724 Clinical Trials for Various Conditions
This study is designed to determine the pharmacokinetic and pharmacodynamic response of dorzagliatin 75 mg twice daily following 7-day administration in individuals with pancreatic insufficient cystic fibrosis and abnormal glucose tolerance when compared to randomized, double-blind 7-day administration of placebo in a cross-over fashion. We hypothesize that dorzagliatin administration will result in significant drug concentrations and improved glucose tolerance, early-phase insulin secretion, glucagon suppression, and hepatic glycogen storage assessed during a standardized mixed-meal tolerance test.
Pancreatic Insufficiency, Cystic Fibrosis-related Diabetes
Elexacaftor/Tezacaftor/Ivacaftor or Trikafta improves lung health in people with cystic fibrosis (CF), including decreased cough and mucous production. Diagnosing lung infections has become more challenging due to the decrease in sputum and rise of telehealth services. While the option of collecting sputum samples at home and sending them by mail may be feasible, uncertainty remains about how the collection of samples outside of clinic and delays in testing while in the mail impact infection detection. This study will compare bacterial cultures using sputum samples collected at home versus samples collected in clinic (saline-induced sputum and throat swab). This study seeks to shed light on how valuable home collected samples can be and help us better understand the usefulness of home-collected sputum samples for both clinical and research purposes.
Cystic Fibrosis, Infections
ARCT-032-02 is a Phase 2, open-label, multicenter, multiple-ascending dose study of ARCT-032 in adults with CF who are not eligible for CFTR modulator therapy or are not taking CFTR modulators due to drug intolerance, poor response, or lack of access to modulators.
Cystic Fibrosis, CFTR Gene Mutation
The goal of this at-home, remote study is to understand how tube feeding affects blood sugars and sleep in adults living with Cystic Fibrosis Related Diabetes (CFRD).
Cystic Fibrosis Related Diabetes, Hyperglycaemia, Insomnia, Cardiometabolic Diseases, Circadian Rhythm Disorders
The primary objective of this pilot study is to develop a CGM-based model to predict the progression from prediabetes to diabetes in individuals with cystic fibrosis.
Prediabetes, Cystic Fibrosis (CF), Cystic Fibrosis-related Diabetes
The purpose of this study is to use a survey to measure reflux symptoms in patients with Cystic Fibrosis who are on reflux treatment, and to monitor their symptoms after stopping the reflux treatment.
GERD in Children, Cystic Fibrosis in Children
The goal of this clinical trial is to help adolescents and young adults between the ages of 13-25 with Cystic Fibrosis (CF), medically stable, able to speak and read English, and are not experiencing a CF - related exacerbation, who are already active to remain, or gradually encourage them to increase their levels of physical activity Participants will be asked to utilize a smartphone program, called NUDGE that we have developed. NUDGE is a chatbot with evidence-based features known to help teens make progress toward health goal: * Set and review goals * Self-monitor progress * Provide feedback on goal attainment * Revise future goals
Cystic Fibrosis (CF)
Many people living with cystic fibrosis (CF) experience pain. However, pain is sometimes unrecognized and under-managed in people with CF. Both medication and non-medication interventions can be used to treat pain and ideally, there is a multicomponent management approach. The goal of this study is to pilot a new 3-session non-medication mind-body pain management intervention specifically designed for adults with cystic fibrosis (CF). The intervention is titled Pain Acknowledgement Coping and Empowerment in CF (PACE CF). PACE CF will be administered by a member of the CF care team via telehealth visits to participants at home or during a hospitalization. The aim of the study is to evaluate the feasibility and patient acceptability of the intervention as well as preliminary evidence of the impact of the intervention on pain and its interference in daily life. The study will also examine outcomes such as perceived coping, quality of life, symptoms of depression or anxiety, and use of prescribed pain medication, in an exploratory manner.
Cystic Fibrosis (CF), Pain Management
The purpose of this study is to find out what causes kidney disease in people with CF. The investigators will study biomarkers in the blood and urine that can either predict who is at risk or detect kidney damage early before it becomes permanent. The study will compare these markers in people with CF over time and during the treatment of lung flare-ups. It will also compare the blood and urine samples obtained from people without CF. The comparison aims to better understand the impact of cystic fibrosis and its treatment on the kidneys, as well as to develop improved methods for preventing, diagnosing, and treating kidney issues associated with CF.
Cystic Fibrosis (CF), Chronic Kidney Disease(CKD), Acute Kidney Injury
Cystic Fibrosis (CF) related diabetes (CFRD) is a unique form of diabetes mellitus, different from type 1 diabetes and type 2 diabetes. The diagnosis of CFRD is associated with a decline in pulmonary function, decreased nutritional status, and increased mortality. CFRD is extremely common in people with CF, occurring in approximately 40-50% of adults with CF. Impaired glucose tolerance or dysglycemia is also very common in CF. It is standard of care to screen for CFRD annually from the age of 10 years with a two-hour Oral Glucose Tolerance Test (OGTT) with 75 g dextrose. The gold standard screening for CFRD is the OGTT which is problematic as it is time consuming for patient and staff and adherence to annual screening is low among CF centers. Survival has improved dramatically with the advent of CFTR modulators and it is presumed that the incidence of CFRD will increase with increased life expectancy. The Cystic Fibrosis Foundation (CFF) has developed the oldest disease specific patient registry, consisting of approximately 35000 patients, so there is vast historical information available on individual patients and larger datasets on the CF community as a whole. Based on the 2021 CFF patient registry data, the current life expectancy for CF patients born between 2017 and 2021 is 53 years - a 15 year increase from a decade ago.
Cystic Fibrosis-related Diabetes, Cystic Fibrosis
This study is a randomized, double-blind, placebo-controlled study designed to assess the efficacy and safety of ensifentrine inhalation suspension (3 mg) delivered twice daily via standard jet nebulizer over at least 24 weeks, compared to placebo, in subjects with non-cystic fibrosis bronchiectasis (NCFBE).
Non-cystic Fibrosis Bronchiectasis
The goal of this study is to investigate the prevalence of dysglycemia with continuous glucose monitoring (CGM) obtained during pulmonary exacerbations, both outpatient and inpatient, in youth with cystic fibrosis (CF).
Cystic Fibrosis Pulmonary Exacerbation
This is a Phase 1/2 multicenter, open-label, single dose trial of SP-101 investigational gene therapy in adults with CF who are ineligible for or intolerant to CFTR modulator therapy.
Cystic Fibrosis
The purpose of the study is to evaluate the natural history of exocrine pancreatic function by assessing Fecal elastase-1 (FE-1) in infants with CF during their first year of life.
Cystic Fibrosis
The goal of this clinical trial is to learn if drug SPL84 is safe for adult patients with cystic fibrosis (CF). It will also learn if the drug works to treat works to treat CF with a specific mutation. The purpose of this research study is to: * test the safety and effectiveness of multiple doses of the study drug, SPL84 * test how multiple doses of the drug are processed by the body Researchers will compare drug SPL84 to a placebo (a look-alike substance that contains no drug) to see if drug SPL84 is safe and if it works to treat CF. Participants will: Take drug SPL84 or a placebo by inhalation every week for 9 weeks months Visit the clinic approximately 14 times over 17.5 weeks for checkups and tests
Cystic Fibrosis
Chronic constipation is a feature of children with cystic fibrosis (CF). This is postulated to be a result of inhibition of secretory activity of the gastrointestinal luminal cells due to ineffective chloride channel function. Typical laxatives that work as osmotic agents fail to produce adequate relief in this population. Maralixibat is a non-systemic bile acid transport inhibitor (IBATi) that acts by interrupting bile acid reabsorption in the ileum thus interrupting the normal enterohepatic circulation. This interruption results in a larger volume of bile acids reaching the colon and being excreted in stool. Bile acids are known to decrease bowel transit time, increase mucosal permeability and secretions, as well as alter gut microbiota resulting in diarrhea. The overarching hypothesis of the study is that Maralixibat will improve stool consistency in children (Age \<18 years) with cystic fibrosis and constipation (Bristol Stool Scale \<4). Specifically, we aim to test the hypothesis that IBATi improves the consistency of stool to Bristol scale \>4 in children with CF and constipation. We will recruit a total of 20 patients with CF and constipation (defined as Bristol Stool Scale \<4 for 1 week prior to enrollment while on a stable laxative regimen for at least 4 weeks.) Design is a 'Within-Subjects' study by which each enrolled patient will take Maralixibat for 2 weeks total in addition to their stable laxative regimen during the study. Stool consistency \& ease of defecation will be recorded before and during the study period by families of enrolled patients via materials provided by the investigators. Stool consistency and ease of defecation will be compared before and after initiation of Maralixibat. The primary endpoint: Improvement in stool consistency to Bristol scale \>4 in children with CF and constipation. The secondary endpoint: Improvement in ease of defecation in children with CF and constipation. This will be measured via survey using a standardized scale (Bristol Stool Scale) and questionnaires developed by the research team. Analysis will involve comparison of pre-intervention to post-intervention stool consistency \& survey
Constipation Chronic Idiopathic, Cystic Fibrosis
The goal of this clinical trial is to test use of losartan in those with cystic fibrosis (CF) on modulator therapy. The main question it aims to answer is if treatment with losartan improves response of the CF transmembrane conductance regulator (CFTR) channel to modulator therapy. Participants will be asked take losartan or placebo for twelve weeks and will have changes in sweat chloride levels measured as a marker of CFTR function.
Cystic Fibrosis
The goal of this study is to determine the extent to which excess dietary simple sugars serve as a secondary mediating factor in Cystic fibrosis-related diabetes (CFRD) development. The main questions it aims to answer are: * Whether conducting a randomized 2x2 factorial design that evaluates acute postprandial changes in glucose over 2 hours following ingestion of a mixed meal challenge that varies by glycemic index and consumption of a sugar-sweetened beverage is acceptable and feasible. * What are the preliminary changes in postprandial hyperglycemia, islet cell function, and incretin response to a high or low Glycemic Index mixed meal tolerance test (MMTT) with and without Sugar-Sweetened Beverages (SSB) in adolescents and young adults with CF Participants will be randomized to a mixed diet and blood will be drawn before and after the mixed meal challenge.
Cystic Fibrosis
This is a small pilot study with the goal of identifying a superior sputum collection method in Cystic Fibrosis patients unable to produce a sputum. Participants will use the Volara System during clinic visit in an attempt to produce sputum.
Cystic Fibrosis
The goal of this observational prospective study is to determine the health impact of parenthood on United States (US) people with CF in the era of CF transmembrane regulator protein (CFTR) modulators. The investigators will collect physical and mental health data to comprehensively evaluate the impact of parenthood in CF with widespread highly effective CFTR modulator use. The main hypotheses this study aims to examine are: H1: Parents with CF and moderate-to-severe depression have more rapid change in ppFEV1 (percent predicted forced expiratory volume in one second) versus those with mild or no depression. H2: Parents with CF who have more parental responsibility and/or stress have more rapid ppFEV1 (percent predicted forced expiratory volume in one second) change than those with less responsibility/stress H3: Parents using CFTR modulators have decreased ppFEV1 (percent predicted forced expiratory volume in one second) change versus those not using CFTR modulators Participants will complete quarterly surveys during the first year of parenthood and biannual surveys, thereafter, using the computer-based survey system on an iPad protected for infection control or via personal device or computer via emailed survey link.
Cystic Fibrosis, Parenthood Status
ACT18018 is a multinational, randomized, double-blind, placebo-controlled, parallel-group, Phase 2 study with 3 treatment groups. The purpose of this study is to evaluate efficacy, safety and tolerability with 2 dosing regimens of itepekimab compared with placebo in male and/or female participants with NCFB aged 18 years of age up to 85 years of age (inclusive). Study details include: * The study duration (screening, 24-52-week treatment, 20-week safety follow-up) will be up to 47-77 weeks. * The treatment duration will be up to 24-52 weeks. * The follow-up duration will be 20 weeks. * Site/phone visits are at a monthly interval.
Bronchiectasis
The investigators will be evaluating bone marrow composition via magnetic resonance imaging in adolescents diagnosed with cystic fibrosis (CF) compared to healthy, matched controls. The investigators will also be assessing their bone mineral density via other imaging modalities, including dual-energy X-ray absorptiometry (DXA) and peripheral quantitative computed tomography (pQCT). This longitudinal project will focus on abnormalities in bone marrow composition, and specifically whether adolescents with diagnosed with CF exhibit increased bone marrow fat, its association with bone mineral density (BMD) and the underlying pathophysiology, including glycemic control, inflammation, and bone turnover markers.
Cystic Fibrosis
This is a prospective, observational study examining the impact of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators on chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) in young children with cystic fibrosis (YCwCF). This study involves two groups: children 2-8 years old, inclusive at initial visit, receiving highly effective modulator therapy (HEMT), and a control group of children 2-8 years old, inclusive at initial visit, not receiving HEMT. Outcomes will include sinus magnetic resonance imaging (MRI) scans, olfactory tests, and quality of life surveys obtained over a two-year period.
Cystic Fibrosis in Children, Cystic Fibrosis, Chronic Rhinosinusitis (Diagnosis), Olfactory Disorder, Olfactory Impairment
BronchConnect is a prospective trial to investigate the impact of support groups on health care related quality of life in those with noncystic fibrosis bronchiectasis (NCFBE). It has been well demonstrated that participation in patient support groups improves quality of life in those who suffer from interstitial lung disease and chronic obstructive pulmonary disease, but the impact is largely unknown for those who live with NCFBE, a chronic lung disease with rising prevalence with no targeted FDA-approved therapy. NCFBE causes chronic cough, dyspnea, recurrent infections, and leads to anxiety and uncertainty. This study seeks to evaluate the impact of a virtual patient support group for patients with NCFBE through questionnaires to assess change of quality of life and anxiety, and exacerbation rates through clinical assessment.
Non-cystic Fibrosis Bronchiectasis
In this exploratory study, the hormonal responses to a mixed meal will be examined in people with cystic fibrosis. The aim of this study is to find correlates with impaired glucose tolerance that is associated with this population.
Cystic Fibrosis
CMTX-101 is a bacterial biofilm disrupting monoclonal antibody being developed as an adjunctive therapy to standard of care antibiotics. The goal of this clinical trial is to assess the safety and tolerability of CMTX-101 in people with cystic fibrosis (pwCF). The main questions the study aims to answer are: * Are single doses of CMTX-101 IV infusion safe and tolerated * What is the pharmacokinetic (PK) profile of single doses of CMTX-101 * Do single doses of CMTX-101 induce development of anti-drug antibodies (ADA) and neutralizing antibodies (Nabs)
Persistent Infection, Cystic Fibrosis
The purpose of the study is to evaluate safety, tolerability, and pharmacokinetics of VX-828 and VX-828 in triple combination (TC) with Tezacaftor (TEZ)/ VX-118 or TEZ/ deutivacaftor (D-IVA) in healthy participants and VX-828 in combination with D-IVA with or without TEZ in participants with cystic fibrosis (CF).
Cystic Fibrosis
This will be a placebo-controlled, randomized, double blind, crossover study with a 4 week wash out period. Collect pilot data on the safety, tolerability, and feasibility of empagliflozin therapy in overweight/obese patients with CFRD to support a future larger randomized controlled trial.
Cystic Fibrosis-related Diabetes, Cystic Fibrosis
This study capitalizes on the emerging technology of 19F MRI, using conventional 'thermally' polarized perfluorinated gas (perfluoropropane, or PFP) mixed with oxygen and studied with magnetic resonance imaging (MRI) to visualize ventilation. This technique has not been studied in children. Children and adolescents (6-17 years old) with cystic fibrosis (CF) who have normal spirometry will undergo 19F MRI with the inhalation of an inert contrast gas to study ventilation. Comparisons will be made to a cohort of healthy children (6-17 years old) who will perform the same measures. The primary outcome measure is the feasibility of conducting these studies in the pediatric population. Parallel performance of multiple breath nitrogen washout (MBW) and spirometry will be used to compare the sensitivity of these outcomes to the presence of mild lung disease in these children. Finally, the investigators will compare data obtained during standard breath holds with a novel "free-breathing" technique that will eliminate the need for breath holds during MRI acquisition.
Cystic Fibrosis in Children
The purpose of this study is to look at lung ventilation in people with cystic fibrosis over time (1 year) using magnetic resonance imaging (MRI) with an inhaled contrast gas, and compare these measures to lung function assessed by spirometry and multiple breath nitrogen washout. This study also looks at how these measures change in response to a pulmonary exacerbation and treatment (if applicable). Over the span of a year, participants would be asked to complete 3-5 visits to the University of North Carolina at Chapel Hill (UNC). with each lasting up to 4 hours. If participants do not have a pulmonary exacerbation during the year they would be asked to complete 3 visits (one at enrollment, a second roughly 2 weeks later, and the third approximately a year later). If participants do experience a CF pulmonary exacerbation they would complete 5 visits (Visit 1, Visit 2, two exacerbation visits with one before treatment and the other after, and Visit 3 at one year after Visit 1). Only one exacerbation per participant will be tracked. Participants are eligible for this study if they are 18 years old or older, have Cystic Fibrosis (CF) with mild lung disease (FEV1 \>/= 60%), and can undergo an MRI. There are no known benefits for participating in this study.
Cystic Fibrosis, Ventilatory Defect