73 results
Main objectivePart A: To evaluate the safety and efficacy of treatment with VX-371, administered with and without 4.2% hypertonic saline (HS) in subjects with primary ciliary dyskinesia (PCD) who are *12 years of age.Part B: To evaluate the safety…
Primary objective: To investigate whether pharmacokinetics of the recommended dose of inhaled tobramycin, defined as serum tobramycin Area Under the Curve (AUC0-24hr), with the I-neb (75 mg) is equivalent to the PariLCPlus (300 mg) nebuliser in…
Primary: To establish the tolerance of camel*s milk and mare*s milk in standardized food provocation tests in patients with double-blind placebo-controlled provocation (DBPCP) proven CMA >=3 years of age. Secondary: To determine the minimum…
To determine the relative bioavailability of ciprofloxacin oral suspension versus oral tablets in pediatric cancer patients, and to determine the absolute bioavailability of tablets and suspension utilizing the 100% bioavailability of intravenous…
The purpose of this trial is primarily to assess the efficacy of inhaled mannitol compared with a true placebo in subjects with cystic fibrosis aged 6 to 17 years.We hypothesize that inhaled mannitol will improve the overall health and hygiene of…
Primary objective:To compare the change in small airways obstruction (FEF75%) in patients with CF when inhaling one ampule of inhaled tobramycin with the Akita® compared to standard of treatment (twice daily nebulization of one ampule using standard…
Our primary objective is to investigate whether oxytocin administration can affect empathy and emotion recognition in healthy and antisocial adolescents. We expect that oxytocin will enhance empathy and emotion recognition in both healthy and…
This study concerns research into the applicability and effectiveness of CBM-I training through an app on the mobile phone. The research is done in adolescents (12-18 years) treated at the Bascule clinic for OCD. The training will take place during…
The aim of this study is to investigate the effectiveness of a brief computer intervention to improve body satisfcation and self-esteem in a clinical population of adolescents with an eating disorder. We will examine whether the intervention is…
Primary objectives: this study will 1) assess the accuracy of the Cortex MetaLyzer® 3B in adolescents (12-17 yr) for measuring REE, using the Deltatrac® II metabolic monitor as a reference; and 2) assess the accuracy and the within-subject, within-…
To evaluate the effects of long-term intranasal oxytocin on social behaviour in children with PWS and to compare the effects of different doses and frequencies of oxytocin administration
Primary objective:The purpose of this study is to evaluate the short term effect of a patellar strap on symptoms and sports participation in children with Osgood-Schlatter disease.Secondary objective: The second objective of this study is to…
to investigate the effect and efficacy of pyridostigmine on muscle strength and fatiquabillity in patients with SMA.
To evaluate the effects of intranasal oxytocin compared to placebo administration on appetite, satiety and food intake.Also effects on social behavior, BMI, body composition, IGF-I levels and genetic differences (deletion / mUPD). To assess oxytocin…
Primary objective- To measure the effect of sapropterin on diurnal and day to day variations of blood phenylalanine concentrations.Secondary objective- To measure the effect of sapropterin on diurnal and day to day variations of blood tyrosine…
18-22 YEAR OLD MALES WIHT A NORMAL WEIGHTExamine the possible attenuating cardiometabolic health effects of (A) hourly, 10-min standing interruptions, when compared to a single bout of prolonged sitting, and (B) sitting on a stability ball, when…
Primary ObjectiveTo evaluate the efficacy of lumacaftor/ivacaftor combination therapy (LUM/IVA) in subjects with cystic fibrosis (CF) 12 years of age and older who have at least one A455E mutation.Other Objectives* To explore the association between…
Our primary objective is to investigate whether a further gain in efficacy of oral Ivacaftor treatment can be reached by co-supplementation of genistein, as suggested by their highly synergistic action in intestinal organoidsOur secondary objectives…
In light of the variability in response between UCCS and Glycosade® in patients with hepatic GSD (personal communication from authors and published data (Bhattacharya et al. 2007; Correia et al. 2008; Corrado M et al, 2013; Hubert A et al, 2013));…
We will determine in a prospective cohortl study, differences between frequent screen users and infrequent screen users in sleep onset time, wake-up time, mid-sleep time, sleep duration, body mass index and melatonin onset. In the randomized cross-…