Cystic Fibrosis Clinical Trials


A Phase 2a, Single-Blind, Placebo-Controlled, Parallel-Group Study to Assess Safety, Tolerability, and Pharmacokinetics of Brensocatib Tablets in Adults With Cystic Fibrosis

  • ClinicalTrials.gov: NCT05090904
  • The main objective of the study is to evaluate the safety and pharmacokinetics of brensocatib in participants with cystic fibrosis over a 4 week treatment period. This drug is an oral drug which is expected to reduce inflammation in the lungs.

SIMPLIFY

  • ClinicalTrials.gov: NCT04378153
  • A protocol to test the impact of discontinuing chronic therapies in people with cystic fibrosis on highly effective CFTR modulator therapy.

PROMISE

  • ClinicalTrials.gov: NCT04038047
  • A prospective study to evaluate biological and clinical effects of significantly corrected CFTR function (the PROMISE Study).

NTM PREDICT Trial

  • ClinicalTrials.gov: NCT02073409
  • Prospective Evaluation of NTM Disease In Cystic Fibrosis. The study is observational in nature, and is designed to advance understanding of nontuberculous mycobacteria (NTM) infection and facilitate the decision making process regarding treatment. (Cystic Fibrosis Foundation).

NTM PATIENCE Trial

  • ClinicalTrials.gov:​​​​​​​​​​​​​​ NCT02419989
  • Prospective Algorithm for Treatment of NTM in Cystic Fibrosis. This trial tests the feasibility of a standardized treatment algorithm for confirmed infection with either M. avium complex or the M. abscessus complex. (Cystic Fibrosis Foundation).

MAYFLOWERS Trial

  • ClinicalTrials.gov:​​​​​​​​​​​​​​ NCT04828382
  • In this study, the investigators aim to evaluate changes in lung function in women with cystic fibrosis (CF) during pregnancy and for 2 years after pregnancy based on exposure to highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators.

VERTEX 20-121-102

  • ClinicalTrials.gov:​​​​​​​​​​​​​​ NCT05033080
  • Prospective randomized clinical trial comparing elexacaftor/tezacaftor/ivacaftor (“trikafta”) with the investigational drug VX121//tezacaftor/deutivacaftor in patients with a single F508 mutation and a minimal function mutation.

VERTEX 20-121-10

  • ClinicalTrials.gov:​​​​​​​​​​​​​​ ​​​​​​​NCT05033080
  • Prospective randomized clinical trials comparing elexacaftor/tezacaftor/ivacaftor (“trikafta”) with the investigational drug VX 121//tezacaftor/deutivacaftor in patients homozygous (two copies) for the F508del mutation or heterozygous for F508 plus a gating or residual function mutation.