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Non-invasive assessment of skeletal muscle involvement in Duchenne muscular dystrophy

To develop non-invasive markers for tissue changes in skeletal muscle using MR methods

Immune response after Human Papillomavirus vaccination in patients with automimmune disease

Objective: The primary goal of the current study is to study the immunogenicity and safety of HPV vaccination in patients with an autoimmune disease. Based on retrospective analysis with other vaccines we hypothesize that patients with autoimmune…

Reorganisation of the sensorimotor cortex following early unilateral brain damage

1- to describe the presence and severity of hemiplegic CP on the basis of neonatal MRI findings and correlate the involvement of different structures (basal ganglia, posterior limb of the internal capsule and hemisphere) with the size of the lesion2…

Pharmacogenetics of Asthma Medication in Children: Medication with Anti-inflammatory effects with special focus on Neutrophils.

The overall aim of the project is to define effects of genetic and non-genetic variants on pharmacological treatment response in children with asthma. The following questions will be answered:- What are the characteristics of children who do not…

A Multicenter, Prospective, Long-term Registry of Pediatric
Patients with Crohn*s Disease and Ulcerative Colitis

The objective of this registry is to obtain long-term safety and clinical statusinformation on pediatric patients with IBD (ie, CD, UC, or IC).

A systematic metabolic approach to the evaluation of nutrition in Rett syndrome according to the cardiorespiratory phenotype in Dutch Rett girls.

The aims of this pilot study are to describe the nutritional status of a well-defined group of Dutch RTT girls with complete clinical, molecular and neurophysiological work-up (full description is presented in the section *study population*) and to…

Impulsivity, empathy and paranoia and the role of gene-environmentinteractions in the aggressive-delinquent phenotype

This research proposal focuses on (i) the development and implementation of experimental approaches to identify dimensions of impulsivity, empathy and paranoia. (ii) Studying the gene-environmentinteractions which are expected to play a role in the…

A randomized, double-blind, placebo controlled, parallel group, multi-centre, 2-week treatment study to evaluate the safety and efficacy of fluticasone furoate nasal spray (FFNS) 110 mcg, administered either once daily or twice daily, compared with placebo, as effective monotherapy in the treatment of uncomplicated acute rhinosinusitis (ARS) in adult and adolescent subjects 12 years of age and older.

The objective of this study is to evaluate the safety and efficacy of two doses of FFNS (110 mcg once daily and 110 mcg twice daily) compared to placebo as monotherapy in the treatment of adult and adolescent subjects 12 years of age and older with…

A Worldwide, Open Label, Clinical Trial to Examine the Long Term Safety and Tolerability of Rizatriptan in Pediatric Migraineurs for the Treatment of Migraine With or Without Aura

The objective of this study is to test the safety of the research study drug, MK-0462 (rizatriptan) and to test the ability of study drug to relieve or reduce migraine for the study population.

A randomized, double-blind, placebo controlled, single-dose study to assess the initial efficacy of canakinumab (ACZ885) with respect to the adapted ACR Pediatric 30 criteria in patients with Systemic Juvenile Idiopathic Arthritis (SJIA) and active systemic manifestations

The primary objective of this study is:To demonstrate that the percentage of patients who meet the adapted ACR Pediatric 30 criteria at Day 15 is higher with canakinumab compared to placebo.Secondary objectives of this study are:* To evaluate the…

A study on the effects of a behavioral-management strategy on the efficiency in Special Education

The main goal of this study is to test the effects of a classroom-based behavior-management strategy (Taakspel) on the emotional and behavioral functioning of the students, the wellbeing and perceived efficacy of the teachers, and the teacher-…

An Open-Label, Randomized, Phase 3 Trial to Evaluate the Efficacy and Safety of Aztreonam 75 mg Powder and Diluent for Nebuliser Solution (AZLI) versus Tobramycin Nebuliser Solution (TNS) in an Intermittent Aerosolized Antibiotic Regimen, in subjects with Cystic Fibrosis followed by an Open Label, Single Arm Extension

The primary objective of this study is to assess the comparative safety and efficacy ofAztreonam Lysine for Inhalation (AZLI) and Tobramycin Nebuliser Solution (TNS) in adultand pediatric cystic fibrosis (CF) patients aged 6 years or older with…

The Ganesh Study. Risk factors for diabetes and cardiovascular disease in Surinamese South Asian children aged 5 through 12 years and the relation to anthropometry.

Aim of this study is to acquire more insight in the relation between risk factors for cardiovascular disease and type 2 diabetes and anthropometric measures such as BMI, waist circumference and body fat percentage and compare the results between the…

Pathogenesis of community-acquired pneumonia, chronic obstructive pulmonary disease and cystic fibrosis: mechanism of inflammatory response and the role of Th17 differentiation

To assess whether Th17 cells play a central role in COPD, CF and pneumonia pathogenesis

ACNS 0331: a study evaluating limited target volume boost irradiation and reduced dose craniospinal radiotherapy (18.00 Gy) and chemotherapy in children with newly diagnosed standard risk medulloblastoma: a phase III double randomized trial.

The aim of the study is to assess if a dose reduction of craniospinal irradiation in children with a medulloblastoma does not reduce event free survival (EFS) or overall survival (OS). Furthermore it will be assessed if a field reduction (only tumor…

Bortezomib (Velcade®): A feasibility and phase II study in childhood relapsed acute lymphoblastic leukemia

To determine the antileukemic activity of combination chemotherapy including bortezomib as reinduction therapy in childhood relapsed/refractory ALL.

Clinical and molecular genetic aspects of idiopathic epilepsies

(1) Identification of novel loci and genes in which mutations are responsible for the development of inherited epileptic syndromes. (2) Establishing genotype-phenotype correlations; defining the yield of mutation screenings in specific patient…

To define the optimal glucose intake in critically ill children

primary objective:-to define the optimal glucose intake by quantifying endogenous glucose production and fractional gluconeogenesis in critically ill children.secundary objectives:-determine glucose utilization in relation with exogenous glucose…

A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms

The objectives of this open-label study are to evaluate the efficacy (GL-3 clearance), pharmacokinetics (PK), and safety parameters (including immunogenicity) for 2 alternative dose regimens of Fabrazyme (0.5 mg/kg every 2 weeks [q2w] and 1.0 mg/kg…

A randomized, double-blind, placebo controlled, withdrawal study of flare prevention of canakinumab (ACZ885) in patients with Systemic Juvenile Idiopathic Arthritis (SJIA) and active systemic manifestations

The primary objectives of this study are:Part II: to demonstrate that the time to flare in Part II is higher with canakinumab than with placebo.Part I: to assess if canakinumab allows tapering of steroids as per protocol in at least 25% of…