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A Phase III, Randomized, Double-Blind, Active Comparator-Controlled Clinical Trial, Conducted Under In-House Blinding Conditions, to Examine the Efficacy and Safety of Aprepitant for the Prevention of Chemotherapy-Induced Nausea and Vomiting (CINV) in Pediatric Patients

The purpose of this current study is to evaluate the efficacy and safety of the 3-day oral aprepitant regimen when administered concomitantly with ondansetron, with or without dexamethasone, in pediatric patients, from 6 months to 17 years of age,…

An outpatient phase 3 efficacy study of Ecopipam (PSYRX 101) in the symptomatic treatment of self-injurious behavior in subjects with Lesch-Nyhan disease.

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…

Effects of melatonin treatment, light therapy, and sleep improvement on psychosocial, cognitive, and behavioural outcomes in children with Delayed Sleep Phase Syndrome and their parents

The general aim of the present study is to investigate, in a longitudinal-experimental design, the effects of melatonin treatment and light therapy in children on sleep, health, and various psychosocial, behavioural, and cognitive outcomes. A second…

A three-arm, randomized, double-blind, placebo-controlled study of the efficacy and safety of two trough-ranges of everolimus as adjunctive therapy in patients with tuberous sclerosis complex (TSC) who have refractory partial-onset seizures

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…

Efficacy of RAD001/everolimus in Autism and NeuroPsychological deficits in children with TSC (RAPIT-trial)

The objective of this study is to determine whether influencing the protein pathway thought to underlie TSC by Everolimus treatment will improve cognitive abilities in these children.

An efficacy, safety and pharmacokinetic study on the short-term and long-term use of METFORMIN in obese children and adolescents.

The primary objective of the METFORMIN study is to determine the efficacy of metformin in combination with lifestyle-intervention in obese children and adolescents with insulin resistance versus placebo with lifestyle-intervention. The secondary…

Reducing the incidence of daily life pain in patients with sickle cell disease

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…

Fear conditioning during specific conditions in antisocial adolescents: a neuroimaging study.

The primary objective is to investigate brain functioning during (reversal) fear conditioning and reward/punishment conditions during different experimental conditions. Secondary objectives include investigation of connectivity between brain…

The NEURAPRO-E Study: A multicenter RCT of Omega-3 Fatty Acids and Cognitive-Behavioural Case Management for Symptomatic Patients at Ultra-High Risk for Early Progression to Schizophrenia and Other Psychotic Disorders

Replication of the study of Amminger et al (2010) in a larger sample.

A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety of AFQ056 in adolescent patients with Fragile X Syndrome

The purpose of the study is to assess the efficacy and safety in adolescents of AFQ056 in treating behavioral symptoms characteristic of patients with FXS. Data from this study will be used to support the registration of AFQ056 in this population…

A DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, MULTICENTER STUDY OF THE EFFICACY AND SAFETY OF PREGABALIN AS ADJUNCTIVE THERAPY IN CHILDREN 4 -16 YEARS OF AGE WITH PARTIAL ONSET SEIZURES

The primary objective of this study is to evaluate the efficacy of 2 dose levels of pregabalin (Level 1: 2.5 mg/kg/day; maximum 150 mg day and Level 2: 10 mg/kg/day; maximum 600 mg day) compared to placebo as an adjunctive treatment in reducing the…

A randomised, multicentre, double-blind, placebo-controlled, crossover trial determining the efficacy of dry powder mannitol in improving lung function in subject with Cystic Fibrosis aged six to seventeen years.

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…

A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy

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…

A Multicentre, Randomised, Placebo-controlled, Double-blinded Study of the Efficacy, Safety, and Pharmacokinetics of Lubiprostone in Paediatric Subjects Aged * 6 Years to < 18 Years with Functional Constipation

To assess the efficacy, safety, and pharmacokinetics of oral lubiprostone 12 or 24 mcg capsules dosed twice daily (BID) (based on subject body weight at baseline) as compared to matching placebo BID, when administered orally for 12 weeks in…

A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

Primary ObjectiveTo evaluate the efficacy of lumacaftor in combination with ivacaftor throughat Week 24 in subjects with cystic fibrosis (CF) who are homozygous for the F508del CFTR mutation on the CF transmembrane conductance regulator (CFTR)…

Trial consisting of an 8-week double-blind placebo-controlled part to evaluate efficacy, safety, tolerability and pharmacokinetics of prucalopride in paediatric subjects with functional constipation, aged ???6 months to &lt;18 years, followed by a 16-week open-label comparator (PEG) controlled part, to document safety and tolerability up to 24 weeks

The primary objective is to evaluate the efficacy of prucalopride compared to placebo for the treatment of functional constipation in a paediatric population, aged *6 months to &lt;18 years. Secondary objectives include:1. Investigation of the…

A Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study of Patient Reported Outcomes in Friedreichâ¤*s Ataxia Patients after withdrawal from Treatment with Idebenone (PROTI Study)

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…

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to 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 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…

Effectiveness and safety of parecoxib in children undergoing scoliosis surgery compared to placebo: a double-blinded randomised controlled trial

Assessing the effectiveness and safety of parecoxib compared to placebo in pain relief children undergoing scoliosis surgery

Assessing the Effect of Missing Doses (Off-Days) of Daily Medication in Patients Stable on Pharmacotherapy for ADHD Receiving Atomoxetine or OROS Methylphenidate: A Parallel Matched Group Clinical Study (On/Off Study)

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…