View clinical trials related to Cystic Fibrosis.
Filter by:Ivacaftor is a novel, FDA approved new therapy that addresses Cystic fibrosis transmembrane conductance regulator (CFTR) dysfunctions in subjects with Cystic fibrosis (CF) and "gating mutations". The primary aim is to determine the mechanism(s) for weight gain in participants whom Ivacaftor treatment was initiated based on clinical indications by CF Care Team. This longitudinal study will assess in detail energy expenditure, weight gain, body composition, and lung function in 24 subjects ≥6 years old with CF with a gating mutation before treatment and after three months treatment with Ivacaftor. All subjects will be seen at the Children's Hospital of Philadelphia's Clinical Translational Research Center.
The primary objective of this study is to determine whether a single overnight, eight-hour administration of a 7% NaCl solution delivered by the Parion Sciences transnasal Pulmonary Aerosol Delivery (tPAD) device has a significant effect on mucociliary clearance in subjects with cystic fibrosis, as compared to no treatment. This study will be conducted at the University of Pittsburgh Medical Center.
This is a Phase 3, international, multicenter, randomized, double-blind, placebo-controlled, efficacy and safety study of ataluren in patients with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.
maldigestion of dietary macronutrients (pancreas not producing enough enzymes for digestion of fat, sugars and proteins) in Cystic Fibrosis
This trial aims to provide prospective evidence of the safety and efficacy of mannitol 400 mg b.i.d. in subjects aged 18 years and above. We hypothesize that inhaled mannitol 400 mg b.i.d. will increase the mean change from baseline FEV1 (mL) compared to control over the 26-week treatment period in adult subjects with cystic fibrosis. Any improvement in FEV1 is considered clinically meaningful, however, this trial has set a threshold of 80 mL for the purposes of determining an appropriate sample size for statistical power while retaining trial feasibility in an orphan disease population
This study is a single center, randomized pilot study to evaluate the clinical effectiveness of nasal high flow 20LPM humidification therapy in subjects with Cystic Fibrosis.
The purpose of this study is to determine whether a nutritional supplement is effective in the treatment of malnutrition in pediatric patients with failure to thrive or cystic fibrosis.
The objective of the investigators research program is to determine whether a combined exercise program (aerobic and resistance) improves abnormal glucose tolerance and diabetes control in CF patients. This program of exercises to increase strength and muscle endurance, exercise tolerance, will be assessed in a group of 12 CF patients with impaired glucose tolerance. The effect of exercise will be compared with a control group of 12 patients also glucose intolerant but do not participate in structured physical activity program. The duration of the exercise program will be 12 weeks and several measures (glucose tolerance, inflammatory profile, lung function, exercise capacity, muscle strength and endurance) will be conducted pre and post program to assess the program's impact .
Exacerbation in cystic fibrosis play an important role in terms of mortality and morbidity. Exacerbation frequencies is relatedto FEV1 decline and mortality. There is a relationship between exacerbation frequency and QoLdeterioration. Moreover exacerbation treatments present health costs burden leading to important physical psychological and social impact. Preventing exacerbation and early detection of these exacerbations may decrease intensity and freqauency of exacerbation leading to increase clinical status and QoLwith a decreased health cost. Patients actually follow in CRCM track exacerbations when visits out patient clinic and during phone call. If patients did not call or did not present regularly to out patient clinic, exacerbation detection came later and so increasde the burden and therapeutic pressure. The objective of our study is to identify earlier the potential exacerbations and so decreased the health costs and increased the patient's QoL. Forthis purpose we propose to use modern technologiessuch as smartphone in order to create alert when patients report weekly health satatus. We will compra patients randomize in control group with standart follow-up to patients randomize in the smartphone group.Moreover we will study the compliance and satisfactory degree of the use of this device in the interventional arm.
The treatment burden for patients with cystic fibrosis (CF) is significant and poor adherence has been well-documented. The investigators hypothesize that a coaching intervention will empower young adults with CF to manage their lives with CF and improve health-related quality of life (HRQoL). The main aim of the study is to establish the feasibility and acceptability of a life-coaching intervention.