Clinical Trial Finder

This is a pharmacokinetic study of Lumacaftor/Ivacaftor (Orkambi) in children between 6 years and 18 years of age who are homozygous for Phe508del-CFTR with severe cystic fibrosis related liver disease

Aim: To determine the safety and efficacy of LUM/IVA in subjects >12 years of age with CF, homozygous for F508del mutation of CFTR and an FEV1<40% of predicted normal, by comparing those patients treated with LUM/IVA with a cohort of age and sex matched CF controls with another set of mutations that lead to severe CFTR dysfunction (belonging to Class I, II or III), with an FEV1<40%5.

Currently, exercise guidelines for people with cystic fibrosis (CF) recommend 30 to 60 minutes of aerobic exercise on most days; a recommendation that is consistent with that provided for the general population. In addition to the usual demands on a person’s time such as work, study and family, people with CF have a high daily treatment burden involving airway clearance, medical and nutritional treatment, which are can take up to 4 hours per day to be completed. Therefore, completing 30 to 60 minutes of exercise per day can be difficult to achieve. A novel training approach, such as high intensity interval training (HIIT) may represent a more efficient option to increase exercise capacity. High intensity interval training has been demonstrated to improve exercise capacity in healthy individuals, as well as people with chronic respiratory disease. The main benefit of HIIT is the reduced time commitment, compared to other forms of exercise training. Currently, there are no guidelines for the use of HIIT in people with CF. The proposed PhD will, in people with CF, implement a HIIT program in a supervised setting over an 8 week period to investigate the effects of the program on exercise capacity, health-related quality of life (HRQoL), exercise self-efficacy, feelings of anxiety, depression, enjoyment and muscle oxidative capacity. It will also report on: (i) the proportion of participants who develop post-exercise muscle soreness and the severity of these symptoms; (ii) the tolerance of the HIIT program; (iii) the cardiorespiratory and symptom responses to HIIT over the 8 week program and; (iv) the behaviour change techniques. We hypothesize that in people with CF, 8 weeks of supervised HIIT will change exercise capacity, HRQoL, exercise self-efficacy, enjoyment and muscle oxidative capacity over and above any change seen with usual care.

Cystic Fibrosis (CF) is a complex, progressive, life-limiting disease that predominantly affects children and young adults. ‘Flare-ups’ of CF lung disease are common in people with this condition and often lead to admission to hospital and decline in lung capacity, imposing considerable burden on patients, their families and the healthcare system. Physical activity (PA) participation is a low-cost, easily accessible treatment option that has the potential to reduce the impact and progression of chronic lung disease in CF and may help reduce ‘flare ups’ of lung disease. However uptake and adherence to PA and exercise rehabilitation programs by young people with CF is poor. Advances in Internet technology and accessibility have made it possible for people to receive specialist medical care and rehabilitation therapy without attending the hospital. By using a secure website, readily accessible on any smartphone, tablet, laptop or computer it is possible for young people with CF to track their PA participation and receive feedback – at any time and place of their choosing. The aim of this project is to determine whether use of an online program to track PA participation and provide feedback, is more effective than usual care at improving PA participation, exercise capacity and quality of life, and prolonging the time to next hospital admission. People who agree to take part in the study will be randomly allocated to use the online program via the Internet, or to usual post-hospital care. At the beginning and end of the 12 weeks of the intervention phase, and at 3-months post completion of the intervention period, all participants will undergo measurements of PA participation, exercise capacity and health status. At 12 months- post completion of the intervention, hospital medical records will be reviewed to determine the frequency of hospital admission and number of hospital days. It is hypothesized that: 1. The web-based intervention will improve uptake and partic

The purpose of the study is to further improve outcomes in CF by developing a better understanding of the reasons for the decline in health status and lung function during adolescence, which is one of the key challenges facing clinicians. We hope to do this by looking at such things such as the 1) pathology involved in CF lung disease, 2) long term risk of emerging organisms, 3) potential effects of early therapeutic interventions, such as P. aeruginosa eradication, 4) metagenomic profile identifying bacteria in respiratory samples, 5) psychosocial factors, and 6) relationship between early life events and outcomes between 9-17 years of age.

Cystic fibrosis (CF) is the most common life-threatening genetic condition affecting Australian children. As well as repeated lung infections, children with CF develop insulin deficiency and eventually diabetes. The CF-IDEA trial will determine whether starting insulin treatment before the onset of diabetes (earlier than current practice) will improve the health of children with CF by improving body weight and lung function.

The general aim of this project is to conduct a randomized double-blind, placebo-controlled clinical trial of azithromycin to determine whether treatment from infancy is safe and will prevent the onset of bronchiectasis. One hundred and thirty infants will be recruited from CF clinics in Australia and New Zealand and treated from 3 months to three years of age. The primary outcome will be the proportion with radiologically-defined bronchiectasis at 3 years of age. Safety and mechanistic evaluations will also be undertaken.

Cystic fibrosis (CF) is the most common life-threatening genetic condition affecting Australian children. As well as repeated lung infections, children with CF develop insulin deficiency and eventually diabetes. The CF-IDEA trial (Cystic Fibrosis - Insulin Deficiency, Early Action) will determine whether starting insulin treatment before the onset of diabetes (earlier than current practice) will improve the health of children with CF by improving body weight and lung function.