Heart Rate Variability in Children and Adolescents With Cystic Fibrosis
Cystic Fibrosis in ChildrenThis study aims to assess the effects of a resistance exercise training program on heart rate variability in a group of children and adolescents with cystic fibrosis. The study design is a randomized controlled trial.
A Study Evaluating the Long-Term Safety of Elexacaftor Combination Therapy
Cystic FibrosisThis study evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ ivacaftor (IVA) triple combination (TC) in participants with cystic fibrosis (CF) who are homozygous for F508del.
Phase 2 Combination Study With Escalating Doses of MS1819-SD on Top of a Stable Dose of PPEs
Cystic FibrosisCystic Fibrosis Gastrointestinal Disease1 moreThis is a Phase 2 study sponsored by AzurRx SAS and involves testing of a new medication for the compensation of exocrine pancreatic insufficiency (EPI) caused by cystic fibrosis (CF). The new medication is called MS1819 spray dried (MS1819-SD) which is a lipase produced by the Lip2 gene of Yarrowia lipolytica using recombinant DNA technology. The primary purpose of this study is to investigate the efficacy and safety of escalating doses of study drug on top of a stable dose of PPEs in CF patients who are not fully compensated by PPEs only. This enzyme has demonstrated an appropriate profile to compensate the pancreatic lipase (enzyme) deficiency that is common in CP (chronic pancreatitis) and CF patients. The design of the study is open-label, meaning that all eligible patients will receive the study drug MS1819-SD. The study drug dose will increase throughout the study during dose escalation visits in each treatment period; study includes a total of three treatment periods. The total duration of the MS1819-SD treatment phase is of 39-51 days. The total duration of patient participation in the study is of 69-81 days. Approximately 24 patients will be enrolled in this study.
MN4000 for Treatment of CF and MND Patients in the Home Setting
Cystic FibrosisMotor Neuron Disease1 moreThe study was a non-randomized open label pilot study. It was an observational design conducted at one (1) site in the US. All enrolled subjects received treatment with the MN4000. This pilot study evaluated subject satisfaction with the therapy and adherence to the therapy during the 90-day treatment period, and also collected clinical outcome data. Outcomes were assessed before, during and after the MN4000 treatment period.
A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for...
Cystic FibrosisThis study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).
The Effects of Telerehabilitation on Muscle Function, Physical Activity and Sleep in Cystic Fibrosis...
Cystic FibrosisCovid191 moreThe aim of this study is to evaluate the peripheral muscle function, sleep disorders and physical activity level in children with cystic fibrosis who are physically inactive at home due to social isolation and to examine the effect of the 6-week online exercise protocol.
Streamlined Treatment of Pulmonary Exacerbations in Pediatrics
Cystic FibrosisSTOP PEDS is a pilot study of children with CF ages 6-18 across 10 sites in North America. The primary goal is to assess the acceptability and feasibility of a multicenter randomized trial comparing immediate antibiotics versus tailored therapy for pulmonary exacerbation (PEx) treatment in this population.
A Pilot Study to Assess the Effect of Intermittent iNO on the Treatment of NTM Lung Infection in...
Non-Tuberculous Mycobacterial PneumoniaCystic Fibrosis3 moreThe purpose of this open-label, multicenter, non-randomized, pilot study is to assess the safety of high dose intermittent iNO for treatment of NTM infection in CF and non-CF patients.
Manual Chest Physiotherapy and Active Cycle of Breathing Techniques (ACBT) in Patients of Cystic...
Cystic FibrosisCystic fibrosis is a genetic disease (autosomal recessive) which involves malfunction of the exocrine glands, leading to abnormal secretions in the body. It is a progressive disease that causes persistent lung infections and limits the ability to breathe over time. Clinical symptoms include persistent coughing, at times with phlegm, wheezing or shortness of breath, fatigue, difficulty with bowel movements sinus infections, poor growth, clubbing of the fingers and toes, and infertility in most males. The disease must be managed throughout life with diet, medication and preventive chest physical therapy as soon as any symptoms are noted in the young child. The purpose of the study was to evaluate the difference between the effects of Manual Chest Physiotherapy (CPT) and Active Cycle of Breathing Techniques (ACBT) in patients of Cystic Fibrosis. The tools of our study were Modified Borg Dyspnea Scale and Quality of well-being Scale. The total sample of our study was 14 out of which 7 were included in GROUP A and 7 Group B. SPSS 23 was used for statistical analysis and parametric tests were used for analysis
A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic...
Cystic FibrosisThis study will evaluate the long-term safety and efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) triple combination (TC) in subjects with CF who are 6 years of age and older with F/MF genotypes.