236 results
The primary objective of the PROTI study is to establish whether patients can correctly determine which treatment assignment they received during the randomised phase of the trial.The key secondary objective is to compare the rate of withdrawal from…
The primary objective of this study is to assess the efficacy of Ecopipam to reduce self-injurious behaviors (SIB) in adults and children with Lesch-Nyhan Disease (LND) in an outpatient setting. The secondary objectives of this study are to assess…
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…
The main objective of this study is to investigate the effect of a single tDCS session in children with mild CP on different motor tasks. The second objective is to identify the optimal site of stimulation. In tDCS research the electrodes can be…
The primary objective is to test the hypothesis that once daily tadalafil administered orally for 48 weeks lessens the decline in ambulatory ability as measured by the 6MWD compared to placebo in boys with Duchenne muscular dystrophy (DMD). Two…
To evaluate the effects of intranasal oxytocin versus placebo on social behaviour and also on eating behaviour in children with PWS.
Primary: safety and tolerability of belimumab in a pediatric population (5-17 y) with SLE.Secondary: PK, efficacy, quality of life.
To assess the efficacy of a loading dose of intravenous salbutamol in children admitted to a PICU for severe acute wheeze or severe acute asthma. Efficacy is measured by the reduction in asthma score (Qureshi) at 1 hour after administration of the…
Specific AimsAs reviewed above, childhood anxiety disorders are highly prevalent and debilitating, and there is an urgent need for improvement of current treatment strategies. An innovative, but scarcely examined, treatment option for childhood…
The primary objective of this study is: To study the effect of controlled discontinuation of long-term used risperidone, for the treatment of challenging behavior, on behaviour and health. Our hypothesis is that long-term use of risperidone for…
To evaluate the ability of ataluren to improve pulmonary function relative to placeboTo determine the effect of ataluren on:1. Pulmonary symptoms2. General well-being3. Health-related Quality Life (HRQL)
Primary: The primary objective of the randomized treatment epoch and for the overall study is to demonstrate that subcutaneous canakinumab administered every 4 weeks is superior to placebo in achieving a clinically meaningful reduction of disease…
Primary objective* To improve the response rate to treatment of severe steroid-refractory acute GvHD grade II-IV (with gut and/or liver involvement) by early addition of MSC to standardized second line treatmentSecondary objectives* To study the…
Objectives: The primary aim of this study is to provide evidence for the effect of ITB treatment on the level of activities in dystonic CP patients.
Posner spatial cueing task will be used to investigate electrophysiological differences in brain oscillatory lateralization during attentional shifts in children with and without ADHD. We hypothesize that the observed difference in the ability to…
Primary Objective: To investigate the behavioral effects of controlled discontinuation as well as the feasibility of discontinuing currently ongoing treatment with risperidone in children and adolescents with behavioral problems who have used…
The primary objective of the proposed study is to investigate whether bumetanide therapy indeed reduces autistic symptomatology. Important secondary goals of this project are to determine whether bumetanide will improve specific behavioral,…
To assess the efficacy of GWP42003-P as an adjunctive antiepileptic treatment compared with placebo, with respect to the percentage change from baseline during the treatment period of the study in convulsive seizure frequency. The dose response…
The primary objective is to compare the efficacy and safety of SD-101-6.0vs. SD- 101-0.0 (placebo) in patients with Simplex, RecessiveDystrophic, or Junctional non Herlitz Epidermolysis Bullosa.The primary endpoint is the complete closure of the…
The primary objective of the study is to assess the effect of missed doses of atomoxetine and OROS methylphenidate in ADHD patients who are stable on pharmacotherapy based on the patient*s daily behavior as assessed by the Daily Parent Report of…