335 results
The overall aim of the study is to develop innovative minimally invasive monitoring techniques that can identify lung inflammation in pwCF when using highly effective modulators, compared to patients whom are not eligible for CFTR modulators (…
to quantify atrial electropathology of the endocardium in adult patients with CHD and to correlate clinical characteristics, atrial volume, electropathology, atrial ectopy and features of pre- and post-procedural atrial fibrillation.
Primary • To evaluate the efficacy of QR-421aSecondary• To evaluate the safety and tolerability of QR-421a • To evaluate changes in Patient-Reported Outcome (PRO) measures in subjects treated with QR-421a• To evaluate systemic exposure of QR-421a
The primary objective of this study is to assess wall shear stress in the left ventricle outflow tract using 4D flow CMR in healthy controls, asymptomatic HCM and before and after surgical myectomy in patients with HOCM,
The primary outcome of this study is the detection of clinical relevant adenomas (>= 5mm) in the ileal pouch.
Comparison of prenatal and postnatal cardiac function assessed by echocardiography using 2-D, 3-D, pulsed wave Doppler, Tissue Doppler and (blood) speckle tracking (focussing on strain and strain rate) between fetuses/neonates with structural heart…
The main aim is to study early neurocognitive functioning and development in very young children with SCD. The secondary aims are to identify determinants (risk and protective factors) and biomarkers of early neurocognitive deficits in SCD and to…
(also see: C1. Protocol, page 9, paragraph 2)Primary: Creating a metabolic bio-database with body material of patients with inherited metabolic diseases, combined with clinical data.Secondary: Achieve better participation in national and…
Primary Objectives: 1. To assess the metabolic profile of patients with Dravet syndrome and identify metabolic variations that contribute to the pathophysiology of seizures and developmental delaySecondary Objectives:2. To assess the association…
In this study we aim to further characterize E/I balance (at different translational levels), and its relation to metabolic- and immune processes, clinical profiles, behavior and cognition
Identification, segregation analysis, and functional characterization of genetic variants possibly causing rare bleeding disorders. The following questions will be answered:1) Can we identify (new) causal variants that explain the phenotype of…
We aim to collect skin and blood cells from two to three WitKoS patients, reprogram these cells into iPSCs and use the iPSCs for differentiation into neurons/neural organoids to study the molecular underpinnings of WitKoS.
The primary objective is to test the hypothesis that S-ICD implant without DFT with PRAETORIAN Score is non-inferior to S-ICD implant with DFT with regard to first shock efficacy in spontaneous events. The secondary objective is to evaluate the…
To evaluate the efficacy of VX-121/TEZ/D-IVA in CF subjects who are homozygous forF508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, orhave at least 1 other TCR CFTR mutation and no F508del mutation
The main objective is to compare laparoscopic fenestration and aspiration sclerotherapy in patients with large symptomatic hepatic cysts on patient-reported outcomes. This information can be used to assess cost-effectiveness in both treatments.
The aim of this study is to assess the relationship between the AGD, the 2D:4D ratio and the presence of MRKH syndrome, endometriosis and PCOS.
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…
To evaluate to what extent primary headache disorders are caused by genetic factors and to identify genes and pathways involved in the pathophysiology of these disorders.
Primary objective of this trial is to investigate in a double blind randomized controlled trial in children undergoing open heart surgery if NO exposure during CPB reduces the postoperative duration of invasive mechanical ventilation (defined as…
- The development of blood-based tests, either platelet or EV-based, for the detection of (the type of) tumors in RB1-mutation carriers.- Determine the non-cancerous baseline in adult RB1-mutation carriers (heritable-Rb-survivors).- Contribute to…