A Study of RPL554 in Patients With Cystic Fibrosis
Cystic FibrosisThis study evaluates two doses of RPL554 and placebo in adult patients with cystic fibrosis. All patients receive all three treatments in a randomised sequence.
A Study to Assess the Pharmacokinetics and Safety of Co-administered Oral Galicaftor, Navocaftor,...
Healthy VolunteersCystic Fibrosis (CF) is a rare, life-threatening, genetic disease that affects the lungs and digestive system, significantly impairing the quality of life, with those affected having a median age of death at 40. The main objectives of this study are to assess the safety and pharmacokinetics of the combination therapy of galicaftor/navocaftor/ABBV-576.
A Study to Evaluate the Relative Bioavailability of a Fixed-dose Combination Tablet of VX-121/Tezacaftor/Deutivacaftor...
Cystic FibrosisThe purpose of this study is to evaluate the relative bioavailability of a fixed-dose combination tablet (FDC) of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA).
An Electrocardiogram Study to Evaluate the Effect of Vanzacaftor on the QT/QTc Interval in Healthy...
Cystic FibrosisThe purpose of this study is to evaluate the effect of Vanzacaftor (VNZ) on QTcF, as well as the pharmacokinetic (PK), safety, and tolerability of VNZ in healthy participants.
Effectiveness of CFTR Modulators According to Co-therapy
Cystic FibrosisUnless CFTR modulators are highly effective, the introduction of CFTR (Cystic fibrosis transmembrane conductance regulator) modulators could lead to concomitant reduction or discontinuation of respiratory co-therapies in real-life. Such reduction/discontinuation of respiratory co-therapies could lead to an overall decrease of the effectiveness of CF care. MODUCO study aims: 1) to compare the clinical effectiveness on lung function and pulmonary exacerbation of CFTR modulator during the year of initiation, according to level of co-therapy among CF patients; 2) to describe the nature and level of respiratory co-therapies (azithromycin, RhDNase, inhaled antibiotics) in the year before the initiation of CFTR modulator; 3) to describe the changes in respiratory co-therapies during the first and the second year following the initiation of CFTR modulator and compare between the two CFTR modulators; 4) to describe adherence to CFTR modulator during the first and the second year following its initiation; 5) to study the association between the nature of respiratory co-therapies and adherence to CFTR modulator during the first and the second year following its initiation. A national population-based comparative effectiveness study will be conducted, based on retrospective analysis of clinical and prescription data of the French CF registry linked with the French national health data system (SNDS).
Effects of Breathing Exercises Combined With Endurance and Strength Training in Patients With Cystic...
Cystic Fibrosis PatientsCystic fibrosis (CF) is a multi-system inherited disease. It's a common autosomal recessive illness. It mostly affects the lungs, liver, and pancreatic exocrine glands, as well as the intestines. The production of viscous mucus and an environment prone to chronic airway blockage. This allows harmful microorganisms to infect the lungs. The role of Exercise as a prognostic indicator or therapeutic aid is important in CF research around the world. The objective of this study is to find out the Effects of Breathing Exercises Combined with Endurance and Strength Training on Dyspnea and Quality of Life of patients with Cystic Fibrosis. It will be Quasi Experimental study. Treatment will be given to all participants 3 sessions in a week for 4 weeks. Pre and Post treat-meant evaluation will be checked by CFQ-R+14. All patients will be treated with exercise program of Active cycle breathing techniques(ACBT), Pursed lip breathing, Endurance Exercise 20 to 30 min ( walking, cycling) and strength training with Thera-Bands (Bilateral arm raising, Bilateral knee extension). Exercise capacity will be measured with 6MWT. Dyspnea and fatigue will be measured with Borg scale.
Analysis T Cells Response for Identification of Aspergillus Bronchitis With Cystic Fibrosis Patients...
Cystic Fibrosis PatientPatient Without Treatment Against A.FumigatusThe study aims to asses the ability of cell tests based on the analysis of the anti-Aspergillus cell responses and identify Aspergillus bronchitis with patients with cystic fibrosis. In addition, the study will evaluate the contribution of biological classification of aspergillosis according to criteria recently proposed by Baxter et al. compared to the classification used in clinical practice in the hospital of Montpellier.
Study of Cavosonstat (N91115) in Patients With CF Homozygous for the F508del-CFTR Mutation
Cystic FibrosisThis will be a double-blind, randomized, placebo-controlled, parallel group study. The purpose of this study is to investigate the efficacy and safety of Cavosonstat (N91115) in adult patients with CF who are homozygous for the F508del-CFTR mutation and being treated with lumacaftor/ivacaftor (Orkambi™).
A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With...
Cystic FibrosisThis is a Phase 3, multicenter, open-label, 3-part rollover study in subjects with CF who are homozygous or heterozygous for the F508del-CFTR mutation and who participated in studies VX13-661-103 (Study 103, NCT02070744), VX14-661-106 (Study 106, NCT02347657), VX14-661-107 (Study 107, NCT02516410), VX14-661-108 (Study 108, NCT02392234), VX14-661-109 (Study 109, NCT02412111), VX14-661-111 (Study 111, NCT02508207), VX15-661-112 (NCT02730208), and VX16-661-114 (NCT03150719). The study is designed to evaluate the safety and efficacy of long-term treatment of VX-661 in combination with ivacaftor.
Safety, Pharmacokinetics and Pharmacodynamics Study of Inhaled QBW276 in Patients With Cystic Fibrosis...
Pulmonary Cystic FibrosisThis is a study of multiple doses of inhaled QBW276 in patients with cystic fibrosis on top of standard of care. The study was divided into 3 Cohorts. Cohorts 1 and 2 are designed to be a randomized, double-blind, placebo-controlled, parallel arm, multiple dose study to assess the safety, tolerability, pharmacokinetics, and preliminary efficacy of inhaled QBW276 over 1 week (cohort 1) or 2 weeks (cohort 2) in patients with cystic fibrosis regardless of their genotype. The study was terminated after Cohort 2 due to the resource issues.