83 results
The objective of this pivotal study is to assess the PK and PD as well as safety and tolerability of RP103 compared to Cystagon® in patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support the registration…
Primary Objective:To evaluate the Safety of OxabactTM with continued exposure.Secondary Objectives:To obtain additional efficacy data with up to 48 weeks continuous exposure to Oxabact*.
The goal of this project is to develop and test a digital file that is expected to improve the communication between professional caregivers (endocrinologists, nurses, dietitians, social workers, psychologists). Additionally, the communication…
To evaluate if the program *Run2gether* can change physical behavior in children with obesity and/or diabetes, and thereby increase physical fitness level and insulin sensitivity en reduce body weight. An additional goal for children with diabetes,…
1) To assess insulin sensitivity and response in critically ill septic neonates and children. 2) To determine protein balance in septic, critically ill children at baseline and during a Hyperinsulinemic Euglycemic Clamp, while receiving standard or…
The objectives of this proposal are1. to compare the effects and costs of two intensive one-year interventions in severely obese children and adolescents of which the first six months are different with respect to the length of hospitalization;…
Objective of the study:1) To assess insulin sensitivity and response in critically ill septic neonates and children.2) To determine protein balance in septic, critically ill children at baseline and during a HyperinsulinemicEuglycemic Clamp, while…
The objective of the study is to establish the effect of genestein in patients with MPS III on urinary and serum GAGs levels, hair morphology, GAG accumulation in skinbiopsy, cognitive functions and behavior (Piotrowska et al, 2008).MPS III is a…
the objective of the current study is to evaluate the safety and efficacy of enzyme therapy in the Dutch population of patients with MPS I, II and VI; to compare this to the natural course of these diseases; to make an inventory of the direct and…
To evaluate the long-term safety and efficacy of BMN 110 administration at 2.0 mg/kg/qw and 2.0 mg/kg/qow in patients with MPS IVA.
The objective of the study is to establish the effect of genestein in patients with MPS III on urinary and serum GAGs levels, hair morphology, GAG accumulation in skinbiopsy, cognitive functions and behavior (Piotrowska et al, 2008). MPS III is a…
The primary objective of this study is to evaluate the tolerance of a new smoothie-style paediatric sip feed compared to a standard paediatric sip feed for a period of 6 weeks in children in need of oral nutritional support. The secondary objectives…
Primary Objectives * To assess the efficacy of rosuvastatin in paediatric patients with familial hypercholesterolaemia. * To establish long-term safety, tolerability and efficacy of rosuvastatin in paediatric patients with familial…
The primary objectives of this study is:* To determine the safety and tolerability of rhHNS via ascending doses administered via a surgically implanted intrathecal drug device (IDDD) once monthly for 6 months, in patientswith MPS IIIA.The secondary…
The current proposal studies the efficacy of a multifactorial intervention with weight reduction, behavioural therapy, and physical exercise on the severity and control of overweight/obese children with asthma. Also the underlying mechanism of these…
This study will evaluate the efficacy and safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome). This study will compare the effects of 24 weeks of infusions of BMN 110 at…
(1) To generate new clinical efficacy and safety data to support the registration of Insuman Implantable 400 IU/ml,(2) To ensure the continuation of the treatment of the patients already implanted and avoid pump explantation, up to the approval of…
Primary objective: to confirm the efficacy of insulin degludec administered once daily plus mealtime insulin aspart in controlling glycaemia with respect to change from baseline in HbA1c after 26 weeks of treatment. Secundary objective: to compare…
We will examine the feasibility of an experimental condition- in which either a passive or active school morning is simulated- to test the effect of physical activity on cognition and cardiovascular health in children. In addition, we will gather…
The objective of the study is to determine what the clinical value is of measuring GE of liquids in 50 SMID children. The primary study parameter is how often DGE of liquids occurs in this study group. Secondary study parameters include…