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68Ga-SATO in paediatric neuroblastoma patient; exploratory, safety, non-randomized, open label, comparative study - GAP NBL study*

This study has been transitioned to CTIS with ID 2024-513843-10-00 check the CTIS register for the current data. Primary objective: Assess the short term safety and tolerability of 68Ga-SATO in pediatric patients with NBLSecondary objectives: -…

Dermal Substitution in Paediatric Burns: A Prospective Case Series

The aim of this study is to investigate the scar quality in a paediatric burn population treated with Glyaderm up to 12 months after surgery.

Towards lifelong healthy LUNGS: a multidisciplinary follow-up framework for preterm infants.

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…

A two-cohort, open-label, single arm, multicenter study to evaluate efficacy, safety and
tolerability, pharmacokinetics and pharmacodynamics, of emapalumab in children and
adults with macrophage activation syndrome (MAS) in Still*s disease (including systemic
juvenile idiopathic arthritis and Adult onset Still*s disease) or with MAS in Systemic lupus
erythematous

This study has been transitioned to CTIS with ID 2024-516153-52-00 check the CTIS register for the current data. Primary objective: To demonstrate efficacy of emapalumab in the treatment of patients in:• Cohort 1: Macrophage activation syndrome (MAS…

Effectiveness of L-serine dietary supplementation in children with a GRIN2B loss-of-function mutation: n-of-1 series

Assess the potential efficay of L-serine dietary supplementation in patients children with a GRIN2B LoS mutation.

The IBIS study: Infant motor development as an early Biomarker in children with SCN1A gene mutation - a pilot project

Objective: The primary objective of this pilot project is to investigate the early course of motor development in children with DS. Secondary objectives are to investigate the relation between type of SCN1A mutation and developmental profile and to…

A randomized controlled trial to investigate the (cost)effectiveness of oral immunotherapy with different allergens in young children with an established food allergy.

What is the clinical- and cost-effectiveness of early low-dose oral immunotherapy aimed at long-term tolerance induction in children under the age of 30 months with an established food allergy compared to routine care?What is the effect of early low…

International multicenter observational study to determine the diagnostic sensitivity of plasma metanephrines and urinary catecholamines and metabolites compared to standard evaluation procedures in children with high risk neuroblastoma

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:•…

Table salt for the treatment of umbilical granuloma

The aim of this observational study is to evaluate the treatment of umbilical granulomas with table salt.

The COllaborative Neonatal NEtwork for the first CPAM Trial, the CONNECT

The main objective is to identify the optimal management for patients with an asymptomatic CPAM, based on functional outcome measures. Furthermore, the primary objective is to stratify asymptomatic CPAM patients into a low and high risk group for…

Integrated Prospective and Retrospective Observational Study to Characterize Biomarkers and Disease Progression in Patients with Pelizaeus-Merzbacher disease

Primary objectives:To prospectively assess longitudinal changes in Proteolipid Protein 1 (PLP1) and additional disease-related biomarkers in cerebral spinal fluid (CSF) and blood and evaluate their utility in support of the development of therapies…

Preterm Immune system development and response to Immunization

Primary objective is to study the antibody immune response to routine vaccinations in very preterm infants (GA<32 weeks). Secondary aim is to study the immune system more extensively using flow cytometry, ELISA and single cell transcriptomics…

SARSLIVA 2.0: Dense saliva sampling for studying SARS-CoV-2 transmission dynamics - a household study

primary objectives:SARSLIA 2.0:1. To study SARS-CoV-2 transmission dynamics within households using saliva, with home self-sampling, storage and transport to the laboratory.2. To study the emergence of SARS-CoV-2 in saliva from asymptomatic or pre-…

Active Monitoring versus an Abduction Device for treatment of Infants with Centered Dysplastic Hips, a Comprehensive Cohort Study (TReatment with Active Monitoring (TRAM)-Trial)

The aim of this study is to compare treatment with an abduction device to active monitoring of infants with centered dysplastic hips (Graf type IIa- or IIb or IIc) during the first year of life.

An 18-month, open-label, single-arm safety extension study of an age-and bodyweight-adjusted oral finerenone regimen, in addition to an ACEI or ARB, for the treatment of children and young adults from 1 to 18 years of age with chronic kidney disease and proteinuria

This study has been transitioned to CTIS with ID 2023-504885-50-00 check the CTIS register for the current data. A study to learn more about how safe the study treatment finerenone is inlong-term use when taken with an ACE inhibitor or angiotensin…

Connection Interrupted: Genetic causes and clinical characteristics of hereditary optic neuropathies

The goal of this study is to identify the genetic causes underlying hereditary optic neuropathies and establish genotype-phenotype correlations. This will lead to more reliable prognosis predictions, better genetic counselling and hopefully form a…

A multi-center, open-label study to determine the dose and safety of oral asciminib in pediatric patients with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP), previously treated with one or more tyrosine kinase inhibitors.

This study has been transitioned to CTIS with ID 2023-508129-28-00 check the CTIS register for the current data. The primary objective of this study is to characterize the pharmacokinetic (PK) profile of asciminib in pediatric patients, with the…

Phase II trial of nivolumab for pediatric and adult relapsing/refractory ALK+ anaplastic large cell lymphoma, for evaluation of response in patients with progressive disease (Cohort 1) or as consolidative immunotherapy in patients in complete remission after relapse (Cohort 2)

Primary objectivesCohort 1 Estimate the efficacy of nivolumab treatment in patients with relapsed/refractory ALK+ ALCL in terms of best objective response rate within thefirst 24 weeksCohort 2 Estimate the efficacy of nivolumab treatment as…

Pharmacokinetic-guided dosing of emicizumab in congenital haemophilia A patients - The DosEmi study

This study has been transitioned to CTIS with ID 2024-515528-35-00 check the CTIS register for the current data. Primary objectiveTo determine whether individualized pharmacokinetic (PK)-guided dosing of emicizumab is non-inferior to conventional…

An open-label, first-in-human, dose-escalation/expansion study of SAR443579 administered as single agent by intravenous infusion in adult and pediatric participants with relapsed or refractory acute myeloid leukemia (R/R AML), B-cell acute lymphoblastic leukemia (B-ALL), high risk-myelodysplasia (HR-MDS) or blastic plasmacytoid dendritic cell neoplasm (BPDCN)

This study has been transitioned to CTIS with ID 2023-508357-58-00 check the CTIS register for the current data. The aim of the escalation portion of this study, in which SAR443579 is administered for the first time in humans, is to establish the…