185 results
The purpose of this trial is primarily to assess the efficacy of inhaled mannitol compared with a true placebo in subjects with cystic fibrosis aged 6 to 17 years.We hypothesize that inhaled mannitol will improve the overall health and hygiene of…
To find out if intravenous MSC is a safe treatment for children with therapy-resistant JIA
Primary Objectives:- Evaluate changes in the immune system upon exposure to FVIII in patients with severe hemophilia A - Identify immunologic predictors of FVIII inhibitor development or tolerance
Primary: To compare the reduction in frequency of partial-onset seizures on each of two trough ranges of everolimus (3-7 ng/mL and 9-15 ng/mL) versus placebo in patients with TSC who are taking one to three AEDs. Key secondary: Ability to completely…
The two primary objectives are to test the hypothesis that (1) structural and functional abnormalities of the fronto-striatal circuits are related to the development of compulsive and impulsive behaviour in ASD and OCD; and that (2) the glutamate…
The aim of this study is to see if we can enhance the effectiveness of the nurse*s comforting capabilities through the addition of maternal scent communication during heellance.
The first objective of this study is to test the effects of a classroom-based, manual-guided intervention Multi-Method Coaching on the skills and the behavioral sensitivity of Dutch primary education teachers in order to improve the relationships…
(i) To compare the DBF response to capsaicin between cystinosis patients, homozygous and heterozygous for the 57-kb deletion, and matched controls.(ii) To compare the skin sensitivity response after capsaicin application between cystinosis patients,…
The primary objective of this study is to examine the effectiveness of the serious game called HealSeeker for improving time management, planning and organisation and social behaviour skills of children with ADHD. The secondary objective of this…
This study has two main objectives. One objective is to investigate which cognitive and affective mechanisms can be influenced through treatment which leads to improved social functioning. We will use a carefully selected set of behavioral,…
Objective: Our general objective is to investigate the neurobiological substrates of a broader ADHD phenotype.
The primary objective to our study is assessing functional outcome, quality of life, and genetic mutations in patients with a cloacal malformation. Functional outcome:- Colorectal functiono Objective: assessing colorectal function through the use of…
The primary objective in this observational study is to determine microcirculatory profile in preterm and term neonates. We would like to determine how the microcirculation changes in time during the phase of adaptation and correlate inter- and…
A:To get insight whether respiratory support in the neonatal population, as artificially ventilation and Nasal Continously Positive Airway Pressure, will give an increased stress or pain respons which only can be detected by a 24 hours SCA…
To investigate long-term outcome, both medical and psychological, of CPR in children.
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…
To determine the maximum tolerated doses (MTDs) and dose-limiting toxicities (DLTs) of nelarabine, etoposide and cyclophosphamide when given in combination to children with T-ALL and bone marrow relapse or T-LL.
•To characterize the natural history and progression of DMD to help inform the design of future studies•To capture biomarkers of safety and disease progression•To provide comparative data for the development of rare exons for which formal controlled…
We aim to increase patient compliance by extending the existing treatment protocol of (oral or intravesical) anticholinergics plus application of CIC with the addition of bladder pressure measurement performed at home during CIC plus access to an…
The primary objective is to investigate the safety and efficacy of LDE225 with respect to overall response rate according to independent central review (ICR).The key secondary objective is to assess LDE225 with respect to progression-free survival (…