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TRAILS - Tracking the next generation

The proposed study has 2 main aims: 1) Understanding the complex interplay of determinants of parenting and examining how specific parenting styles explain associations between parent characteristics (such as personality or mental and physical (ill…

Fluorescence visualisation during lymph node procedure in pediatric patients

The standard-of-care sentinel lymph node (SLN) procedure in paediatric cancer patients consists of a preoperative intradermal injection with 99mTechnetium-nanocolloid as radiotracer in combination with an intraoperative injection of blue dye for…

A Phase I/II study of Brigatinib in pediatric and young adult patients with ALK+ Anaplastic Large Cell Lymphoma, Inflammatory Myofibroblastic Tumors or other solid tumors

This study has been transitioned to CTIS with ID 2024-513412-10-00 check the CTIS register for the current data. Phase 1: • To determine the MTD/RP2D regimen of brigatinib monotherapy when administered in pediatric and AYA patients with ALK+ ALCL or…

TEMOkids study (ORP-TMZ-I- b): A Population pharmacokinetic, acceptability and safety study for KIMOZO, a paediatric oral suspension of temozolomide

Primary objective:• Evaluate PK parameters of the oral suspension of temozolomide in the paediatric population aged 1 year and over.Secondary objectives:• Evaluate the safety of the oral suspension of temozolomide,• Evaluate the acceptability of the…

A feasibility study to evaluate PDE MAX, a food for special medical purposes (FSMP) for use in the dietary management of Pyridoxine Dependent Epilepsy (PDE) with regards to acceptability, tolerability, adherence and effect on metabolic control.

To evaluate product acceptability, tolerance and adherence during an 8-week intake of PDE MAX in patients with Pyridoxine Dependent Epilepsy.To evaluate the effects on metabolic control by comparing the changes from baseline after an 8-week intake…

A phase II trial of [177]Lutetium-DOTATATE in children with primary refractory or relapsed high-risk neuroblastoma

This study has been transitioned to CTIS with ID 2023-503684-42-00 check the CTIS register for the current data. Main Study Objective• To confirm the dose and assess response to single agent 177Lutetium-DOTATATE treatmentin patients with relapsed or…

Studying respiratory infections and colonisation in children using daily minimally-invasive nasal sampling

Primary: Associate acquisition of pneumococcal colonisation with levels of pre-existing polysaccharide specific memory B cells. Secondary: a) To validate the use of synthetic absorptive matrices (SAM) for detection of other (non-pneumococcal)…

Protecting against Respiratory tract Infections through human Milk Analysis.

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.…

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 non-traditional complex intervention for the treatment of paediatric sleep problems.

Assessing the feasibility of the research procedures, the burden on participants, the degree of dropout and reason, the interest in participating.What is the main treatment effect for parents / carers motivating them to participate in this study?•…

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…

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 Phase 1/2 Open-Label, Multicenter, Dose Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects with Early Infantile Krabbe Disease (Globoid Cell Leukodystrophy) (GALax-C)

This first-in-human study is planned to include both a dose ranging phase and a confirmatory phase, with the intention that all data collected throughout the study may support registration. The dose ranging phase will be conducted in cohorts of 3-4…

International cooperative prospective study for children and adolescents with standard risk ALK-positive anaplastic large cell lymphoma (ALCL) estimating the efficacy of Vinblastine.

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…

An open-label, single-arm, multicenter study of intracerebral administration of adeno-associated viral vectors serotype rh.10 carrying the human N-sulfoglycosamine sulfohydrolase (SGSH) cDNA for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA)

Primary: To assess the efficacy of intracerebral delivery of AAVrh.10SGSH gene therapy (LYS-SAF302) in improving or stabilizing the neurodevelopmental status of MPS IIIA patients after 24 months (main cohort), compared to the expected evolution…

Evaluation of genetic, enzymatic, biochemical and clinical characteristics of OCTN2/CPT2/CACT/BKT deficiency to determine if new born screening is useful and feasible

To survey the clinical and biochemical characteristics of individuals diagnosed with OCTN2, CPT2, CACT or BKT deficiency or suspected with OCTN2 deficiency after NBS in the Netherlands to aid the decision on whether or not to include these disorders…

Third population-based immune surveillance study used for the evaluation of immunity against SARS-CoV-2

To assess achieved immunity against COVID-19 across the different age groups in The Netherlands by testing a representative part of the Dutch population for the presence of SARS-Cov-2 specific antibodies in serum

Selective fetal growth restriction in monoChOrioNic Twins - an inteRnAtional inveSTigation

Primary objective: To improve outcome prediction in sFGR by developing a prediction model at the time of sFGR diagnosis and by evaluating specific ultrasound parameters throughout the pregnancy. Secondary objectives: - To increase our knowledge of…

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…

Prospective Analysis of Pharmacokinetic Infliximab data in Paediatric Inflammatory Bowel Disease patients

This study has been transitioned to CTIS with ID 2023-507352-72-00 check the CTIS register for the current data. The primary study objective of our study is to assess the efficacy of an IFX intensified induction scheme vs. a standard dosing schedule…