Cystic Fibrosis

Researchers are evaluating the safety, tolerability, and early effects of 4D-710, an investigational gene therapy, in adults with cystic fibrosis CF lung disease who cannot use or tolerate existing CFTR modulator therapies. A sub-study also includes adults with advanced CF lung disease or frequent lung flare-ups who are currently on CFTR modulator therapy. This Phase 12 open-label trial aims to find appropriate dosing and assess potential benefits for these patient groups. Participants receive a single inhaled dose of 4D-710, which is a gene therapy designed to deliver a corrected version of the CFTR gene to lung cells. The study includes a dose exploration phase for those ineligible for modulator therapy, a dose expansion phase at selected doses, and a sub-study for participants on modulator therapy receiving various doses. Each participant undergoes one administration of the therapy during the trial. Throughout the study, participants are monitored for adverse events over a 60-month period. Evaluations include lung function tests, oxygen saturation measurements, and tracking of pulmonary exacerbations. Participants maintain their existing treatments if applicable, and researchers assess safety and early signs of effectiveness. The total study duration extends up to approximately nine years, including long-term observation after dosing.

Children with short bowel syndrome SBS have reduced intestinal length or function, causing poor absorption of nutrients and requiring intravenous nutrition parenteral nutrition, PN to sustain hydration and nutrition. Long-term PN use can lead to serious complications including liver failure. This study evaluates the RELiZORB enzyme cartridge, a device designed to improve fat digestion and absorption by predigesting fats in enteral feeding tubes, potentially reducing the need for PN in children aged 2 to 18 years who are PN dependent. Participants will use the RELiZORB device connected inline with their enteral feeding tubes daily for 90 days. The device mimics pancreatic lipase to digest fats before they enter the intestine, bypassing the need for bile acid emulsification. Tube feeds run across the device to enhance fat absorption. This open-label, single-group trial monitors changes in PN calories, growth, fecal fat, plasma fatty acids, and nutrition intake over the study period. During the study, participants will have assessments at multiple timepoints including days 7, 14, 28, 60, and 90. Researchers will measure effectiveness by changes in PN calorie requirements and weight, monitor safety by tracking adverse events, and evaluate tolerability. The study also involves tracking growth, stool fat content, blood fatty acid levels, and nutrition intake. The total participation duration is 90 days, with regular clinic visits for monitoring and data collection.

The GENESIS clinical study aims to map HLA genetic variation in the Greek population and evaluate possible correlations with selected underlying diseases. It is a multicenter, prospective, non-interventional clinical study targeting 12,000 subjects over an anticipated duration of 36 months, with the goal of creating a pilot HLA map for medical research and possible clinical applications. Each subject will complete one visit at a participating site and provide demographic information, including date of birth, gender, race, ancestry, height, and weight, as well as information about smoking or vaping, alcohol consumption, arterial blood pressure, diagnosed diseases, and current treatments. Recent clinical laboratory results from up to 12 months before sample collection may also be collected when available, including blood count, metabolic, liver enzyme, and biochemical parameters. Two buccal swabs will be collected from each subject for DNA extraction and HLA genotyping analysis. Selected DNA samples will also undergo low-pass whole genome sequencing to further investigate associations between the HLA region and autoimmune diseases. After the analysis is completed, an individualized ancestry report will be securely available to study subjects if they elect to access it.

The PALMIRE project is a prospective, monocentric study conducted at the University Hospital of Reims, France, involving patients diagnosed with chronic inflammatory pulmonary diseases including asthma, chronic obstructive pulmonary disease COPD, bronchiectasis, cystic fibrosis CF, primary ciliary dyskinesia PCD, and interstitial lung diseases ILD. These diseases are characterized by lung inflammation and remodelling, with highly variable clinical, functional, microbiological, biological, pathological, and prognostic features. The study aims to analyze phenotypic characteristics, including clinical, demographic, biological, morphological, pathological, and microbiological features, together with respiratory exposures and underlying mechanisms involving airway epithelium and inflammation processes. It also considers shared phenotypic traits between different diseases and the coexistence of components of several diagnoses in the same patient, which may suggest shared underlying mechanisms and possible therapeutic targets beyond the initial medical diagnosis.

Acute respiratory failure is a serious medical emergency that can quickly lead to death without prompt treatment. Researchers are evaluating a new portable device designed to provide Continuous Positive Airway Pressure CPAP support outside of hospital settings. This device aims to be easy to use by non-medical individuals and to improve outcomes for patients experiencing respiratory failure. The study will test the devices effectiveness and usability in two groups healthy elderly adults who will self-apply the device, and hospitalized patients with mild oxygenation impairment. The device is designed to deliver positive end-expiratory pressure PEEP between 4 and 7.5 cmH2O, and its performance will be assessed in real-world clinical settings across three hospitals. Participants will be monitored for up to 2 hours during device use, with assessments including how easy the device is to use, duration of proper pressure delivery, battery consumption and duration, and changes in blood oxygen levels SpO2. The study will collect data to support the devices future use as a first-aid tool for respiratory failure outside hospitals. The total study duration and follow-up details are not specified.

Coughing is a common and distressing symptom for people with interstitial lung disease ILD, affecting daily activities, work, and social life. This research explores a non-drug cough control therapy combined with pulmonary rehabilitation to see if it is practical and helps reduce chronic cough lasting more than eight weeks in ILD patients. The study aims to assess how well patients accept this therapy and whether it improves quality of life, cough symptoms, and fatigue compared to standard pulmonary rehabilitation alone. Participants will join a pulmonary rehabilitation program involving aerobic and strengthening exercises, disease-specific education, and self-management as usual care. Two weeks before finishing this program, they will receive four virtual sessions of 45 to 60 minutes each, focusing on cough control education and techniques. These sessions include assessing cough, learning suppression methods, breathing retraining, and reinforcing strategies to manage cough in daily life. During the study, researchers will track enrollment and attendance to measure feasibility, along with various clinical outcomes like cough severity questionnaires, fatigue scales, breathlessness ratings, and patient satisfaction interviews. The intervention is delivered online via Zoom, allowing remote participation. The total study duration averages one year, with ongoing assessments to understand the therapys impact and participants experiences.

Researchers are evaluating SP-101, an investigational gene therapy, in adults with cystic fibrosis CF who cannot use or tolerate CFTR modulator therapy. This Phase 12, first-in-human study aims to assess the safety, how the body processes the drug, and its biological effects with varying doses. The trial is open-label and conducted at multiple centers. Participants receive a single inhaled dose of SP-101 combined with doxorubicin at different dose levels. The study includes three groups Dose 1, Dose 2, and a selected dose expansion group. Each participant will receive only one inhalation treatment during the trial. During the study, researchers will monitor participants for adverse events over 52 weeks to evaluate safety. They will also measure how the drug behaves in the body and its impact. Participants will have regular assessments including lung function tests and oxygen levels. The trial lasts about a year, during which participants will be closely observed and evaluated.

Researchers are evaluating the safety, tolerability, and biodistribution of a drug called RCT2100 in both healthy adults and people with cystic fibrosis CF. This phase 2 study aims to provide initial safety data to support future clinical research. The study includes participants aged 18 to 60 years and assesses different dosing regimens and co-administration with another drug, ivacaftor, in those with CF. The study is divided into three parts. Part 1 involves a single ascending dose of inhaled RCT2100 given via nebulizer to healthy participants. Part 2 evaluates multiple ascending doses of inhaled RCT2100 in participants with CF over 4 to 12 weeks. Part 3 assesses the safety of RCT2100 combined with oral ivacaftor for 6 weeks in participants with CF after an initial ivacaftor run-in period. Placebo doses are also used in Part 1 for comparison. Participants will undergo various assessments including monitoring for adverse events and serious adverse events from baseline through follow-up periods up to 24 weeks for parts involving CF participants. They will receive study treatment by inhalation and oral dosing ivacaftor in Part 3 and have regular safety evaluations including clinical labs, vital signs, lung function tests, and ECGs. The study involves randomized allocation and double-blinding to evaluate safety and tolerability comprehensively over the treatment and follow-up periods.

Researchers are evaluating the combination of Adebrelimab with carboplatin or cisplatin plus etoposide chemotherapy and concurrent radiotherapy as a first treatment for patients with extensive-stage oligometastatic small cell lung cancer. This Phase II open-label study aims to assess the safety and effectiveness of this regimen in this patient group. The trial is led by Nanfang Hospital, Southern Medical University and focuses on patients with limited metastatic lesions and organs affected. Participants will first receive Adebrelimab intravenously along with carboplatin or cisplatin and etoposide for two cycles during the induction phase. Next, participants undergo concurrent chemoradiotherapy, including thoracic radiation and stereotactic body radiation therapy SBRT to metastases, combined with one to two cycles of chemotherapy. Following this, they will receive one to two additional cycles of Adebrelimab combined with chemotherapy, and then continue on Adebrelimab alone as maintenance therapy until disease progression, unacceptable side effects, or withdrawal from the study. During the study, participants will be closely monitored through clinical assessments, imaging, and laboratory tests to track progression-free survival and other measures such as overall survival and response rates, with follow-up lasting up to approximately 30 months. Safety will be monitored by recording adverse events throughout the treatment and maintenance phases. The total participation time includes induction, concurrent chemoradiotherapy, maintenance, and follow-up periods to fully evaluate treatment outcomes and tolerability.

This research aims to advance treatments for people with cystic fibrosis CF who do not take cystic fibrosis transmembrane conductance regulator CFTR modulators. It focuses on those genetically ineligible or not using these modulators, a group with different health challenges compared to most people with CF. The study seeks to collect detailed health data and specimens to support new therapy development and improve clinical trial designs for this underserved population. The study is observational and will follow participants over time, collecting research-quality CF outcome data aligned with clinical trial endpoints important for developing new therapies. Sub-studies will gather specialized measures to help evaluate safety and effectiveness of future treatments. Researchers also aim to understand research participation and engagement in this community. Participants will provide health information and samples during scheduled visits over at least 12 months. Researchers will monitor lung function using measures like ppFEV1, CFQ-R-RD scores, lung clearance index LCI, and mucociliary clearance MCC indices. The study will help characterize this CF group and support the development of innovative trials. It will also assess research involvement and provide data for comparison with new therapies.