40 results
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…
This study has been transitioned to CTIS with ID 2024-516123-13-00 check the CTIS register for the current data. Part I:Primary Objective: Evaluate the effects of tiratricol on neurodevelopment in young MCT8 deficiency patients, as measured by the…
This study has been transitioned to CTIS with ID 2024-514917-36-00 check the CTIS register for the current data. PRIMARY OBJECTIVES*Rx-induction: Comparison of the 3 year EFS rate of 2 induction regimens, GPOH and RAPID COJEC, in patients with high-…
The project*s overarching aim is to diminish respiratory disease burden in moderate-late preterm born infants in their first 18 months of life. We have formulated the following specific objectives: 1. Determine whether the introduction of our follow…
The primary objective of this study is to investigate whether the use of the EOS calculator safely reduces antibiotic exposure in newborns with suspected EOS in the first 24 hours after birth compared to the current Dutch guidelines.Secondary…
Assess the potential efficay of L-serine dietary supplementation in patients children with a GRIN2B LoS mutation.
Research question: Is maternal microbiome dysbiosis in MOB an underlying mechanism in the pathophysiology of adverse maternal pregnancy and offspring outcome?Objectives: Analyse the differences between the gut and vaginal microbiome, maternal and…
Primary objectives:To demonstrate that diagnostic sensitivity of plasma metanephrines (free and total) is superior to standard evaluation procedures (urinary Dopamine, HVA and VMA) at diagnosis and end-of-induction assessment.Secondary objectives:•…
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…
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…
This study has been transitioned to CTIS with ID 2024-510579-40-00 check the CTIS register for the current data. PRIMARY OBJECTIVES*Phase 1 Dose Finding Studies:-To determine the recommended phase II dose (RP2D) of new systemic therapy regimens. *…
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…
Investigate whether, in a routine clinical environment across a number of centers, the fabian-PRICO can adequately maintain oxygen saturation, with minimal staff intervention.
This study has been transitioned to CTIS with ID 2023-507582-25-00 check the CTIS register for the current data. PRIMARY OBJECTIVESThe primary objective of MAKEI V is to assess in a randomized comparison whether the efficacy of Carboplatin (600 mg/…
This study has been transitioned to CTIS with ID 2022-502503-30-00 check the CTIS register for the current data. The primary objective is to improve the outcome (in terms of event-free survival (EFS) as the primary endpoint) of newly diagnosed KMT2A…
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.…
The main objective of the study is to dissect the genetic and stress-based mechanisms on the development of pediatric heart failure in congenital heart disease (ConHD) with the use of induced pluripotent stem cell-derived cardiomycoytes (iPSC-CMs).…
The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia…
This study has been transitioned to CTIS with ID 2022-501454-11-00 check the CTIS register for the current data. PRIMARY OBJECTIVE:- To show that it is possible to cure at least 75% of patients belonging to the SR group with Vinblastine-monotherapy…