View clinical trials related to Cystic Fibrosis.
Filter by:This is a first in human study of ETD001 a new drug being developed for the treatment of cystic fibrosis. The study is a randomised, placebo-controlled, double-blind interventional study to assess the safety, tolerability and pharmacokinetics of single and multiple ascending doses of inhaled ETD001in healthy male and female subjects.
Five Cystic Fibrosis (CF) centers, key stakeholders, and a palliative care institute have collaborated to create a novel primary palliative care intervention for patients with CF, "Improving Life with CF: A Primary Palliative Care Partnership," and established the infrastructure and support necessary for a follow-on implementation study. This intervention provides a framework for a nationally generalizable model to improve best practices in generalist-level palliative care in CF. Objectives: Aim 1: Implement a primary palliative care intervention comprising screening-and-triage workflows, best practice treatment guides for high frequency problems, patient/family and provider education, and a quality improvement (QI) toolkit. Aim 2: Evaluate feasibility, uptake, and preliminary outcomes during a multisite pragmatic, implementation trial of the intervention at 5 diverse Cystic Fibrosis (CF) Centers. Subaim 2.1: Evaluate feasibility and uptake as measured by rates of screening and treatment delivery. Hypothesis 1: Related to feasibility and uptake of the intervention: 1. > 80% of individuals with CF of all ages will receive an annual palliative care screening. 2. > 25% of individuals with CF will receive a palliative care screening prompted by hospitalization, new diagnosis of CF-Related Diabetes, need for transplantation, or another disease- or treatment-specific trigger. Hypothesis 2: Related to provider education: a) > 80% will access >1 training(s) (on-demand webinars or in-service by trained site educators). Subaim 2.2: Evaluate data on preliminary outcomes for individuals with CF by comparing ratings on patient- and caregiver-reported outcome measures (e.g., Integrated Palliative Care Outcome Scale, Cystic Fibrosis Questionnaire-Revised, Memorial Symptom Assessment Scale-Cystic Fibrosis, and Brief Assessment Scale for Caregivers) during the trial to baseline ratings and exploring covariates of change (age, race/ethnicity, gender, disease severity, CFTR modifier treatment, psychological distress, and varied indicators reflecting intervention implementation).
This study will evaluate the performance of wearable technology in cystic fibrosis (CF) participants taking commercial Elexacaftor (ELX)/Tezacaftor (TEZ)/Ivacaftor (IVA) utilizing a fully decentralized trial design.
Evaluate the feasibility of a randomized controlled trial of non-surgical urinary incontinence (UI) management options for women with CF. The investigators will complete a pilot, feasibility study (n=30) to compare tolerability and symptom relief in women with CF and UI. Subjects will be recruited from the University of Pittsburgh Cystic Fibrosis Center after demonstrating bother from UI on initial phone script. Participants will undergo UI questionnaires and undergo a pelvic examination, non-invasive bladder scan ultrasound and a provocative stress test and then be randomized to either a disposable urethral support device (Impressa®), an absorbent product (Speax Reusable Underwear), or Pelvic floor muscle therapy. The primary outcome will be to determine the feasibility and tolerability of these options. Hypothesis: All three non-surgical UI management options for women are feasible (as measured by 80% adherence to treatment assignment over 7 days) and tolerable (as measured by patient report via questionnaire). The results from the proposed aims will provide important information about the experiences and symptom burden of women with CF and UI. Importantly, the investigators will also be able to answer the important questions of "Can it work?" and "Does it work?" as the investigators seek to construct the definitive, adequately powered trial of these therapies in women with CF and UI.
This is a project that will determine whether the use of daily bright light therapy has an effect on depressive symptoms experienced by adult inpatients with CF and COPD. The purpose of this project is to apply a daily 30-minute BLT intervention to hospitalized adult CF and COPD patients in order to decrease symptoms of depression as measured by depression inventory scoring.
Coronavirus disease 2019 (COVID-19) which is caused by the virus SARS-CoV-2 has resulted in an ongoing global pandemic. It is unclear whether the relatively low number of reported cases of COVID-19 in people with CF (pwCF) is due to enhanced infection prevention practices or whether pwCF have protective genetic/immune factors. This study aims to prospectively assess the proportion of pwCF, including both adults and children with CF who have evidence of SARS-CoV-2 antibodies over a two-year period. This study will also examine whether pwCF who have antibodies for SARS-CoV-2 have a different clinical presentation and what impact this has on their CF disease. The proposed study will recruit pwCF from paediatric and adult CF centres throughout the United Kingdom. Serological testing to detect antibodies will be performed on blood samples taken at month 0, 6, 12, 18 and 24 with additional time-points if bloodwork is available via normal clinical care. Clinical data on, lung function, CF-related medical history, pulmonary exacerbations, antibiotic use, and microbiology and vaccination receipt, will be collected during routine clinical assessments. Associations will be examined between socio-demographic and clinical variables and serologic testing. The effects of SARS-CoV-2 infection on clinical outcomes and analyse end-points will be examined to explore any age-related or gender-based differences, as well as subgroup analysis of outcomes in lung-transplant recipients and pwCF receiving CFTR modulator therapies. As pwCF receive COVID-19 vaccination a comparison of the development and progression of anti-SARS-CoV-2 antibodies in pwCF following natural infection and vaccination SARS-CoV-2 over time will be performed.
31 CF patients and 23 healthy sex and age-matched volunteers were enrolled. All subjects underwent pulmonary function tests, 6-minute walking tests. Body Mass Index and Fat Free Mass Index were measured via a body composition analyser. Diaphragm ultrasonography (to measure thickening fraction), quadriceps femoris muscle ultrasonography were performed. A dynamometer was used to measure each subjects handgrip strength.
Diabetes affects half of cystic fibrosis patients aged 30 years and older. It develops asymptomatically for a long time. Also, two options are possible: start insulin treatment now with the additional constraints associated with cystic fibrosis or wait while monitoring the patient's clinical status and initiate insulin treatment when he has developed symptoms and therefore later. In practice, the choice between these two options takes place over two medical consultations without a formalized shared decision-making process between the doctor and the patient. Shared decision-making is a decision-making process in which the healthcare provider and the patient learn about patients care options and then deliberate to reach a common agreement on the decision taken. Shared decision-making seemed particularly relevant to us in cystic fibrosis where there are complex treatment options with variable short-, medium- and long-term side effects and where the disease and its treatments have a high impact on the patient's quality of life.
This study compare an usual training program (in continuous endurance) with an ITHI program over a period of 3 weeks, in France. The aim is to evaluate the safety and feasibility of this type of program in CF adults, and also more specifically, in subgroups: patients divided according to the severity of their FEV1 ; patients treated with modulating CFTR canal therapy ; diabetic patients on insulin ; undernourished patients.
This is pilot study of the immunologic effects of intradermal Bacille Calmette-Guerin (BCG) vaccination of adults with cystic fibrosis (CF), non-CF bronchiectasis (NCFB), and healthy volunteers.