Table 2 CFTR drugs in current phase 2 or 3 clinical trials
From: The potential of antisense oligonucleotide therapies for inherited childhood lung diseases
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) | 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 |