22 results
To assess the PK/PD relationship of prednisolone as either prophylaxis or therapy of GVHD in children after HCT.
We hypothesize that functional Near Infrared Spectroscopy (fNIRS), eye-tracking, Indirect Calorimetry (IC), the BOD POD, and Bio-Impedance Analysis (BIA) are feasible outcome measures for children with Angelman syndrome.
There might be discrepancies in number and type of Cancer Predisposition Syndrome diagnoses between the genotype first and the phenotype first approach.
Critical illness, along with the lack of zeitgebers on the PICU environment, and iatrogenic influences like medication and procedures, results in disturbed circadian rhythms in critically ill children, which is important for their recovery.
Primary objective: 1. To identify the components in human milk (e.g. nutrients, oligosaccharides, fatty acids and (pathogen specific) immunoglobulins) that have a protective effect against respiratory tract infections during the first year of life.…
PrimaryTo characterise the single and multiple dose to steady state pharmacokinetics of IV zanamivirin hospitalised neonates and infants under 6 months of age with influenza infection.SecondaryTo evaluate the safety and tolerability of IV zanamivir…
Part I:Primary Objective: Evaluate the effects of tiratricol on neurodevelopment in young MCT8 deficiency patients, as measured by the Gross Motor Function Measure (GMFM)-88 and Bayley Scales of Infant Development (BSID-III) Gross Motor Skill Domain…
To evaluate the performance of a *genotype first approach* (WES-based panel analysis) in diagnostics of genetic predisposition in children with cancer or neoplasms, compared to the current *phenotype first approach* (standard of care). In particular…
PRIMARY OBJECTIVES*Phase 1 Dose Finding Studies:-To determine the recommended phase II dose (RP2D) of new systemic therapy regimens. *Frontline chemotherapy questions:-To compare systemic therapy regimens for patients with VHR disease at diagnosis (…
Estimate key transmission parameters of SARS-CoV-2 in Europe from observing within household virus spread and seroconversion of household members, and to characterize the views and experiences of households regarding perceptions, practices regarding…
Patients will be included in 3 prospective cohorts, cohort A, B, C and 1 retrospective cohort, cohort 0. Cohort A: The primary objective of cohort A is to show that by implementing a standard infrastructure all newly diagnosed girls with cancer (or…
The main objective of this longitudinal study is to gain insight into the neurodevelopmental trajectories following neonatal critical illness, from the perinatal period to school-age compared to healthy controls. This insight may lead to the…
PRIMARY OBJECTIVES*Rx-induction: Comparison of the 3 year EFS rate of 2 induction regimens, GPOH and RAPID COJEC, in patients with high-risk neuroblastoma.*Rx-HDC: Comparison of the 3 year EFS rate from randomization of single HDC with Bu-Mel versus…
Objectives safety run-in: The primary objective is:• To assess the safety of i.v. teicoplanin prophylaxis three times per week with a two to three days interval in children with newly-diagnosed AML. A patient will be considered evaluable for safety…
The primary objective of the study is the following:• To characterize the safety and tolerability of DNL310 in pediatric subjects with MPS II (all cohorts)The secondary objectives of the study are as follows:• To characterize the CNS effects of…
To assess the PK/PD relationship of prednisolone as either prophylaxis or therapy of GVHD in children after HCT.
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…
Primary objectiveTo establish whether saliva samples could be used as an alternative for blood samples in the therapeutic drug monitoring for amikacin and flucloxacillin. To meet this objective, a PK mod-el will be developed for amikacin and…
To determine wheter the use of the taurolidine 1.35%, citrate 4%, and heparin 100 IU/ml lock solution (TauroLock*-Hep100) reduces the incidence of first tunneled central line associated bloodstream inections (CLABSI) compared to the heparin 100 IU/…
The primary objective is to demonstrate the superiority versus placebo of a 4 weeks intranasal OT administration on oral skills assessed by the Neonatal Oral-Motor Assessment Scale (NOMAS) in infants with PWS aged less than or equal to 3 months at…