Table 3.
Completed clinical trials of CFTR modulators in CF patients.
| ClinicalTrials.gov ID (formerly name) | Phase | Subjects | Age (years) | Drug(s) | Follow up | Reference(s) |
|---|---|---|---|---|---|---|
| NCT00237380 | 2 | Nonsense mutationsa,b | ≥18 | Ataluren | 56 days | Kerem et al., 2008 |
| NCT00351078 | 2 | Nonsense mutationsa,b | ≥18 | Ataluren | 112 days | Wilschanski et al., 2011 |
| NCT00457821 | 2 | G551D-homozygous and -heterozygous | ≥18 | Ivacaftor | 28 days | Accurso et al., 2010, 2014 |
| NCT00458341 | 2 | Nonsense mutationsa,c,d | 6–18 | Ataluren | 28 days | Sermet-Gaudelus et al., 2010 |
| NCT00803205 | 3 | Nonsense mutationsa,c | ≥6 | Ataluren | 48 weeks | Kerem et al., 2014 |
| NCT00865904 | 2 | ΔF508-homozygous | ≥18 | Lumacaftor | 28 days | Clancy et al., 2012 |
| NCT00909532 (STRIVE) | 3 | G551D-homozygous and -heterozygous | ≥12 | Ivacaftor | 48 weeks | Ramsey et al., 2011; Quittner et al., 2015 |
| NCT00953706 (DISCOVER) | 2 | ΔF508-homozygous | ≥12 | Ivacaftor | 16 weeks | Flume et al., 2012 |
| NCT01117012 (PERSIST) | 3 | G551D-homozygous and -heterozygous | ≥6 | Ivacaftor | 96 weeks | McKone et al., 2014 |
| NCT01225211 | 2 | ΔF508-homozygous and -heterozygous | ≥18 | Lumacaftor and Ivacaftor | 56 days | Boyle et al., 2014 |
| NCT01262352 (ENVISION) | 2 | G551D-homozygous and -heterozygous | ≥6 | Ivacaftor | 48 weeks | Davies et al., 2013a,b |
| NCT01521338 (GOAL) | 4 | G551D-homozygous and -heterozygous | ≥6 | Ivacaftor | 6 months | Rowe et al., 2014; O’Connor and Seegmiller, 2016 |
| NCT01531673 | 2 | ΔF508-homozygous and -heterozygous | ≥12 | VX-661 and/or Ivacaftor | 28 days | ∗∗∗ |
| NCT01614457 (KONDUCT) | 3 | R117H-homozygous and -heterozygous | ≥6 | Ivacaftor | 24 weeks | Moss et al., 2015 |
| NCT01614470 (KONNECTION) | 3 | Non-G551D gating mutations in at least one allelee | ≥6 | Ivacaftor | 24 weeks | De Boeck et al., 2014 |
| NCT01685801 | 2 | R117H and/or CFTR mutations with residual function in at least one allele b,f, G551D and/or other gating mutations in at least one allelee | ≥12 | Ivacaftor | 24 weeks | ∗∗∗ |
| NCT01705045 (KIWI) | 3 | G551D-homozygous and heterozygous | 2–5 | Ivacaftor | 24 weeks | Davies et al., 2016 |
| NCT01707290 | 3 | Non-G551D gating or residual function mutations in at least one alleleb,e,f |
≥6 | Ivacaftor | 24 weeks | ∗∗∗ |
| NCT01807923 (TRAFFIC) | 3 | ΔF508-homozygous | ≥12 | Lumacaftor and Ivacaftor | 24 weeks | Wainwright et al., 2015; Elborn et al., 2016 |
| NCT01807949 (TRANSPORT) | 3 | ΔF508-homozygous | ≥12 | Lumacaftor and Ivacaftor | 24 weeks | Wainwright et al., 2015; Elborn et al., 2016 |
| NCT01897233 | 3 | ΔF508-homozygous | 6–11 | Lumacaftor and Ivacaftor | 24 weeks | ∗∗∗ |
| NCT01931839 | 3 | ΔF508-homozygous | ≥12 | Lumacaftor and Ivacaftor | 96 weeks | ∗∗∗ |
aG542X, W1282X; b3849 + 10 kbC→T; CR553X, R1162X; dQ492X, E1104X, W846X, W882X, Q1313X; eG178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349D; fR117H, E56K, P67L, D110E, D110H, R117C, R347H, R352Q, A455E, D579G, S945L, L206W, R1070W, F1074L, D1152H, S1235R, D1270N, 2789 + 5G→A, 3272-26A→G, 711 + 5G→A, 3120G→A, 1811 + 1.6 kbA- > G, 711 + 3A→G, 1898 + 3A→G, 1898 + lG→A, 1717-lG→A, 1717-8G→A, 1342-2A→C, 405 + 3A→C, 1716G/A 1811 + lG→C, 1898 + 5G→T, 3850-3T→G, IVS14b + 5G→A, 1898 + lG→T, 4005 + 2T→C, 621 + 3A→G, 621 + lG→T. ∗∗∗Manuscript not available yet.