173 results
The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with the study drug. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a…
The primary objective of this study is: To study the effect of controlled discontinuation of long-term used risperidone, for the treatment of challenging behavior, on behaviour and health. Our hypothesis is that long-term use of risperidone for…
The main objective of this study is to investigate whether a music intervention as complementary treatment is effective in reducing anxiety and distress around surgery. Secondary objectives are effects of music on pain, on preoperative anxiety, on…
The purpose of this study is to assess the safety, tolerability and efficacy of long term drisapersen in subjects with DMD.
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.
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.
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…
1. To identify genetic variants associated with an increased risk of ASD using different genetic models (dominant, recessive, interaction).2. To determine the relationship between different genetic variants and the associated neuropsychiatric ASD…
The objective of this study is to evaluate whether an in- or outpatient initiation of the brace treatment has an influences short and long term brace compliance.
The primary objective is to assess the treatment effect on response rate (MDS: either completeremission [CR], partial remission [PR], or marrow CR; JMML: either clinical completeremission [cCR] or clinical partial remission [cPR]); at Cycle 3 Day 28…
To assess the long-term safety, efficacy, and pharmacokinetics of oral lubiprostone 12 or 24 mcg capsules dosed twice daily (BID) when administered orally for 36 weeks in paediatric subjects with functional constipation. Evaluation of lubiprostone…
The aim of this study is to validate known, and identify novel allelic variants which may play a role in the risk of cisplatin-induced ototoxicity in a substantial international cohort of children diagnosed with cancer.
Primary Objectives:1) To determine immunogenicity of NIP vaccines in preterm infants when vaccinated according to the current Dutch NIP schedule and with rotavirus vaccine following the RIVAR study.2) To unravel the mechanism of immature host…
Research questions: Primary questions:* Are the new point-of-care instruments for the measurement of VOCs in exhaled breath feasible for use in children and adolescents aged 6 to 20 years?* Are these techniques able to differentiate between healthy…
The primary objective of this study will be to determine the pharmacokinetic profile of lorazepam oral liquid 1 mg/ml in the paediatric ICU population, with specific attention for the population aged up to 12 years of age.The secondary objectives…
Primary Objective: To prospectively obtain the (parenteral/enteral) intake of protein, fat, carbohydrates and calories in moderately preterm infants (32-36 weeks) and obtain prospectively growth data in the first 2 years of life. Secondary Objective…
To study sensory processing and hyperexcitability in children with ASD and epilepsy using neurophysiological measurements (EEG/ERP).
The objective of the study is to investigate if it is possible to predict the clinical outcome of a patient with SCN1A related epilepsy based on the findings of advanced genotyping. We aim to answer the following questions:1. Can clinical outcome in…