Table 2.
CFTR drugs in current phase 2 or 3 clinical trials
| Name | Clinical trial ID | Co-treatment | Phase | Trial status | Mutation | Comments |
|---|---|---|---|---|---|---|
| GLPG2222 |
NCT03119649 NCT03045523 |
– Ivacaftor |
2 2 |
Recruiting Recruiting |
508/508 508/class III |
Corrector |
| Cavosonstat (N91115) |
NCT02589236
NCT02724527 |
Orkambi Ivacaftor |
2 2 |
Complete Ongoing, but not recruiting |
508/508 508/class III |
Increase levels of a signalling molecule S-nitrosogluthione (GSNO) |
| Tezacaftor (VX-661) |
NCT02392234 NCT03150719 NCT02951182 NCT02951195 |
Ivacaftor Ivacaftor Ivacaftor + VX-440 Ivacaftor + VX-152 |
3 3 2 2 |
Complete Recruiting Recruiting Recruiting |
508/RF 508/508 508/508; 508/MF 508/508; 508/MF |
Move the defective CFTR protein to the proper place in the airway cell surface |
| CTP-656 | NCT02971839 | – | 2 | Recruiting | Gating mutations | Altered form of ivacaftor |
| Riociguat (BAY63-2521) | NCT02170025 | – | 2 | Recruiting | 508/508 | Stimulates soluble guanylyl cyclases (sGC), an enzyme in the cardiopulmonary system |
| QR-010 | NCT02532764 | – | 1/2 | Recruiting | 508/508 | RNA-based oligonucleotide |
Data is correct as of 19/07/2017 from https://clinicaltrials.gov/ct2/home
MF minimal CFTR function mutation, RF residual CFTR function mutation, 508 p.Phe508del