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Found 9 clinical trials
A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation

and a gating (F/G) or residual function (F/RF) mutation, or have at least 1 other TCR CF transmembrane conductance regulator (CFTR) gene mutation and no F508del mutation.

fibrosis
forced expiratory volume
tezacaftor
cftr gene
  • 4 views
  • 05 Jul, 2022
  • 130 locations
Validation of Therapeutic Efficacy Targeting the Splicing Variants in Cystic Fibrosis and CFTR Pathologies (ONB-CFTR)

Cystic Fibrosis, an inherited autosomal recessive disease, arises from mutations in the CFTR gene. For intronic mutations affecting splicing events, oligonucleotides therapy has the potential to

fibrosis
  • 0 views
  • 19 Apr, 2022
  • 1 location
Mutation-specific Therapy for the Long QT Syndrome

developed and currently indicated for the treatment of cystic fibrosis (CF) in patients homozygous for the F508del mutation in the CFTR gene. LUM corrects protein folding and trafficking defects of mutant

beta blockers
electrocardiogram
atrial flutter
fibrillation
myocarditis
  • 0 views
  • 01 Sep, 2021
  • 1 location
Comparison Between Ultra-low-dose Computed Tomography and Lung MRI in Cystic Fibrosis (UBD-IRM)

The purpose of this study is to compare the performances of ultra-low dose computed tomography (CT) and lung magnetic resonance imaging (MRI) for morphological assessment of cystic fibrosis-related lung disease and to compare their performances to conventional low dose CT

fibrosis
diagnostic procedures
  • 0 views
  • 15 Jun, 2022
  • 1 location
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

fibrosis
pulse oximetry
oximetry
  • 0 views
  • 22 Mar, 2022
  • 1 location
Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis

The purpose of this pilot study is to explore wither ivacaftor in refractory CRS patients will demonstrate safety and tolerability; restore CFTR-mediated Cl- secretions as measured by EDSPD testing; produce detectable improvements in validated measures of CRS including the SNOT-22 questionnaire, Lund-MacKay CT scan grading, and Lund-Kennedy endoscopic scores; and …

rhinosinusitis
ct scan
ivacaftor
cavities
  • 6 views
  • 30 May, 2022
  • 1 location
Rare CFTR Mutation Cell Collection Protocol (RARE)

codons CF patients with two mutations in the CFTR gene: i. One allele must be a F508del ii. The other allele must be a pre-mature stop codon mutation CF patients with two mutations in

fibrosis
cystic fibrosis transmembrane conductance regulator
nasal potential difference
sweat test
cftr gene
  • 27 views
  • 08 Dec, 2021
  • 7 locations
Correction of Nonsense Mutations in Cystic Fibrosis

correction of nonsense mutations of the CFTR gene in cystic fibrosis in a targeted way for a patient.

fibrosis
nonsense mutation
cftr gene
  • 7 views
  • 14 Feb, 2022
  • 6 locations
Performance and Patient Acceptance of a Commercially Available Beverage as Compared to an Oral Glucose Solution for Oral Glucose Tolerance Tests in Cystic Fibrosis (CF) Patients Who Are Screened for CF-related Diabetes.

Rationale: Cystic fibrosis (CF) is an incurable genetic disease that affects the pulmonary system, digestive system, reproductive system and the sweat glands. 85 percent of patients with cystic fibrosis have pancreatic insufficiency, more than half of whom will develop CFRD. CFRD affects patients nutritional state and is associated with a …

  • 0 views
  • 25 May, 2022
  • 1 location