158 results
The main research objectives for the proof-of-concept study are to show the feasibility and safety of a daily cycle of feeding and fasting in critically ill children of different age-groups, that will trigger an adequate fasting response while…
The objective of this study is to investigate body image disturbances in adolescents with anorexia nervosa. To capture the multidimensional perspective, body image disturbances will be studied on a perceptual, cognitive-affective and a behavioural…
primary objectives- To asses sequelae of COVID-19, at 4 to 12-months following a COVID-19 diagnosis among pediatric patients receiving care in the hospital or outpatient setting in the Netherlands.- To determine risk factors for long-COVID among…
The primary objective is to compare the efficacy, as measured by recurrence-free survival (RFS) by blinded independent central review (BICR), of bempegaldesleukin plus nivolumab versus nivolumab in patients with completely resected Stage IIIA (lymph…
The objective of this study is to identify and develop laboratory measurements and other tests that may help develop treatments for Hunter Syndrome and, potentially, related diseases. The information learned from this study may also help doctors,…
The first objective is to explore the biological profile (e.g. the immunological profile, hypothalamic- pituitary-adrenal (HPA) axis, mitochondrial dysfunctioning, the gut microbiome) of adolescents with QFS and related fatigue disorders (CFS/ME,…
The main objective is to assess long-term neurodevelopmental, cardiovascular, pulmonary, and growth outcomes in a cohort of MC twins with sFGR and to compare outcomes within sFGR twin pairs.
The primary purpose of this study is to assess humoral immune responses of 3 dose levels of Ad26.COV2.S administered intramuscularly (IM) as a 2-dose schedule (56 days apart); Ad26.COV2.S administered IM as a single vaccination; safety and…
Primary aimTo determine the prevalence of children with hearing loss at time of discontinuation of cancer therapy in a prospective full cohort of 600 children with solid and CNS tumors.The secondary objectives of this study are:- To study the…
We aim to investigate if aortic stiffness, measured by pulse wave velocity (PWV), is present in patients with DMD and whether treatment with ACEi reduces aortic stiffness.
The primary objectives are: 1) to obtain in vivo pharmacokinetic data of 4*-PPT (the study product) measured in plasma of PKAN patients; 2) to obtain pharmacodynamic data of the biomarker COASY in circulating lymphocytes of PKAN patients, when 4*-…
to assess the diagnostic accuracy of CSI (at 5, 10 and 15 cm proximal to the upper border of the lower esophageal sphincter) for the presence of active disease and/or complete remission in pediatric EoE.
Primary Objective:To evaluate the effect of sonlicromanol on motor symptom severity in children with genetically confirmed mitochondrial disease affecting oxidative phosphorylation during a 6 month treatment period (GMFM).
The primary objective of the trial is to demonstrate a non-flat curve and evaluate the doseresponse relationship for 3 subcutaneous dosing regimens of BI 655130 (spesolimab) (with each regimen consisting of a single loading dose and a separate…
This study has been transitioned to CTIS with ID 2023-503973-39-00 check the CTIS register for the current data. To evaluate the efficacy of darvadstrocel in combined remission at Week 24 for the treatment of complex perianal fistula in pediatric…
The objectives of this study:• To generate the proof of concept for lanadelumab for treatment of symptoms of COVID-19 disease • To demonstrate its safety after intravenous administration.
This study has been transitioned to CTIS with ID 2024-518652-23-00 check the CTIS register for the current data. Investigating the long-term safety of personalized cholic acid treatment in patients with defects in bile acid synthesis based on…
To evaluate the effects of N-acetylcysteine (NAC; Fluimucil® in Dutch) on skin picking behaviour in children and young adults with PWS.