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221 active trials for Cystic Fibrosis

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.

Start: December 2018

Use of Capnography for the Follow-up of Cystic Fibrosis Children

The respiratory system involvement in cystic fibrosis(CF) influences the prognosis and course of disease. Respiratory assessment is based on spirometry, but its main parameter, the maximal expiratory volume in the first second (FEV1), does not reflect the initial peripheral impairment of airways. Another pulmonary function test (PFT) validated for CF children follow-up is measurement of "gas trapping", reflecting ventilation inhomogeneity and incipient airways impairment. "Gas trapping" can be obtained by lung volume measurement (functional residual capacity, FRC) by plethysmography and helium dilution technique, but these tests are inconvenient for children due to their long length (~30min). A complete PFT is routinely performed once a year. It also includes a measurement of Lung Clearance Index (LCI) reflecting ventilation inhomogeneity. Capnography is a non-invasive PFT technique, does not require subject's active cooperation, is of short duration and could replace the traditional PFT for CF children follow-up. The capnograph is integrated into the device measuring LCI and data can be retrieved and analyzed afterwards. Capnographic indices reflect ventilation inhomogeneity. The hypothesis is that capnographic indices change in the presence/absence of "gas trapping" in CF children. The main objective is to show that the capnographic index of efficacy (EFFi) is significantly different between CF children "with gas trapping" and CF children "without gas trapping". The secondary objectives are: to compare the other capnographic indices between CF children "with gas trapping" and CF children "without gas trapping": the slope of the ascending phase, ?; the slope of the alveolar plateau, ?; the angle Q between ? and ?; the positive peak of the first-order derivative, F'CO2, which reflects the ascending phase; the first negative peak of the 2nd order derivative, F "CO2, which reflects the curvature between the ascending phase and the alveolar plateau. to compare the results of the capnographic indices with the results of the FEV1 in identifying the presence / absence of "gas trapping"; to compare the results of the capnographic indices with the results of the LCI in identifying the presence / absence of "gas trapping"

Start: April 2021

Vitamin D and Prebiotics for Intestinal Health in Cystic Fibrosis

The study will assess if administration of high-dose vitamin D and a commonly used prebiotic (inulin) is effective to reduce gastrointestinal dysbiosis and to improve critical intestinal functions in Cystic Fibrosis with the additive or synergistic effects of the combination of vitamin D + inulin.

Start: March 2020

Project 2 Airway Potential Hydrogen (pH) in Asthma

This study is testing a non invasive way to measure airway pH in individuals with Asthma and Cystic Fibrosis using a new inhaled drug. The airway pH will help health care providers in creating tailored treatment plans for individuals suffering from these specific conditions.

Start: November 2018

Phase 2 Combination Study With Escalating Doses of MS1819-SD on Top of a Stable Dose of PPEs

This is a Phase 2 study sponsored by AzurRx SAS and involves testing of a new medication for the compensation of exocrine pancreatic insufficiency (EPI) caused by cystic fibrosis (CF). The new medication is called MS1819 spray dried (MS1819-SD) which is a lipase produced by the Lip2 gene of Yarrowia lipolytica using recombinant DNA technology. The primary purpose of this study is to investigate the efficacy and safety of escalating doses of study drug on top of a stable dose of PPEs in CF patients who are not fully compensated by PPEs only. This enzyme has demonstrated an appropriate profile to compensate the pancreatic lipase (enzyme) deficiency that is common in CP (chronic pancreatitis) and CF patients. The design of the study is open-label, meaning that all eligible patients will receive the study drug MS1819-SD. The study drug dose will increase throughout the study during dose escalation visits in each treatment period; study includes a total of three treatment periods. The total duration of the MS1819-SD treatment phase is of 39-51 days. The total duration of patient participation in the study is of 69-81 days. Approximately 24 patients will be enrolled in this study.

Start: June 2019

Study to Evaluate the Safety of CB-280 in Patients With Cystic Fibrosis

This is a phase 1b multiple ascending dose escalation study to evaluate the safety and tolerability of arginase inhibitor CB-280 in subjects with cystic fibrosis.

Start: July 2020

Effects of SIMEOX on Airway Clearance in Cystic Fibrosis

This study will investigate the contribution of SIMEOX technology on the effectiveness of bronchial drainage. This is a crossover study to evaluate the contribution of SIMEOX on the effectiveness of bronchial drainage (verified by the amount of sputum secretions, the rheology of sputum secretions and the subjective sensation of ease of sputum) in patients with cystic fibrosis. Patients will perform, in randomized order (1) a 30-min session of autogenic drainage, (2) a 30-min session of autogenic drainage with the SIMEOX device. Sputum will be collected during and after the session. The two sessions will be performed with minimum washout time of 24 hours.

Start: November 2019

Role of Toilet Bronchoscopy in RICU

Toilet bronchoscopy is a potentially therapeutic intervention to aspirate retained secretions within the endotracheal tube and airways and revert atelectasis. Aspiration of airway secretions is the most common indication to perform a therapeutic bronchoscopy in the intensive care unit (ICU) . Toilet bronchoscopy is particularly beneficial when retained secretions are visible during the procedure and when air-bronchograms are not present at the chest radiograph. It is also beneficial when there is an indication to reverse lobar atelectasis, rather than simply to remove accumulated mucus. Toilet bronchoscopy is used in lobar and complete lung collapse in mechanically ventilated patients who fail to respond to treatments such as physiotherapy or recruitment manoeuvres. The success rates (defined as radiographic improvement on chest X-ray [CXR] or an improved PaO2/PAO2 ratio) in the ICU patient population had. Patients with acute hypoxaemic respiratory failure may already be on non-invasive ventilation (NIV), or require NIV preemptively for Fiberoptic Bronchoscopy (FB). These patients should be considered high risk for requiring intubation post-procedure; therefore, Fiberoptic Bronchoscopy should be performed by an experienced operator in a setting allowing facilities to safely secure the airways. NIV with early therapeutic FB rather than mechanical ventilation can help avoid intubation and reduce tracheostomy rate. Hospital mortality, duration of ventilation, and hospital stay remain similar

Start: July 2021

Real World Clinical Outcomes With Novel Modulator Therapy Combinations in People With CF (RECOVER)

RECOVER is a prospective, multicenter observational study designed to measure the real world clinical effectiveness of elexacaftor, tezacaftor and ivacaftor triple combination therapy (Kaftrio) in people with cystic fibrosis over a two year period. Measured outcomes include measures of lung function, lung inflammation, lung imaging, abdominal symptoms, gut inflammation, liver function, pancreatic exocrine function, nasal inflammation, quality of life and adherence to therapy. The study will examine outcomes in children aged six years and above over a period of two years. The first phase of the study will commence in 2020, recruiting children 12 years and older who have started on clinical treatment with Kaftrio.

Start: September 2020

Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis)

We propose to test the effectiveness of the combination of CF pancreatic enzyme replacement therapy (PERT) on absorption of Ravicti® and subsequent restoration of nasal epithelial cystic fibrosis transmembrane conductance regulator (CFTR)-mediated chloride transport during the nasal potential difference (NPD) test. Funding source FDA Office of Orphan Products Development.

Start: December 2018

Impact of Bronchial Drainage Technique by the Medical Device Simeox® on Respiratory Function and Symptoms in Adult Patients With Cystic Fibrosis

The main objective of the study is to evaluate the short-term effects of the airway clearance technique by the medical device Simeox (Physio Assist, France) versus Autogenic Drainage, on the pulmonary function of adult patients with stable cystic fibrosis. To analyse these effects, the investigators used forced oscillation technique measured by TremoFlo™ C-100 Airwave Oscillometry System™ (THORASYS Thoracic Medical Systems Inc. Montreal, Quebec, Canada) with an evaluation of the perceived clinical benefits.

Start: September 2019

Tele-Coaching Intervention to Improve Treatment Adherence in Cystic Fibrosis

This is a prospective, multicenter pilot study to investigate the feasibility and preliminary effectiveness of a tailored tele-coaching intervention to enhance medical adherence in patients with CF.

Start: January 2020

Impact of Triple Combination CFTR Therapy on Sinus Disease.

The study's main goal is to observe how effective elexacaftor-tezacaftor-ivacaftor is for improving the symptoms and signs of CF-related sinus disease.

Start: September 2019

Assessment of CFTR-Modulator Treatment in Cystic Fibrosis Lung Disease Using Novel Structural and Functional MRI

In this study, MRI of the lungs of healthy volunteers and participants with cystic fibrosis (stable and participants initiating CFTR modulator treatment) will be performed over a period of 6 months to determine if lung MRI is able detect structural and functional abnormalities/changes in early cystic fibrosis disease. During the 6 month period, 3 study visits will occur. 70 subjects aged 6 and older will participate in this study. Xenon MRI is a non-invasive imaging technique that does not involve x-rays or ionizing radiation. Rather, this imaging method utilizes the same hardware and software principles that are used for conventional proton MRI of patients in a hospital.

Start: October 2020

Regional Phenotyping of CF and Non-CF Bronchiectasis

The Investigators propose to study pediatric subjects who are diagnosed with cystic fibrosis (CF) and patients with non-CF bronchiectasis, with the goal of developing markers of CF lung disease severity, progression, and therapy response. The Investigator's central hypothesis is that image-based markers can forecast pathophysiology prior to spirometric changes.

Start: March 2021

Impact of Sinus Surgery on Individuals With Cystic Fibrosis

This study will be a prospective, observational study of patients who undergo endoscopic sinus surgery for cystic fibrosis-related chronic rhinosinusitis (CRS). Individuals who do not undergo surgery but are treated medically for CRS will also be enrolled to serve as a control group. Outcomes analyzed will include pulmonary, quality of life, and others.

Start: July 2020

Inhaled Sodium Nitrite as an Antimicrobial for Cystic Fibrosis

This study will assess the safety of inhaled sodium nitrite in adults with Cystic Fibrosis and chronic Pseudomonas infections, and determine the ability of sodium nitrite to reduce the burden of Pseudomonas.

Start: February 2016

Genomic Resources for Enhancing Available Therapies (GREAT1.0) Study

This is a prospective, descriptive, observational research study designed to observe and document the clinical practice by domain experts, and how the knowledge of new findings that are published in the medical literature affect clinical decision making. The study will evaluate risk factors and co-variants, including genetic variants that are associated with disease progression such as pain, inflammation, organ dysfunction, disability and quality of life.

Start: November 2014

Multilevel Models of Therapeutic Response in the Lungs

When developing new medications for lung diseases like Cystic Fibrosis (CF), scientists perform lab experiments using cells from the airways, physiology studies of how the lungs change when a drug is given, and clinical studies to determine how drugs affect overall health. The investigators of this study are seeking to develop computer models that will predict how patients will respond to drugs by just doing lab studies on cell samples from their noses. Such models would allow for medications to be developed more rapidly for all patients and allow treatments to be personalized as well. In order to develop these computer models a series of tests will be performed on patients who have CF. Tests will include sampling cells from the nose and measuring lung physiology using a combination of different imaging, breathing, and other studies performed both before and after participants take a therapy. Similar tests will be performed on people who do not have CF, and on the parents of the CF participants who carry a single CF gene because this will provide information on how specific genes might affect CF lung disease.

Start: January 2017

Pharmacokinetics of Omadacycline in Cystic Fibrosis

The purpose of this study is to characterize the pharmacokinetics of intravenous and oral omadacycline in patients with cystic fibrosis.

Start: July 2021