41 results
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…
This study has been transitioned to CTIS with ID 2024-518684-35-00 check the CTIS register for the current data. The current project proposal continues on our findings of the performed prospective cohort study, aiming to develop a biomarker guided…
To validate FDA-approved dosing recommendation for once daily darunavir/ritonavir in children 6-12 years old
To minimize the occurrence of sub-therapeutic and supra-therapeutic C0 of tacrolimus on day 3, 7 and 10 after transplantation by basing the starting dose of tacrolimus on a dosing algorithm, rather than the standard bodyweight-only-based approach.…
The main purpose of this study is to determine the appropriate age (10, 12 or 15 years) for a second MenC conjugate (MenCC) vaccine immunization in Dutch children that received a primary MenCC vaccination at a young age. A conclusion will be based…
The primary objective is to evaluate the safety of ReFacto AF in previously untreated patients (no prior exposure to factor products or any blood products) of less than 6 years of age.
The main objective of this study is to determine if Low-Flow is non inferior in reducing apneic spells compared to caffeine. Secondary objectives are investigating if these treatments have an effect on the need of supplemental ventilation and on the…
Primary objective: Assessment of the value of the short-term (2 weeks) response of IGF-I (peak IGF-I SDS) to GH in a dosage of 1.4 mg/m2/day (as part of a dose-escalation IGF-I generation test) in comparison to baseline IGF-I to predict the 1 year…
What is the function of the forearm after a torus fracture in children at an age of 4 to 10 with different immobilisation techniquesWhat is the time that children after a torus fracture are using their limb, without making use of a form of…
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…
The potential for risperidone to induce hyperprolactinemia in pediatric patients is well known.However, the effects of drug-induced serum prolactin elevations on growth and sexual maturation havenot been as well characterized in pediatric…
To study whether a novel plaster (Rapydan) is more successfull in diminishing pain/ discomfort than the usual plaster (EMLA).
Primary: To determine trends in nasopharyngeal colonization with total vaccine- and non-vaccine serotypes of S. pneumoniae in healthy 11-month-old and 24-month-old infants who have been immunized according to the Dutch National Immunization Program…
To protect children with hiv and children using immune-suppressive medication against hepatitis A and B, and to determine the immunogenicity of the combined hepatitis A and B vaccine in these children.
To estimate the percentage of quantitative RT-PCR negative pediatric CML patients in which Imatinib discontinuation result in sustained complete molecular remissionTo determine whether restarting of Imatinib in case of molecular relapse results in a…
A Cross-sectional Study of Renal Function in Treatment-naïve, Young Male Patients with Fabry Disease
The objectives of this study are (1) to document renal function and other Fabry disease manifestations across age in treatment-naïve, young male patients with Fabry disease; and (2) to provide a reference group for comparison with interventional…
Primary objectiveTo determine the development of the cellular immune response (plasma B cells and memory B-cells), immediately before and after the booster of the 3+1 Prevenar® vaccination schedule at 11 months of age and before and after the…
The objective of the study is two-fold:First, we want to improve the design of the study for future extension, by critical evaluation of the efficacy, the instruments used and the outcome measures. Second, we want to assess the cognitive and…
The objectives are to:A. Identify subjects with VWD who may benefit from prophylaxis by determining patterns of bleeding prior to evaluation for enrollmentB. Study the effect of prophylaxis on bleeding frequencyC. Establish optimal treatment…
The objective of the study is to assess the long-term safety of tacrolimus ointment 0.03% or 0.1% in the treatment of subjects with atopic dermatitis under actual use conditions, including the risk of developing cutaneous or systemic malignancies.…