Topics

Clinical Trials About "Beyond cystic fibrosis transmembrane conductance regulator therapy perspective" RSS

17:39 EST 10th December 2019 | BioPortfolio

We list hundreds of Clinical Trials about "Beyond cystic fibrosis transmembrane conductance regulator therapy perspective" on BioPortfolio. We draw our references from global clinical trials data listed on ClinicalTrials.gov and refresh our database daily.

More Information about "Beyond cystic fibrosis transmembrane conductance regulator therapy perspective" on BioPortfolio

We have published hundreds of Beyond cystic fibrosis transmembrane conductance regulator therapy perspective news stories on BioPortfolio along with dozens of Beyond cystic fibrosis transmembrane conductance regulator therapy perspective Clinical Trials and PubMed Articles about Beyond cystic fibrosis transmembrane conductance regulator therapy perspective for you to read. In addition to the medical data, news and clinical trials, BioPortfolio also has a large collection of Beyond cystic fibrosis transmembrane conductance regulator therapy perspective Companies in our database. You can also find out about relevant Beyond cystic fibrosis transmembrane conductance regulator therapy perspective Drugs and Medications on this site too.

Showing "Beyond cystic fibrosis transmembrane conductance regulator therapy perspective" Clinical Trials 1–25 of 15,000+

Extremely Relevant

Phase I Study of Liposome-Mediated Gene Transfer in Patients With Cystic Fibrosis

OBJECTIVES: Evaluate the efficacy and safety of lipid-mediated transfer of the cystic fibrosis transmembrane conductance regulator gene to nasal epithelium in patients with cystic fibrosis.


Detection of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Activity in Rectal Tissues From Human Subjects

This is a pilot study. The purpose of the study is to facilitate the development of a new biomarker of cystic fibrosis transmembrane conductance regulator (CFTR) function using rectal tissue.

Miglustat / OGT 918 in the Treatment of Cystic Fibrosis

Cystic fibrosis is a genetic disease caused by mutation of the cystic fibrosis transmembrane conductance regulator (CFTR). The purpose of the study is to investigate the effects of miglustat on CFTR function in cystic fibrosis patients.


In Vitro Model of the Cystic Fibrosis Bronchial Epithelium Via iPS Technology

In order to further enable physiopathology studies, the aim of this project is to validate an in vitro model of cystic fibrosis created using induced pluripotent stemcell (iPS) differentiated bronchial epithelium from cystic fibrosis (CF) patients homozygous for the p.Phe508del mutation of the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation

Exploratory proof of concept study to determine whether intranasal administration of QR-010 in subjects with cystic fibrosis, homozygous or compound heterozygous for the ΔF508 mutation, can increase the function of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR).

Study of VX-770 in Cystic Fibrosis Subjects Age 12 and Older Homozygous for the F508del-CFTR Mutation

Cystic Fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The encoded protein, CFTR, is an epithelial chloride ion channel responsible for aiding in the regulation of salt and water absorption and secretion in various tissues. Although the disease affects multiple organs, the leading cause of mortality is the progressive loss of lung function. Obstruction of airways with thick mucous, chronic bacterial infection of th...

Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations

This study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation.

New Markers of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Function in Sweat

The aim of the study is to identify new biomarkers of CFTR function in sweat and in sweat gland.

Orkambi Treatment in 2 to 5 Year Old Children With CF

The purpose of this observational research study is to determine the effects of clinically prescribed Orkambi treatment on 2 to 5 year old children homozygous for the F508del Mutations in the Cystic fibrosis transmembrane conductance regulator (CFTR) gene on sleeping energy expenditure, growth status and gut health and function.

A Study Evaluating the Safety and Efficacy of VX-440 Combination Therapy in Subjects With Cystic Fibrosis

This is a Phase 2, randomized, double-blind, placebo- and active-controlled, parallel group, multicenter study to evaluate the safety, tolerability, and efficacy of VX-440 in dual and triple combination with tezacaftor (TEZ; VX-661) and ivacaftor (IVA; VX-770) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation of the CF transmembrane conductance regulator (CFTR) gene (F508del/F508del), or who are heterozygous for the F508del mutation and a minimal...

A Study Evaluating the Safety of VX-152 Combination Therapy in Adults With Cystic Fibrosis

This is a Phase 2, randomized, double blind, placebo and active-controlled, parallel group, multicenter study designed to evaluate the safety and tolerability of VX-152 in Triple Combination (TC) with tezacaftor (TEZ; VX-661) and ivacaftor (IVA; VX-770) in subjects with cystic fibrosis (CF) who are heterozygous for the F508del mutation and a minimal function (MF) CFTR mutation not likely to respond to TEZ and/or IVA therapy (F508del/MF), or who are homozygous for the F508del mu...

A Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis

This is a first-in-human and proof-of-concept study of VX-445. The study includes 5 parts. Parts A, B, and C will be conducted in healthy subjects. Parts D and E will be conducted in subjects with Cystic Fibrosis (CF) who are homozygous for the F508del mutation of the CF transmembrane conductance regulator (CFTR) gene (F/F genotype), or who are heterozygous for the F508del mutation and a minimal function (MF) CFTR mutation not likely to respond to TEZ, IVA, or TEZ/IVA (F/MF gen...

A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation

Study to evaluate the efficacy of VX-661 in combination with ivacaftor through Week 12 in subjects with cystic fibrosis (CF) who are heterozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene and with a second CFTR mutation that is not likely to respond to VX-661 and/or ivacaftor therapy (F508del/not responsive [NR]).

Safety and Efficacy of Inhaled OligoG CF-5/20 for the Treatment Cystic Fibrosis

Cystic fibrosis (CF) is a disease caused by a mutation in the gene that makes the cystic fibrosis transmembrane regulator protein. As a result mucus stagnation, obstruction and plugging take place in the respiratory and gastrointestinal tract, the biliary and pancreatic duct, and in the reproductive system. OligoG CF-5/20 has been shown to reduce sputum from CF patients. The objective of this study is to determine the safety and tolerability of 3 days of daily dosing of OligoG ...

A Pilot Study to Evaluate the Use of Smart Adherence Technology to Measure Medication Adherence in CF Subjects Who Are Homozygous for the F508del CFTR Mutation

The study will evaluate the impact of smart adherence technologies for medication monitoring on lumacaftor/ivacaftor (LUM/IVA) adherence rates among subjects 16 years of age and older with Cystic Fibrosis (CF) who are homozygous for the F508del Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) mutation.

A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

To evaluate the clinical mechanisms of action in lung and extrapulmonary systems of VX-661 in combination with ivacaftor (IVA) (VX-661/IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.

Extension Study of Ataluren (PTC124) in Cystic Fibrosis

Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A specific type of mutation called a nonsense (premature stop codon) mutation is the cause of CF in approximately 10% of subjects with the disease. Ataluren (PTC124) is an orally delivered investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 3 extension trial ...

PTC124 for Cystic Fibrosis

In some patients with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein. PTC124 has been shown to partially restore CFTR production in animals with CF due to a nonsense mutation. The main purpose of this study is to understand whether PTC124 can safely increase functional CFTR protein in the cells of patients with CF due to a nonsense mutation.

PTC124 for Pediatric Patients With Cystic Fibrosis

In some patients with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein. PTC124 has been shown to partially restore CFTR production in animals with CF due to a nonsense mutation. The main purpose of this study is to understand whether PTC124 can safely increase functional CFTR protein in the cells of patients with CF due to a nonsense mutation.

Nasal Potential Difference (NPD) Protocol in Chronic Rhinosinusitis

The purpose of this study is to determine if acquired (partial) Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) deficiency contributes substantially to the pathogenic mechanisms underlying Chronic Rhinosinusitis (CRS), creating a localized environment that impairs mucociliary clearance (MCC).

Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor

Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109 (NCT02514473) or Study 011B (NCT01897233). Study 110 is designed to evaluate the safety and efficacy of long term treatment of lumacaftor in combination with ivacaftor.

A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation

To evaluate the efficacy and safety of lumacaftor in combination with ivacaftor in subjects aged 6 Through 11 years with cystic fibrosis (CF), homozygous for the F508del CF transmembrane conductance regulator (CFTR) mutation

Study of Ataluren (PTC124™) in Cystic Fibrosis

Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A specific type of mutation called a nonsense (premature stop codon) mutation is the cause of CF in approximately 10% of subjects with the disease. Ataluren (PTC124) is an orally delivered investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 3 trial that will ...

Study of VX-770 in Cystic Fibrosis Subjects Age 6 to 11 With the G551D Mutation

Cystic Fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The encoded protein, CFTR, is an epithelial chloride ion channel responsible for aiding in the regulation of salt and water absorption and secretion in various tissues. Although the disease affects multiple organs, the leading cause of mortality is the progressive loss of lung function. Obstruction of airways with thick mucous, chronic bacterial infection of th...

Study of VX-770 in Cystic Fibrosis Subjects Age 12 and Older With the G551D Mutation

Cystic Fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The encoded protein, CFTR, is an epithelial chloride ion channel responsible for aiding in the regulation of salt and water absorption and secretion in various tissues. Although the disease affects multiple organs, the leading cause of mortality is the progressive loss of lung function. Obstruction of airways with thick mucous, chronic bacterial infection of th...


More From BioPortfolio on "Beyond cystic fibrosis transmembrane conductance regulator therapy perspective"

Quick Search