62 results
To determine long-term scar quality in patients admitted to a Dutch burn centre with burn wound of indeterminate depth.
Primary objective is to objectively investigate the therapeutic potential of Ivacaftor in Dutch CF patients carrying a mutation associated with residual CFTR function. A secondary objective is to evaluate the correlations between individual…
(1) To determine the frequency of antibody-mediated encephalopathy in children with seizure-related or (sub)acute onset neuropsychiatric disorders. (2) To identify the target auto-antigens of selected seizure-related and (sub)acute onset…
Main objective: To investigate Differences in sustainability of inhalation technique of Diskus® and Autohaler® in the home situation. This will be measured with the inhalation checklist for the Diskus® and Autohaler®. Secondary objectives: * To…
Primary: The primary objective of the Double-blind Acute Phase of the study is to assess clinical response to MMX mesalamine/mesalazine between a low and high dose in children and adolescents aged 5-17 years with mild to moderate UC.The primary…
To assess the pharmacokinetics and safety of naloxegol in paediatric patients ages > 6 months to < 18 years receiving treatment with opioids.
Primary:To evaluate if the proportion of patients in clinical remission on canakinumab 4mg/kg (+/- concomitant NSAID only) who are able toremain on a reduced canakinumab dose (2mg/kg every 4 weeks) or prolonged canakinumab dose interval (4mg/kg…
Primary objective- To measure the effect of sapropterin on diurnal and day to day variations of blood phenylalanine concentrations.Secondary objective- To measure the effect of sapropterin on diurnal and day to day variations of blood tyrosine…
The primary objective of this trial are to assess the clinical usefulness (in terms of sensitivity and positive predictive value of 1. Combined heart rate and movement detection2. And the added value of video and audio detection in a population of…
The overall study objective is to evaluate the safety and efficacy of NiCord®: single ex-vivo expanded cord blood unit transplantation in patients with hematological malignancies following myeloablative therapy.
To investigate the correlation between the CAP-value measured with the FibroScan® device and liver fat percentage measured with 1H-MRS in a well-characterized cohort of obese children at risk of having fatty liver disease.
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.
To find new genes causing congenital MPHD associated with the characteristic MRI abnormalities of ectopic posterior pituitary gland and absent/thin pituitary stalk.
The objective of the study is to identify genetic causes of developmentalanomalies of the face and skull (craniofacial anomalies), such as orofacial clefting, craniosynostosis, abnormal dental development and rare craniofacial syndromes.
The main objective is to determine whether children with CF are able to achieve an inhalation manoeuvre as required for aerosol generation and transport into the peripheral airways with the test inhaler. The secondary objectives are to determine…
The aim of this prospective cohort study is threefold: (1) for a better understanding of MTSS' underlying pathophysiology, (2) to assess the QU's reliability in healthy subjects and subjects with MTSS and (3) to assess the validity of QU…
It is expected that LF111 will offer benefits to adults in the form of efficacy similar to COCPs, absence of estrogen-related risks, reduced tendency to weight gain and extended dosing window. It is likely that adolescents will share these benefits…
Primary Objective is to determine whether (A) patients with dystonia(plus) syndromes exhibit more psychiatric symptoms compared to a healthy control group and (B) whether dystonia patients show an altered serotonin metabolism (serotonin transporter…
Primary ObjectivesPhase 1 portion:• to determine the maximum tolerable dose (MTD) of cytarabine (up to 2 g/m2/day x 5) that can be administered on Days 8-12 following treatment with DACOGEN 20 mg/m2/day on Days 1-5 of a 28 day cycle. • to determine…
The Cardiomyopathy Registry Study (CARS)-II aims to1) Create a national cohort with biorepository of patients with paediatric DCM2) To elucidate (epi) genetic causes of iDCM in a uniform and state of the art way.3) Identify clinical risk factors for…