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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: -…

An open label, multi-center roll-over study to assess long-term effect in pediatric patients treated with Tafinlar (dabrafenib) and/or Mekinist (trametinib)

This study has been transitioned to CTIS with ID 2023-509276-42-00 check the CTIS register for the current data. Primary:To assess the long-term safety of treatment with dabrafenib, trametinib or the combination.Secondary:To assess the long-term…

A Randomized, Double-Blind, Placebo-Controlled Study of Galcanezumab in Adolescent Patients 12 to 17 Years of Age with Chronic Migraine - the REBUILD-2

This study has been transitioned to CTIS with ID 2023-505835-11-00 check the CTIS register for the current data. PrimaryTo demonstrate the superiority of galcanezumab versus placebo in the prevention of migraine in an adolescent population (12 to 17…

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.

CANDY MULTIPLEX STUDY

1) Assess the whole genome sequence (WGS) and clinical profile of the MPX families to identify how rare and common genetic variants interact to produce shared versus distinct NDDs and clinical features in individuals, or different combinations of…

Individualized trajectories of upper arm disease progression in Duchenne muscular dystrophy patients

Main objective1. To assess the longitudinal trajectories of upper arm flexor muscle MRI parameters, muscle force and upper arm function in a cohort of ambulant and non-ambulant DMD patients, to assess the value of this muscle as target for…

Clinical safety study on 5-Aminolevulinic acid (5-ALA) in children and adolescents with brain tumors

This study has been transitioned to CTIS with ID 2024-517575-20-00 check the CTIS register for the current data. This study proposes a safety study in children in which tumors are operated on using fluorescence-guided resection which are similar to…

MyopiaX Treatment for the Reduction of Myopia Progression in Children and Adolescents: Safety and Efficacy Investigation

Primary objectiveTo evaluate the effects of treatment with MyopiaX on the rate of myopia progression in children and adolescents as reflected in spherical equivalent refraction and axial length changes at 6months relative to baseline.Secondary…

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…

A nationwide natural history study in Becker muscular dystrophy: Modeling a slowly progressive neuromuscular disease to prepare for clinical trials.

The primary objective is to develop a disease model predicting clinical meaningful changes based on a combination of results of tests on clinical function, muscle MRI, echocardiography and circulating biomarkers applicable in various stages of…

THE IMMUNE SYSTEM IN FOXP1 SYNDROME

Primary Objective: We aim to provide a clear phenotype of the immune system in patients with FOXP1 syndrome. Secondary Objectives:We aim to correlate the immunophenotype of patients with FOXP1 syndrome with frequency and type of infections reported…

A Phase 3, Randomized, Double-blind, Placebo-controlled, Single-dose Study to Evaluate the Efficacy and Safety of Suvratoxumab in Mechanically Ventilated Adults and Adolescents for the Prevention of Nosocomial Pneumonia

Primary:- To evaluate the effect of suvratoxumab on reducing the incidence of nosocomial all-cause pneumonia.Secondary:- To evaluate the safety of a single IV dose of suvratoxumab.- To evaluate the effect of suvratoxumab on reducing the incidence of…

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.

Early prediction of treatment response in children with Inflammatory Bowel Disease with Intestinal Ultrasound

To assess the early predictive value of IUS for therapy response in children with IBD (new onset or known IBD).

Contrasting the Neural Systems of Social and Non-social Threat Regulation in the Adolescent Brain

The general aim for this project would be to understand the neurodevelopmental differences with respect to implicit and explicit forms of threat regulation, by focusing on brain development, sex-steroid hormones and social sensitivity in adolescents…

SIMBA study: Seven Tesla Imaging Biomarkers of Cognitive Outcomes after Treatment for Pediatric Brain Tumor

The primary objective is to examine relationships between neurocognitive performance and MRI parameters of brain vasculature, metabolism, and white matter diffusion in children who have been treated for posterior fossa tumor. Secondary objectives…

A Randomized, Double-Blind, Placebo-Controlled Study of Galcanezumab in Patients 6 to 17 Years of Age with Episodic Migraine - the REBUILD-1

This study has been transitioned to CTIS with ID 2023-505836-36-00 check the CTIS register for the current data. Main Objective:To demonstrate the superiority of galcanezumab versus placebo in the prevention of migraine in (at least) 1 of the…

A Phase 3 Double Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients with Hereditary Angioedema (HAE)

Primary objective: The primary objective of the study is to evaluate the clinical efficacy of ISIS 721744 in patients with hereditary angioedema (HAE).Secondary objective: Evaluate the effects of ISIS 721744 on the quality and pattern of HAE attacks…

Prospective clinical evaluation of Stargardt disease patients with the
appropriate genotype for development of antisense oligonucleotide therapy

The goal of this study is to characterize the natural history of STGD1 patients with the appropriate genotype for AON therapy and to identify the best clinical outcomes to measure therapeutic efficacy in clinical trials following completion of this…

An Open-Label Uncontrolled Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Activity of Nipocalimab in Children Aged 2 to less than 18 years with Generalized Myasthenia Gravis

This study has been transitioned to CTIS with ID 2022-502539-21-00 check the CTIS register for the current data. The purpose of this study is to determine the effect of nipocalimab on total serum immunoglobulin G (IgG) in pediatric participants 2 to…