159 results
To establish a healthy control group for microcirculatory parameters for current and future studies on the paediatric microcirculation.
The aim of this proposal is threefold: (i) test a neuroscientific model of prosocial development by relating neuroscience discoveries to changes in several dimensions of prosocial development in a comprehensive study including children, adolescents…
In this study, the aim is to perform a clinical evaluation of the NovioMini in children during behavioural bladder training to examine the performance of the NovioMini as a full-bladder-based notification system during natural bladder filling.
To assess whether nutritional status can affect the outcome of spatial cognition training in the brain.
The aim of this study is to develop a safe and fast MRI protocol for the imaging of neonatal and pediatric BPD patients. This METC application addresses the first part of the MRI- BPD project: *Ventilation, Inflammation, perfusion and structure in…
The primary aim of this study is to investigate the safety and feasibility of ultra-high field magnetic resonance imaging (MRI) of the brain in neonates and to optimize scan protocols.
The purpose of the HiN-6 study is:(1) To describe the health status of the Dutch haemophilia population, with special focus on viral infections, inhibitor development and age-related co-morbidities.(2) To gain insight into the health-related quality…
Primary objective- To evaluate the effect of lumasiran on percent reduction in urinary oxalate excretion Secondary objectives - To characterize the effect of lumasiran on absolute levels of urinary oxalate excretion, oxalate:creatinine ratios, and…
This study has been transitioned to CTIS with ID 2023-506739-14-00 check the CTIS register for the current data. Primary ObjectivesThe primary objectives of this study are as follows:• To evaluate the safety and tolerability of risdiplam.• To…
General- To compare physical activity levels of pediatric patient subjects with healthy control subjects.- To compare mean heart rate and variation in heart rate during the day of patients and healthy controls.- To compare sleep duration of patients…
The purpose of this study is to evaluate the possible risks and efficacy (improvement of disease) with an experimental oral study drug named eliglustat in pediatric patients from 2 to 18 years with Gaucher disease.
This study has the following objectives:1) To assess if dEMG measurement is feasible during pulmonary transition in the delivery room* Does the signal quality (expressed in heart rate and respiratory rate) of dEMG during the transition agree with…
Primary objective: To evaluate the efficacy of alirocumab, on low-density lipoprotein cholesterol (LDL-C) levels of treatment in children with homozygous familialhypercholesterolemia (hoFH) 8 to 17 years of age on top of background treatments.…
Primary:* To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.Secondary:* To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study,…
We want to investigate whether blood levels of vitamin K are lower in children with O.I. compared to children without O.I.
The purpose of the study intends to assess safety, activity and pharmacokinetics of ruxolitinib treatment with corticosteroids in treatment-naïve and steroid refractory (SR)- acute Graft versus Host Disease (aGvHD) patients aged *28 days to <…
Primary Objective:To characterize a cohort of STXBP1-encephalopathy patients in a standardized, integrative manner, including in-depth clinical profile characterization and EEG recordings. Moreover, for a subgroup of patients, iPSC-derived induced…
Study Objectives:* To monitor the long-term safety profile of NI-0501* To assess HLH patients* survival after NI-0501 treatment* To assess duration of response to NI-0501 treatment (i.e. maintenance of HLH control)* To assess post-HSCT outcome…
The primary aim of this study is to examine prospectively the effects of BOOSTH on moderate to vigorous PA (MVPA) in primary school children (5th to 7th grade).
The overall aim of the study is to correlate the anatomical and functional anatomy of the larynx, as seen on MRI to various functional outcome measures in patients who underwent open airway surgery for LTS. The specific aims of this study are: 1) to…