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Clinical Trials About "Emerging gene therapies cystic fibrosis" RSS

07:30 EST 17th November 2019 | BioPortfolio

We list hundreds of Clinical Trials about "Emerging gene therapies cystic fibrosis" on BioPortfolio. We draw our references from global clinical trials data listed on ClinicalTrials.gov and refresh our database daily.

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Showing "Emerging gene therapies cystic fibrosis" Clinical Trials 1–25 of 8,100+

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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.


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.

Safety and Efficacy of Recombinant Adeno-Associated Virus Containing the CFTR Gene in the Treatment of Cystic Fibrosis

The purpose of this study is to confirm the improvement in pulmonary function and cytokine levels observed in the recently completed multidose aerosol study for the treatment of Cystic Fibrosis (CF).


Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-809 in subjects with cystic fibrosis who have the ∆F508-CFTR gene mutation.

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 ...

Phase I Randomized Study of Adeno-Associated Virus-CFTR Vector in Patients With Cystic Fibrosis

OBJECTIVES: I. Determine the maximum tolerated dose of recombinant adeno-associated virus-CFTR vector in patients with cystic fibrosis. II. Assess the safety of this gene therapy in these patients. III. Assess the in vivo gene transfer of this vector in these patients. IV. Assess the CFTR gene expression and physiologic activity following gene transfer in these patients. V. Assess the clinical impact of CFTR gene expression following gene transfer in thes...

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.

Surrogate Markers of Response to New Therapies in Cystic Fibrosis Patients

The purpose of this study is to determine which biological marker, or association of biological markers, best predict clinical response of cystic fibrosis patients to CFTR modulators.

Cystic Fibrosis and Cognitive Function

The long-term goal of this project is to examine the effects of cystic fibrosis (CF) and cystic fibrosis related diabetes (CFRD) on brain structure and function

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.

Gene Modifiers of Cystic Fibrosis Lung Disease

Ths purpose of this study is to examine genetic modifiers of the severity of cystic fibrosis lung disease.

Educational Program Through Video Conference for Inhaled Therapies in Cystic Fibrosis Patients

Educational programs are essential for cystic fibrosis patients who need to take several different drugs every day. Compliance is often klow in these patients, and clinicals and paramedics try to increase it, through educational programs. As for inhaled therapies, the CF center of Montpellier uses a video conference tool to educate patients. The program may involve either the physiotherapist or the doctor or both. Through the present study, the investigators would like to evalu...

High Resolution Micro OCT Imaging

The purpose of this study is to learn about using the imaging to make images of the lungs and nose with the long-term goal of the research leading to potential treatments and new therapies for patients with cystic fibrosis.

Exercise-Induced Bronchospasm in Cystic Fibrosis

Exercise is an important clinical feature in cystic fibrosis. Better exercise capacity has been associated with better patient outcomes and quality of life. Exercise-induced bronchospasm is a condition, often associated with asthma, which may make exercise difficult. The role that exercise-induced bronchospasm has in people with cystic fibrosis is unknown. This study is designed to determine how often exercise-induced bronchospam occurs in cystic fibrosis.

Telavancin Pharmacokinetics in Cystic Fibrosis Patients

Due to emerging resistance, new antibiotic options are needed to treat CF acute pulmonary exacerbations caused by methicillin resistant Staphylococcus aureus (MRSA). There is established evidence that adult patients with cystic fibrosis (CF) may have altered antibiotic pharmacokinetics compared with non-CF patients. Telavancin is a lipoglycopeptide antibiotic that has activity against gram-positive bacteria including MRSA. This study will determine the pharmacokinetics and tole...

Study of Uridine Triphosphate (UTP) as an Aerosol Spray for Cystic Fibrosis

OBJECTIVES: I. Determine the stability of uridine triphosphate (UTP) and examine the metabolism of exogenous nucleotides on airway epithelial surfaces in patients with cystic fibrosis. II. Determine the acute safety and efficacy of aerosolized UTP in children with cystic fibrosis.

Determination of the Predictive Factors in the Reversibility or the Aggravation in the Disorders of the Glucose Metabolism in Cystic Fibrosis Patients

For some years, the investigators observe an increase of the arisen of diabetes in cystic fibrosis patients However, this diabetes may be reversible. The investigators speak about " Cystic fibrosis related diabetes.". The objective of this project, is to know better what facilitates the appearance and the reversibility of the diabetes, such as the genetic mutations, the respiratory state and the lung infections. Theses knowledges should allow to adapt the screening of diabetes,...

Registry Study on Cystic Fibrosis in Chinese Children

This study is a multicenter, prospective cohort study of patients diagnosed with cystic fibrosis, the clinical information of recruited patients, including clinical manifestations, lung function, chest imaging, quality of life and other indicators, will be followed for 10 years.

Ready to Use Therapeutic Food (RUTF) to Promote Growth in Cystic Fibrosis

Children with cystic fibrosis require increased caloric intake to maintain appropriate growth, an important determinant of long-term outcomes. This study seeks to determine the feasibility of using a novel therapeutic food to promote weight gain and growth in children with cystic fibrosis.

Effect of Losartan in Cystic Fibrosis (CF)-NIH Grant #133240

The goal of this study is to execute a small clinical proof of concept trial: To examine the effects of losartan on mucociliary clearance (MCC) in patients not eligible for CFTR rescue therapies

Non-Invasive Biomarkers in Cystic Fibrosis

Background Chronic airway inflammation is present in cystic fibrosis. Non-invasive inflammometry may be useful in disease management. Objective We studied 1) the ability of fractional exhaled nitric oxide and inflammatory markers (acidity, nitrite, nitrate, hydrogen peroxide, 8-isoprostane, interferon-γ, tumor necrosis factor-α, interleukin-2,-4,-5,-10) in exhaled breath condensate, to discriminate between cystic fibrosis and control children, and, 2) the relationship of bio...

Durability of Hypertonic Saline for Enhancing Mucociliary Clearance in Cystic Fibrosis

Direct measurement of mucociliary and cough clearance (MCC/CC) has been used as a biomarker in cystic fibrosis (CF). Additional knowledge of the performance of this biomarker is needed to inform exploratory clinical trial design in support of programs to develop new inhaled therapies for CF. We hypothesize that MCC/CC measurements can be used to determine the durability of action of agents like hypertonic saline (HS) which increase epithelial lining fluid height.

Single Dose of pGM169/GL67A in CF Patients

The study objectives are to assess safety, tolerability and gene expression after a single dose of non-viral CFTR gene therapy (pGM169/GL67A) administered to the nose and lungs of patients with cystic fibrosis.

FDG-PET Imaging in Young Cystic Fibrosis Patients

The purpose of this research is to determine how a person's lungs will uptake [18F]fluorodeoxyglucose (FDG), as measured with positron emission tomography (PET) scanning in young cystic fibrosis patients.


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