weeks	Rosenfeld et al., 2018
NCT02797132	III	F508del-homozygous	2–5	Lumacaftor/Ivacaftor	24 weeks	McNamara et al., 2019
NCT02807415	IV	F508del-homozygous	≥6	Lumacaftor/Ivacaftor	16 weeks	Graeber et al., 2018
NCT02953314	III	F508del-homozygous and -heterozygous	6–11	Tezacaftor/Ivacaftor	24 weeks	Walker et al., 2019
NCT02965326	IV	F508del-homozygous	≥12	Lumacaftor/Ivacaftor	6 months	Pranke et al., 2019
NCT03029455 and NCT03224351	II	F508del-homozygous and -heterozygous with a residual function mutation in trans	≥18	VX-659/Tezacaftor/Ivacaftor	4 weeks	Davies et al., 2018
NCT03045523	II	F508del-heterozygous with a gating mutation in trans	≥18	ABBV-2222 on top of Ivacaftor	29 days	Bell et al., 2019
NCT03119649	II	F508del-homozygous	≥18	ABBV-2222	29 days	Bell et al., 2019
NCT03227471	I/II	F508del-homozygous and -heterozygous with a residual function mutation in trans	≥18	Elexacaftor/Tezacaftor/Ivacaftor	4 weeks	Keating et al., 2018
NCT03525444	III	F508del-heterozygous with a minimal function mutation in trans	≥12	Elexacaftor/Tezacaftor/Ivacaftor	24 weeks	Middleton et al., 2019
NCT03525548	III	F508del-homozygous	≥12	Elexacaftor/Tezacaftor/Ivacaftor	4 weeks	Heijerman et al., 2019
NCT03474042	IIa	F508del-homozygous	≥18	ABBV-2737 on top of lumacaftor/ivacaftor	28 days	van Koningsbruggen-Rietschel et al., 2019

(*)This table is a continuation of Table 3 published in Lopes-Pacheco, 2016.