Active clinical trials for "Cystic Fibrosis"

Regular bronchial clearance is essential in patients with cystic fibrosis for their bronchial health. SIMEOX® (Physio-Assist, Aix en Provence, France) is an innovative medical device for the drainage of the bronchial tree. By changing the rheological properties of mucus, SIMEOX® helps to mobilize secretions and assists their transport to the upper airways. This technology is based on fundamental research on bronchial mucus rheology. At the present time, SIMEOX® device is mainly used over a short period at the time or after an exacerbation in healthcare structures (hospitals, physiotherapy practices, post-care, and rehabilitation units, etc…). The clinical effects observed in the short term encourages long-term autonomous use by the patients themselves. The overall objective of this study is to evaluate the efficiency and acceptability of SIMEOX® used at home by the patient himself for bronchial clearance in patients with cystic fibrosis.

This study will provide: (1) new insights in the prevalence of Aspergillus infection in children and adolescents with CF aged 8-17 yrs; (2) an in silico modelled dose of posaconazole for children and adolescents with CF and Aspergillus infection aged 8-17 yrs; (3) an intensive sampling PK study to define the optimal dose in a limited number of children and adolescents with CF and Aspergillus infection aged 8-17 yrs; (4) a prospective clinical validation to reduce the residual variability and to allow investigation into PK-PD; and (5) an efficacy evaluation of this dosing regimen to treat Aspergillus infection in children and adolescents with CF to inform future primary efficacy trials.

This open label, single arm pilot study, will examine the safety and tolerability of GLP-1RA semaglutide as an add-on therapy to insulin for overweight/obese adult patients with cystic fibrosis related diabetes (CFRD).

The goal of this study is to pilot a new CF-specific cognitive-behavioral therapy intervention (CF-CBT-A) for prevention and treatment of depression and anxiety for adolescents with CF. CF-CBT-A is a 10-session program that was developed with input from adolescents with CF and parents and CF care teams to be highly relevant to the unique needs of adolescents with CF. The program will be piloted at 3 U.S. CF centers with 10 to 12 adolescents with cystic fibrosis who have mild to moderately severe symptoms of depression and/or mild to severe symptoms of anxiety. It will be delivered by mental health coordinator members of the participant's CF care team who receive training, with sessions occurring in-person or via telehealth. We will examine feasibility and acceptability of the intervention as indicated by measures of completion, intervention fidelity, and adolescent and parent satisfaction ratings. We will also examine preliminary evidence of effectiveness. If this intervention is successful, symptoms of depression and anxiety and perceived psychological stress will decrease and coping self-efficacy and health-related quality of life (HRQoL) will improve.

The goal of this clinical trial is to learn about the role of estrogen in bone development in adolescent and young adult women with cystic fibrosis (CF) and low estrogen levels. The main questions it aims to answer are: Is a study of transdermal estradiol (estrogen skin patches) feasible in this group? How does transdermal estradiol impact bone health and quality of life? Participants will apply transdermal estradiol patches (or placebo patches -- patches that look like the estradiol patches but do not contain any estradiol) once weekly for 12 months, fill out brief quality-of-life surveys twice per month, and complete blood draws and questionnaires at four study visits (baseline, 3 months, 6 months, and 12 months). The participants will also have x-rays and DXAs (bone density assessment) performed at the baseline and 12 month visits. Researchers will compare changes in various measurements between the treatment and placebo groups to determine the impact of transdermal estradiol.

Some cystic fibrosis patients are unable to digest food and absorb nutrition appropriately as they have a condition known as exocrine pancreatic insufficiency (EPI). Currently, these patients take pancreatic enzymes that are obtained from pig pancreas to aid the digestion of food. The goals of this clinical study are to evaluate the safety and efficacy of a novel formulation of a non-porcine lipase, called adrulipase, in patients with EPI due to cystic fibrosis. The main question[s] the study aims to answer are: Is the novel formulation of adrulipase safe to use at the doses being evaluated in the clinical study. Is adrulipase as effective, or more effective, compared to the pig enzymes the patients currently use. Researchers will compare the results obtained with adrulipase to how the patients typically respond to their pig enzymes to see if adrulipase helps patients digest fats adequately and if their stomach feels good (signs and symptoms of malabsorption).

The goal of this clinical trial is to investigate the effects of a-12-week virtual reality exercise program, applied as tele-exercise, on the exercise capacity of children with cystic fibrosis, whose physical activity level is further restricted during the pandemic. The main questions it aims to answer are: Does aerobic tele-exercise increase functional capacity in cystic fibrosis patients? Will there be a difference in treatment outcomes between the virtual reality group and the online exercise group? Participants will be randomized into two groups; virtual reality group and online group. Physical activity will be provided to the virtual reality group in the form of team activities in a virtual environment, and the other group will be provided online exercises at their home. For this purpose, virtual reality headsets will be provided to patients to give the impression of a natural, immersive environment and realistic experience. Researchers will compare exercise compliance and functional capacity between the virtual reality group and the online group.

The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with CF who are ineligible for or unable to tolerate CFTR modulator therapy.

Aim of the randomized controlled trial is to investigate the effects of endurance training with different intensities on physical performance, body composition and appetite regulation in people with cystic fibrosis.