Active clinical trials for "Cystic Fibrosis"

The purpose of this study is to demonstrate that Miglustat restores the function of the cystic fibrosis transmembrane conductance regulator (CFTR) in adult patients with cystic fibrosis homozygous for the F508del mutation.

It is the goal of the proposed study to compare the efficacy, as assessed primarily by sputum weight, of these two different devices (the Electroflo 500 and VEST therapy) for airway clearance (AC) in CF patients with mild to moderate lung disease, who have stable lung health and perform AC at home as part of their routine therapeutic regimen.

Cystic fibrosis-related diabetes (CFRD) occurs in almost 20% of teens and 50% of adults. The investigators' long term goal is to determine the cause of CFRD in order to translate this knowledge into therapies aimed at preventing CFRD. Since CFRD and type 2 diabetes share several clinical features and since oxidative stress is a key factor in the development of type 2 diabetes, the investigators explored the role of oxidative stress in CFRD. The investigators discovered a unique CF biochemical signature that they believe could be implicated in the development of CFRD. The investigators found that glucose ingestion in CF teens and young adults causes an acute and profound systemic redox imbalance to the oxidizing state. The degree of redox imbalance was quite severe and would be expected to damage the insulin producing cells as these cells are particularly vulnerable to oxidative stress. Thus, these findings could prove to be a critical factor in the pathogenesis of CFRD. This proposal will test the hypothesis that glucose-induced redox imbalance is an intrinsic, metabolic defect in CF. In addition, because CF people are required to consume a high calorie diet to maintain their weight, the investigators also hypothesize that certain high caloric foods will recapitulate the redox imbalance induced by ingesting glucose and thus hasten the development of CFRD. Specifically, the investigators aim to: Determine whether young children with CF have glucose-induced redox imbalance Determine whether eating a meal with a high glycemic index induces acute redox imbalance Determine whether commonly consumed beverages containing simple sugars (i.e., soda or fruit juice) induce acute redox imbalance

This Phase 1 study in F508del-CFTR homozygous CF patients is being conducted to assess the pharmacokinetics and absorption dynamics of N91115 compared with healthy subjects in order to identify an initial starting dose for Phase 2 trials.

Safety, tolerability and pharmacokinetics following single doses

The Lung Clearance Index (LCI) is a non invasive measure of lung function that is more sensitive than FEV1. It can be used to measure lung function in children younger than 6 years of age. Therefore, it has a future role in assessing novel therapeutics in the Cystic Fibrosis (CF) population. As such, determining if it can be used as a short term pharmacodynamic biomarker is paramount.

This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).

The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.

The purpose of this study is to evaluate the safety, tolerability and efficacy of VX-121 combination therapy in subjects with cystic fibrosis (CF).

This study will evaluate the safety, tolerability, and efficacy of ABBV-3067 given alone and in combination with various doses of ABBV-2222 in adults with Cystic Fibrosis who are homozygous for the F508del mutation.