Active clinical trials for "Cystic Fibrosis"

Evaluate the safety and tolerability of VX-659 in healthy subjects

Physical activity (PA) in individuals with cystic fibrosis (CF) improves exercise capacity, slows decline in lung function, increases mucus clearance and improves health-related quality of life (HRQoL). Establishing and maintaining an exercise routine remains challenging and programs promoting PA in people with CF have poor participation. Moreover, while the positive effects of physical conditioning on lung function have been well reported, conventional measurements of lung function may lack the sensitivity to reveal improvement in mild lung disease. This randomized control trial (RCT; N = 60) evaluates the Do More, B'More, Live Fit, a 6-month fitness program designed to optimize exercise habits of 12-21 year-olds with CF through structured exercises with personalized coaching, exercise equipment including the FitBit Flex, online support and motivational messages delivered electronically. The intervention incorporates fitness preferences and encompasses endurance, strength and flexibility exercises while adjusting to physical fitness needs. The hypothesis is that intervention participants will have increased and sustained engagement and better health outcomes compared to control group participants. The investigators' specific aims are to: Increase daily PA and measures of fitness Improve lung clearance index (LCI) and participant HRQoL Demonstrate feasibility, accessibility and satisfaction of intervention using semi-structured interviews The results of this pilot evaluation of the Do More, B'More, Live Fit program will offer novel insight into factors that sustain engagement in exercise programs and identify if LCI is an appropriate clinical outcome to assess PA interventions. Results will inform future RCT of interventions to optimize exercise habits of adolescents with CF.

Ascending dose, 7-day, open label safety trial of SPX-101 Inhalation Solution in adult subjects with cystic fibrosis.

The present study aims at evaluating the effect of respiratory muscle training in adult patients with cystic fibrosis on lung function, exercise performance and quality of life.

This is a Phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate 4 different doses of GLPG2222 administered for 4 weeks to adult subjects with a confirmed diagnosis of CF and homozygous for the F508del Cystic Fibrosis Transmembrane conductance Regulator (CFTR) mutation.

A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for F508del.

This is a Phase 3, 2-part (Part A and Part B), open-label, multicenter study evaluating the pharmacokinetics (PK), safety, tolerability, and pharmacodynamics (PD) of multiple doses of lumacaftor/ivacaftor (LUM/IVA) in subjects 2 through 5 years of age (inclusive) with cystic fibrosis (CF), homozygous for F508del. Subjects who participate in Part A may participate in Part B, if they meet the eligibility criteria.

Cystic fibrosis (CF), a life-shortening genetic disease, is marked by acute episodes during which symptoms of lung infection increase and lung function decreases. These pulmonary exacerbations are treated with varying antibiotics for varying time periods based on needs determined by individual patients, their families, and the health care providers. Cystic fibrosis pulmonary guidelines for the treatment of pulmonary exacerbation published by the Cystic Fibrosis Foundation (CFF) in 2009 provided recommendations for treatment and also identified key questions for which additional studies were needed. A strong desire among clinicians to reduce treatment durations (and reduce cost, inconvenience, and potential toxicities) is in conflict with belief that patients not responding robustly to treatment might benefit from extending treatment. This randomized, controlled, open-label study is designed to evaluate the efficacy and safety of differing durations of IV treatment, given in the hospital or at home for a pulmonary exacerbation in adult patients with CF.

The purpose of this study is to demonstrate that Miglustat restores the function of the cystic fibrosis transmembrane conductance regulator (CFTR) in adult patients with cystic fibrosis homozygous for the F508del mutation.

The purpose of this study is to evaluate the efficacy and safety of VX-661 in combination with ivacaftor (IVA, VX-770) and IVA monotherapy in participants with Cystic Fibrosis (CF) who are heterozygous for F508del-CFTR allele and a second allele with a CFTR mutation predicted to have residual function.