2369 results
To evaluate the safety and tolerability of IV administration of SBC-103 in subjects with MPS lllB, Sanfilippo B.
To explore the safety, tolerability and the pharmacokinetic profile of 2-IB when given on top of therapeutic hypothermia.
In the present study we will evaluate the standardized care protocol developed in our outpatient clinic, intended to guide counselling for medication use prior to pregnancy. This protocol guides treatment decisions based on disease activity,…
Primary: The main objective of the study is to evaluate the feasibility, safety and tolerability of allogeneic IL15-activated NK cell infusions in children transplanted for refractory or relapsed leukemia.Secondary: To document immune reconstitution…
To determine the accuracy of the URIKA urinary bladder sensor in measuring the distance between the anterior - and posterior wall of the bladder and to determine the range in bladder diameters at which the child should be alarmed to void.
PrimaryEffective treatment regimes and preventive tools of SIB in CdLS by studying phenomenology, etiology and pathogenesis.Secondary a. Development of a flow chart for daily care (diagnostics; management) b. Evaluation whether the present study can…
PrimairyTo clinically validate a finger prick DBS method compared to conventional venous sampling for the analysis of 5 immunosuppressive and 4 azole antifungal drugs in the pediatric population. Secondairy• Feasibility of the novel finger prick DBS…
In this study one of the goals is to compare 7 tesla MRI with 3 tesla MRI in terms of lesion detection in epilepsy patients and depiction of specific dysplasia-related imaging characteristics. Furthermore, resected tissue will be scanned ex-vivo at…
The current study is a continuation of the cohort 5-8 years post-diagnosis and aims to evaluate:1.The course of the nutritional status, and the related factors and consequences of malnutrition in survivors of childhood cancer until 5-8 years post-…
1. Immunology and Pathology:To determine the immune response of patients with simple and complex appendicitis in order to identify markers which distinguish between simple and complex appendicitis.2.Clinical and Imaging:To analyze the differences in…
Monitoring and evaluation of the Dutch guideline for treatment of aHUS in children and adults during two years.
The main objective of the study is to develop an optimal and individualized asparaginase treatment protocol. The study consist of four sub-studies. The objective of the population pharmacokinetic study is to get more insight in the pharmacokinetics…
To validate two less complex techniques, electrical activity of the diaphragm and ultrasound of the diaphragm, compared to the gold standard (transdiaphragmatic pressures) for monitoring diaphragm function and inspiratory effort during assisted MV…
main objective: Investigate the prevalence of liver fibrosis according to TE values in children enrolled in a HPN-program. Secondary objectives: Investigate the correlation between TE values and ELF test score and APRI in children enrolled in a HPN-…
To determine the reproducibility and validity of specific outcome measures for skeletal and respiratory fatigability in subjects with SMA.
1. Normalization of the abnormal serum TH parameters and thereby improving the clinical condition of the AHDS patients2. Observation of changes in cognitive and motor function.
Main objective:To assess the efficacy of thromboprophylaxis with high prophylactic dose LMWH as compared with standard care without systemic thromboprophylaxis in children treated for primary ALL during asparaginase treatment.Secondary objectives:1…
The main objectie of the study is to map the developmental trajectory of processing perceptual and social information in infants using fNIRS. In addition, with the measurement in 5 month-old children we aim to replicate previous research. The…
To establish a healthy control group for microcirculatory parameters for current and future studies on the paediatric microcirculation.
Primary objective of this study is to improve identification of epileptic HFOs in EEG and MEG, by comparing them to HFOs in corticography. Secondary objectives are to study the predictive value of non-invasively recorded HFOs for disease activity…