156 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: 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 main objective of this pilot study is twofold: 1) To investigate whether and to what extent raped adolescents experience symptoms of posttraumatic stress disorder at maximum 18 months of follow-up, measured by standardised questionnaires and…
(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…
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…
Primary Objective: The primary goal is to evaluate the effectiveness of a prevention program for adolescents. These adolescents are children of parents who receive treatment at a mental health care institution for their internalizing problems (…
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…
The online tailored advice is providing adolescents relevant insight in the negative G-scheme (based on RET). This negative G-scheme leads to inadequate coping strategies. The intervention teaches victims to replace negative irrational beliefs into…
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…
To investigate motor connectivity in vivo in patients with SMA using MR- techniques for brain imaging.We will investigate connectivity of upper motor neurons in patients with SMA compared to disease controls (patients with myopathic disorders) and…
In this study the effect of the administration of NAC on the frequency and severity of pain and painful crises in daily life will be assessed as well as the related frequency and length of hospital admissions and health related quality of life. In…
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 (…
Primary Objective: The primary objective of this study is to understand to which degree the language system of pre-pubertal children is still plastic compared to adults and which brain areas are recruited by these groups when learning an artificial…
Primary objective:To determine sAF levels in children and young adults with T1DM and in children with obesity with or without T2DM and determine the correlation between sAF levels and HbA1c in children and young adults with T1DM and in children with…