Active clinical trials for "Cystic Fibrosis"

Due to the hypobaric hypoxia during air travel, some patients with Cystic Fibrosis (CF) need supplemental oxygen during air travel. The study aims to evaluate if adding exercise desaturation to the pre-flight evaluation will help predict requirements for in-flight supplemental oxygen.

The objective of this study is to evaluate the predictive nature of the biomarker Porphyromonas catoniae measured at the age of 12 months in the occurrence of colonization with Pseudomonas aeruginosa at 36 months of age in children with cystic fibrosis.

Goal is to physiologically detect unsuspected small airways obstruction in children and adults with treated heterozygous and homozygous cystic fibrosis. Unsuspected refers to normal routine pre bronchodilator spirometry including normal FEV1(L), FVC (L). and FEV1/FVC%. This is a retrospective study.

This study relies on the hypotheses that (1) exhaled breath is intimately correlated to the patient's lung condition and that (2)the composition of exhaled breath , i.e. the VOCs profile, will be significantly modified from the first days of treatment by CFTR modulators in a or pauci/symptomatic patients such as young children under 12 years old. The non-invasive and longitudinal collection and analysis of exhaled breath may reveal modifications in signaling pathways impacted by these treatments on the very short term. This study is a single-center pilot study.

This multi-center study will compare multi-target DNA and quantitative FIT stool-based testing to colonoscopy in individuals with Cystic Fibrosis (CF) undergoing colon cancer screening with colonoscopy. The primary endpoint is detection of any adenomas, including advanced adenomas and colorectal cancer (CRC).

The aim of this study is to determine the relationship between diets of children with cystic fibrosis and glycemic responses and some inflammatory markers.

To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and preventative methods for specific and complex conditions.

There are many techniques that can allow for the quantification of lung function in children; some are being used clinically and others are under development. Many of these tools are available at BC Children's Hospital. This registry study will act as a central repository for the results of traditional and novel pulmonary function tests done at BC Children's Hospital to allow for future analysis.

Cystic fibrosis is a multi-organ disease. It most often results from a genetic mutation, the delta F508 mutation, which prevents the expression of the CFTR 'régulateur de conductance transmembranaire de la fibrose kystique) protein. If the poor prognosis of the disease is correlated to the pulmonary damage, we observe, at the naso-sinus level, a significant functional impact, with chronic rhino-sinusal damage that can alter the quality of life of patients. In addition to this functional impact, some studies suggest that these chronic naso-sinus attacks are involved in the creation of a bacterial reservoir that is secondarily responsible for pulmonary colonization and therefore partly responsible for the poor prognosis of the disease. The clinical and paraclinical examinations can be used to determine the extent of these disorders. Their functional impact can be assessed using quality of life questionnaires such as the SN-5 scale. Treatment with CFTR modulators in patients with mutations compatible with the treatment seems to transform their vital prognosis. The scientific rationale of this treatment consists in restoring the activity of the CFTR protein, allowing the recovery of the hydro-electrolytic balance of the mucous secretions, and thus reducing the viscosity of the biological fluids. The various studies carried out all prove a dramatic improvement in pulmonary parameters under treatment, with very limited toxicity. A marketing authorization for this treatment has been issued on the European market for patients over 18 years of age in 2020, for children over 12 years of age in 2021, and will soon be issued for children aged between 6 and 11 years. Since the pathophysiology of pulmonary and nasosinus involvement are similar, and since this treatment will be marketed for children between 6 and 11 years of age, we expect an improvement in rhino-sinus symptomatology. To date, clinical studies have focused primarily on pulmonary outcomes. There are only few publications dealing with the evolution of nasosinus symptomatology under treatment, and none concerning the pediatric population. The aim of our study is to evaluate the evolution of naso-sinusal symptomatology under treatment with CFTR modulators in children aged 6 to 11 years. This will allow us to confirm or deny the interest of these treatments in the extra-pulmonary manifestations of the disease.

Background: - Noncirrhotic Portal Hypertension (NCPH) is caused by liver diseases that increase pressure in the blood vessels of the liver. It seems to start slowly and not have many warning signs. Many people may not even know that they have a liver disease. There are no specific treatments for NCPH. Objectives: - To learn more about how NCPH develops over time. Eligibility: - People age 12 and older who have NCPH or are at risk for getting it. In the past year, they cannot have had other types of liver disease that typically result in cirrhosis, liver cancer, or active substance abuse. Design: Participants will have 2 screening visits. Visit 1: to see if they have or may develop NCPH. Medical history Physical exam Urine and stool studies Abdominal ultrasound Fibroscan. Sound waves measure liver stiffness. <TAB>- Visit 2: Blood tests Abdominal MRI Echocardiogram Questionnaire Liver blood vessel pressure (hepatic venous portal gradient (HVPG)) measurement. This is done with a small tube inserted in a neck vein. They may have a liver biopsy. All participants will visit the clinic every 6 months for a history, physical exam, and blood tests. They will also repeat some of the screening tests yearly. Participants with NCPH will also have: Upper endoscopy test. A tube inserted in the mouth goes through the esophagus and stomach. At least every 2 years: Esophagogastroduodenoscopy. At least every 4 years: testing including HVPG measurements and liver biopsy. Participants without NCPH will also have: Liver biopsy and HVPG measurements to see if they have NCPH. Every 2 years: abdominal MRI and stool studies. The study will last indefinitely.