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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…

E-TOP - Digital information for parents of very and moderat preterm born infants

The aim of this study is to evaluate the feasibility of the E-TOP module for both VP infants as an addition to the TOP program as well as for MP infants in an adapted TOP program.

Multicentre prospective trial for extracranial malignant germ cell tumours including a randomized comparison of Carboplatin and Cisplatin

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/…

Reduction of blood loss in pediatric osteotomies around the hip
- A randomized placebo-controlled trial with tranexamic acid -

This study has been transitioned to CTIS with ID 2024-516324-34-01 check the CTIS register for the current data. To evaluate (reduction of) intraoperative blood-loss with and without pre-operative TXA administration, in children undergoing a…

International collaborative treatment protocol for infants under one year with KMT2A-rearranged acute lymphoblastic leukemia or mixed phenotype acute leukemia

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…

A Proof-of-Concept Study to Explore the Potential Efficacy of Deferiprone in Patients With
Pelizaeus-Merzbacher disease (PMD)

This study has been transitioned to CTIS with ID 2024-511968-81-00 check the CTIS register for the current data. Studying the effect of deferiprone on the motor function of patients with Pelizaeus-Merzbacher disease.

A phase 3 study of PTC923 in subjects with phenylketonuria

Primary:• To evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria (PKU) as measured by mean change in blood Phe levels from baseline to Weeks 5 and 6 (ie, the average of each respective…

Combined use of induced pluripotent stem cell derived cardiomyocytes and 3D-heart tissue to develop Therapies for pediatric heart failure in Tetratology of Fallot.

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).…

Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

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…

A clinical study to investigate Interferon gamma (IFN*) signature in patients post HSCT and in patients with impaired HSC proliferation pre-transplant

HSCT cohort:* -To investigate the relationship between IFNγ levels and IFNγ activity by measuring CXCL9 levels and the risk of graft failure* -To investigate the relationship between IFNγ levels and IFNγ activity by measuring CXCL9 levels and the…