vx-770 has been researched along with Body-Weight* in 5 studies
1 trial(s) available for vx-770 and Body-Weight
Article | Year |
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Growth in Prepubertal Children With Cystic Fibrosis Treated With Ivacaftor.
Cystic fibrosis (CF) is known for its impact on the lung and pancreas of individuals; however, impaired growth is also a common complication. We hypothesized that targeting the biological defect in the CF transmembrane conductance regulator (CFTR) protein may affect growth outcomes.. In this post hoc analysis, we assessed linear growth and weight in 83 children (aged 6-11 years) enrolled in 2 clinical trials, the longitudinal-observation GOAL study and the placebo-controlled ENVISION study, to evaluate the effects of ivacaftor, a CFTR potentiator. We calculated height and weight z scores and height and weight growth velocities (GVs).. In ivacaftor-treated children in GOAL, height and weight z scores increased significantly from baseline to 6 months (increases of 0.1 [P < .05] and 0.26 [P < .0001], respectively); height GV increased significantly from 3 to 6 months (2.10-cm/year increase; P < .01). In ivacaftor-treated children in ENVISION, height and weight z scores increased significantly from baseline to 48 weeks (increases of 0.17 [P < .001] and 0.35 [P < .001], respectively). Height and weight GVs from baseline to 48 weeks were also significantly higher with ivacaftor than with placebo (differences of 1.08 cm/year [P < .05] and 3.11 kg/year [P < .001], respectively).. Ivacaftor treatment in prepubescent children may help to address short stature and altered GV in children with CF; results from these analyses support the existence of an intrinsic defect in the growth of children with CF that may be ameliorated by CFTR modulation. Topics: Aminophenols; Body Height; Body Weight; Child; Cystic Fibrosis; Double-Blind Method; Female; Follow-Up Studies; Humans; Longitudinal Studies; Male; Quinolones; Treatment Outcome | 2017 |
4 other study(ies) available for vx-770 and Body-Weight
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Effect of elexacaftor-tezacaftor-ivacaftor on body weight and metabolic parameters in adults with cystic fibrosis.
Though weight gain has been reported in some clinical trials of CFTR modulators, the effect of elexacaftor-tezacaftor-ivacaftor on body weight, body mass index (BMI), blood pressure, lipids and glycemic control in the real-world setting remains incompletely described.. We performed a single-center, retrospective, observational analysis of the effect of elexacaftor-tezacaftor-ivacaftor on body weight and cardiometabolic parameters in 134 adult CF patients of the Washington University Adult Cystic Fibrosis Center. Body weight, BMI, and blood pressure were extracted from outpatient clinic visits for the year preceding and the period following the initiation of elexacaftor-tezacaftor-ivacaftor. Other metabolic parameters were extracted at baseline and at latest available follow-up.. In this single-center, retrospective, observational study of 134 adults with CF, initiation of elexacaftor-tezacaftor-ivacaftor was associated with increases in BMI at a mean follow up of 12.2 months. Changes in other cardiometabolic risk factors were also observed. Widespread use of elexacaftor-tezacaftor-ivacaftor may be expected to increase the incidence of overnutrition in the CF population. Topics: Adult; Aminophenols; Benzodioxoles; Body Weight; Cardiometabolic Risk Factors; Chloride Channel Agonists; Cholesterol; Cystic Fibrosis; Humans; Indoles; Pyrazoles; Pyridines; Pyrrolidines; Quinolones; Retrospective Studies | 2022 |
Females with Cystic Fibrosis Demonstrate a Differential Response Profile to Ivacaftor Compared with Males.
Topics: Adolescent; Adult; Age Factors; Aminophenols; Body Mass Index; Body Weight; Child; Chloride Channel Agonists; Chlorides; Cystic Fibrosis; Disease Progression; Female; Forced Expiratory Volume; Humans; Male; Quinolones; Sex Factors; Sweat; Young Adult | 2020 |
Sweat chloride is not a useful marker of clinical response to Ivacaftor.
Clinical trials have revealed that Ivacaftor significantly reduces sweat chloride in patients with cystic fibrosis who carry the G551D mutation. This finding has been incorporated into the commissioning guidelines in the UK with a sweat chloride reduction of 30% or below 60 mmol/L, specified as the main criteria for continued funding of Ivacaftor for individual patients. In a cohort of 24 adults who were prescribed Ivacaftor, there was no correlation between absolute or relative reductions in sweat chloride and improvements in lung function. This questions the validity of sweat chloride as a surrogate marker of clinical efficacy. Topics: Aminophenols; Biomarkers; Body Height; Body Weight; Chlorides; Cohort Studies; Cystic Fibrosis; Forced Expiratory Flow Rates; Humans; Lung; Prospective Studies; Quinolones; Spirometry; Sweat | 2014 |
Ivacaftor. Uncertain harm-benefit balance.
Cystic fibrosis is a serious genetic disease due to mutations in the gene encoding CFTR (Cystic FibrosisTransmembrane conductance Regulator), a protein involved in cellular transmembrane transport, particularly of chloride ions. Ivacaftor is described as a selective potentiator of CFTR protein. It is now licensed for the treatment of cystic fibrosis patients 6 years of age and older who carry the CFTR G551D mutation, which is the case for about 4% to 5% of cystic fibrosis patients. Two randomised placebo-controlled trials of ivacaftor have been carried out in this setting, in a total of 213 patients. The frequency of pulmonary exacerbations fell in one of the two trials during the first 48 weeks of treatment, but there was no impact on the number of hospitalisations or the use of intravenous antibiotics. In both trials, addition of ivacaftor improved FEV1 by an average of about 10% at week 24 and increased body weight by an average of about 2.5 kg after 1 year. Upper respiratory tract infections occurred in the ivacaftor groups during these trials, with more cases of bacterial contamination and, possibly, more pulmonary exacerbations in the longer term. Ivacaftorcarries a risk of numerous pharmacokinetic interactions that can require dose adjustment, particularly for patients with hepatic impairment and those receiving cytochrome P450 isozyme 3A inhibitors or inducers. Given the absence of data, ivacaftor should be avoided during pregnancy. In practice, in 2013, it is not possible to determine the harm-benefit balance of ivacaftor due to the high risk of drug interactions, as well as a possible increased risk of infections and liver toxicity. Topics: Aminophenols; Body Weight; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Interactions; Forced Expiratory Volume; Humans; Quinolones; Randomized Controlled Trials as Topic; Uncertainty | 2013 |