View clinical trials related to Cystic Fibrosis.
Filter by:This clinical study will enroll 42 participants without the F508del mutation, carrying partial function or N1303K mutations not approved for Trikafta, and who are not expected to be approved for CFTR modulator treatment in the immediate future. Each participant will be given Trikafta for approximately four weeks. The study researchers will monitor clinical endpoints that include forced expiratory volume (FEV1) and sweat chloride. Additionally, the researchers will obtain skin biopsy material and/or blood sample from each subject so that induced pluripotent stem (iPS) cells can be modified into airway cell monolayers and tested for response to Trikafta. In this way, the study will evaluate an emerging and readily accessible in vitro endpoint as a predictor of clinical response. This study will serve as a pilot/test case for other clinical protocols relevant to patients with rare CFTR variants who do not currently receive modulator therapies. It is hypothesized that a robust correlation will be established between in vitro Trikafta responsiveness of iPS cells and in vivo benefit (FEV1) to patients, and will provide a new tool for utilizing iPS to identify patient populations most suitable for cystic fibrosis modulator therapy.
The purpose of this study is to validate the user interface of TOBI Podhaler by establishing that the product can support safe and effective use for the intended users
Background/Rationale Acute rejection (AR) is common in the first year after lung transplantation. AR has usually been reversible with treatment, but it can trigger chronic rejection that is the leading causes of late morbidity and mortality. Extracorporeal photopheresis (ECP) has emerged as a promising treatment for chronic rejection. The investigators postulate that the immunoregulatory property of ECP could promote graft tolerance immediately after lung transplantation. Objectives The aim of this trial is to evaluate the safety and efficacy of ECP as induction therapy for prevention of AR in recipients affected with cystic fibrosis in the first year after lung transplantation. The extracellular vesicles in the cell-to-cell communication and immunomodulation will be also investigated. Preliminary results (personal) A preliminary study, conducted in Vienna, demonstrated that 9 patients treated with ECP as induction therapy had 0% of chronic rejection versus 50% in the control group. The Institution hosting the current project is among largest lung transplantation centers in Italy with high rate of cystic fibrosis recipients. The Institution has experience in ECP and a dedicated instrument was specifically bought for the project. Internal collaborators have strong expertise in biological aspects including the extracellular vesicle compartment.
The study is a randomized, double-blind, placebo-controlled, study that will be conducted at multiple centers in subjects with Cystic Fibrosis (CF) who are either homozygous for the F508del mutation or heterozygous with at least copy of the F508del mutation.
The purpose of this study is to use perfluorinated gas imaging to highlight regions of functional variation within the lungs of participants with cystic fibrosis (CF), and to correlate this with changes in spirometry, lung clearance index, and quality of life of CF subjects undergoing treatment for a pulmonary exacerbation.
Cytobacteriological examination of sputum and bacteriological sampling in the middle meatus.
The aim of the study is to look for risk factors of developing osteoporosis in patients with cystic fibrosis. Blood cells called monocytes will be obtained from a blood sample. These monocytes will make possible to form another class of cells called osteoclasts in the laboratory. These latter cells are responsible for bone degradation and therefore promote the increase of bone fragility. The research will focus on the characterization of these cells to better understand their potential (low, medium or high) to increase bone fragility in patients with cystic fibrosis. Finally the action of CFTR modulators on these bone cells will be tested.
To evaluate the micro-biologic efficacy and safety of a streamlined treatment for early onset methicillin-resistant staphylococcus aureus (MRSA) in patients with cystic fibrosis.
An open label non-randomized study enrolling up to 20 healthy participants and up to 30 participants with cystic fibrosis to establish a healthy versus disease comparison. Each participant will receive a mixture of inert gas (perfluoropropane (PFP)) in a ratio of 79% PFP to 21% oxygen as a contrast agent to enhance visualization of the airway and alveolar spaces using magnetic resonance imaging of inert gas/oxygen mixtures. The study consists of a screening visit followed by up to 2 study visits.
This study evaluated the safety and pharmacokinetics of multiple ascending doses of VX-440 in combination with tezacaftor/ivacaftor (TEZ/IVA) (triple combination [TC]) administered for 13 days to healthy male and female subjects