Skip to main content

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) 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
  1. Data is correct as of 19/07/2017 from https://clinicaltrials.gov/ct2/home
  2. MF minimal CFTR function mutation, RF residual CFTR function mutation, 508 p.Phe508del