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Bumetanide in Autism Medication and Biomarker study

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,…

Acute Nutritional Ketosis in VLCAD Deficiency: testing the metabolic basis for therapeutic use

To test if acute NK boosts muscle mitochondrial function in vivo in patients with VLCADD in order to establish a rational basis for therapeutic use in this disorder. A secondary objective is to gather data to test the working hypothesis that the…

A Multi-center, Randomized Parallel Group, Placebo-Controlled Double-Blind Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of Belimumab, a Human Monoclonal Anti-BLyS Antibody, Plus Standard Therapy in Pediatric Patients with Systemic Lupus Erythematosus (SLE) (BEL114055)

Primary: safety and tolerability of belimumab in a pediatric population (5-17 y) with SLE.Secondary: PK, efficacy, quality of life.

A phase 2, double-blind, randomized, placebo-controlled pharmacokinetic trial in two parallel groups to investigate possible drug- drug interactions between stiripentol or valproate and GWP42003-P in patients with epilepsy.

Primary Objective: To determine whether GWP42003-P affects the pharmacokinetic (PK) profile of stiripentol (STP) or valproate (VPA).Secondary Objective: To assess the safety and tolerability of GWP42003-P in the presence of STP or VPA.To assess…

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.

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 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, parallel group, multi-centre, 2-week treatment study to evaluate the safety and efficacy of fluticasone furoate nasal spray (FFNS) 110 mcg, administered either once daily or twice daily, compared with placebo, as effective monotherapy in the treatment of uncomplicated acute rhinosinusitis (ARS) in adult and adolescent subjects 12 years of age and older.

The objective of this study is to evaluate the safety and efficacy of two doses of FFNS (110 mcg once daily and 110 mcg twice daily) compared to placebo as monotherapy in the treatment of adult and adolescent subjects 12 years of age and older with…

The effect of intranasal insulin on development and behaviour of children with Phelan-McDermid syndrome

The aim of this project is to validate the hypothesis that intranasal insulin improves development and behaviour in children with Phelan-McDermid syndrome.

A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy

Primary objective: • To assess the efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 24 weeks in ambulant subjects with DMD.Secondary objectives:• To assess the safety and tolerability of 2 different dosing…

A Phase 2/3, Randomized, Double-Blind, Multicenter, Multinational, 4-Arm,
Controlled, Dose-Ranging Study to Evaluate Efficacy and Safety of Teplizumab
(MGA031), a Humanized, FcR Non-Binding, Anti-CD3 Monoclonal Antibody, in
Children and Adults with Recent-Onset Type 1 Diabetes Mellitus

Primary:The primary objective of this study is to assess, relative to placebo, the efficacy, tolerability, andsafety of teplizumab when administered according to 3 different teplizumab dosing regimens insubjects with recent-onset (onset within past…

The effect of nebulisation of ipratropiumbromide on oxygenation and end-expiratory lung volume in mechanically ventilated children: a pilot study

We hypothesize that nebulisation of ipratropiumbromide results in decreased production of sputum resulting in a better lung aeration (defined by an increase in EELV) and improved oxygenation (as defined by the PaO2/FiO2 ratio and the oxygenation…

A phase 2, double-blind, randomized, placebo-controlled pharmacokinetic trial in two parallel groups to investigate possible drug- drug interactions between stiripentol or valproate and GWP42003-P in patients with epilepsy.

Primary ObjectiveTo determine whether GWP42003-P affects the pharmacokinetic (PK) profile of stiripentol (STP) or valproate (VPA).Secondary Objective(s)To assess the safety and tolerability of GWP42003-P in the presence of STP or VPA.

The effect of Lamotrigine on cognitive deficits associated with Neurofibromatosis type 1: a phase II randomized controlled multi-centre trial (NF1-EXCEL)

The objective of this proposal is to determine the effect of Lamotrigine on cognitive functioning and neurophysiology in adolescents with NF1.

European phase-II clinical trial evaluating efficacy of low dose rhIL-2 in patients with recently diagnosed type 1 diabetes

Primary objective1. To evaluate efficacy of ILT-101 for the preservation of residual pancreatic β cells function2. To select the optimal regimen of administration of ILT-101.Secondary objectivesTo assess:1. Tregs expansion after an induction period…

A Phase 2, Randomized, Double blind, Controlled Study to Evaluate the Safety and Efficacy of VX 440 Combination Therapy in Subjects Aged 12 Years and Older With Cystic Fibrosis

1. Primary Objectives• To evaluate the safety and tolerability of VX 440 monotherapy and VX 440 in dual and triple combination with VX-661 and IVA• To evaluate the efficacy of VX 440 monotherapy and VX 440 in dual and triple combination with VX 661…

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy of Lumacaftor/Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis Who Have an A455E-CFTR Mutation

Primary ObjectiveTo evaluate the efficacy of lumacaftor/ivacaftor combination therapy (LUM/IVA) in subjects with cystic fibrosis (CF) 12 years of age and older who have at least one A455E mutation.Other Objectives* To explore the association between…

Randomized, double-blind, placebo controlled, multi-centre superiority phase II study to evaluate the safety, pharmacokinetic, efficacy of gabapentin liquid formulation as add-on to morphine in children from 3 months to less than 18 years of age experiencing severe chronic neuropathic or mixed pain.

The primary objective of this study is to assess the efficacy of gabapentin as add-on to morphine for the treatment of severe chronic neuropathic or mixed pain in children from 3 months to less than 18 years of age. Secondary objectives 1. To assess…

Genistein as an add-on treatment for CF?

Our primary objective is to investigate whether a further gain in efficacy of oral Ivacaftor treatment can be reached by co-supplementation of genistein, as suggested by their highly synergistic action in intestinal organoidsOur secondary objectives…

A multi-centre, phase II, randomized, double-blind, placebo-controlled study to investigate efficacy and safety of sevuparin infusion for the management of acute vaso-occlusive crisis (VOC) in subjects with sickle-cell disease (SCD)

Primary objective:The primary objective of this study is to assess the time to painful VOC resolution, measured from the first dose of sevuparine given to achievement of crises resolution, as compared to placebo. Secondary objectives: The secondary…