Active clinical trials for "Cystic Fibrosis"

This is a Phase 3, randomized, double blind, placebo controlled, parallel group, multicenter study in people with cystic fibrosis (CF) who are homozygous for the F508del CF transmembrane conductance regulator (CFTR) gene mutation.

The purpose of this study is to assess the efficacy of IV gallium to improve pulmonary function as measured by a 5% or greater relative improvement in forced expiratory volume in one second (FEV1) from baseline to Day 28. Funding Source - FDA OOPD

The purpose of this study is to assess whether inhalation of 7% hypertonic saline (HS) twice daily for 48 weeks improves the lung clearance index by multiple breath nitrogen washout in comparison with inhalation of 0.9% isotonic saline (IS) in preschool children (ages 3 to 5) with cystic fibrosis.

The purpose of this research study is to learn about the safety of transplanting lungs obtained from non-heart-beating donors (NHBDs) that have been ventilated (attached to a breathing machine or ventilator to deliver oxygen) and perfused with a lung perfusion solution (Steen solution™, made by Vitrolife). This ventilation and perfusion will be done outside the body (ex-vivo) in a modified cardiopulmonary bypass circuit (the kind of device used routinely during most heart surgeries). The purpose of performing ex-vivo perfusion and ventilation is to learn how well the lungs work, and whether they are likely safe to transplant.

The purpose of this study is to determine whether PUR118 is safe and tolerable in a population of subjects with Cystic Fibrosis.

The purpose of this study is to assess the safety and tolerability of ascending doses of a novel oral formulation of Fenretinide to adult cystic fibrosis (CF) patients, once-daily for 21 days (treatment cycle). This study will include up to three (3) dose levels with minimum 7 day breaks in between treatment cycles. For each dose level, blood samples will be collected for exploratory pharmacokinetic (PK) and pharmacodynamic (PD) evaluation.

This Phase 1b study in F508del-CFTR homozygous CF patients is being conducted to assess the safety of N91115 as the sole cystic fibrosis transmembrane conductance regulator (CFTR) modulator at doses near the expected therapeutic exposure level in preparation for Phase 2 studies of N91115 added to the CFTR modulator combination lumacaftor/ivacaftor when launched.

The primary aim of the research is to evaluate whether an alternative model of cystic fibrosis (CF) physiotherapy care can produce statistically significant improvements in clinical and patient reported outcomes, and whether this alternative model is economically advantageous and/or sustainable. Children randomised to the control group will receive 24-months of current model of CF care at Great Ormond Street Hospital (GOSH). Children randomised to the intervention group will receive 24-months of current model of CF care at GOSH PLUS a weekly structured, individually prescribed and personally supervised exercise intervention at a local fitness facility or at school. The exercise prescription will include aerobic, anaerobic, strength, core conditioning and stretching components. The main objectives of the study are: Determine differences, if any, in lung function between the two groups; Determine differences, if any, in exercise capacity between the two groups; Evaluate cost of care of alternate model of care versus current model of care.

The purpose of the study is to further evaluate the safety and efficacy of EUR-1008 as compared to Kreon® in the treatment of exocrine pancreatic insufficiency associated with Cystic Fibrosis in subjects 12 years of age and older.

The purpose of this study is to confirm and extend the Phase 1-2 KB001 findings of an airway anti-inflammatory effect in CF individuals with chronic Pseudomonas aeruginosa (Pa) airway infection. It is hypothesized that steady-state levels of KB001-A in CF subjects with airway Pa infection will be safe and well-tolerated, and will increase the time-to-need for antibiotic treatment (IV, inhaled, or oral) for worsening of respiratory tract signs and symptoms compared with placebo.