221 results
The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with the study drug. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a…
The purpose of this study is to assess the safety, tolerability and efficacy of long term drisapersen in subjects with DMD.
The objective of this study is to obtain physiological data on lung aeration during pulmonary transition in newborn infants.
To evaluate the effects of long-term intranasal oxytocin on social behaviour in children with PWS and to compare the effects of different doses and frequencies of oxytocin administration
To evaluate the safety and tolerability of IV administration of SBC-103 in subjects with MPS lllB, Sanfilippo B.
To determine whether the attenuated salivary cortisol response in children with asthma can be explained by the maintenance use of inhaled cortiocosteroids, the chronic inflammatory disease, or the attenuated exercise capacity.
Determine efficiency and safety of two different doses of pregabalin as compared to placebo.
1. To identify genetic variants associated with an increased risk of ASD using different genetic models (dominant, recessive, interaction).2. To determine the relationship between different genetic variants and the associated neuropsychiatric ASD…
Primary:The purpose of this study is to evaluate the safety, tolerability, orally administered LMI070 in patients with Type 1 SMA.pharmacokinetics (PK), pharmacodynamics (PD) and efficacy; and to estimate the Maximum Tolerated Dose (MTD) and optimal…
Safety (Primary) ObjectiveThe primary objective for this study is as follows:- To evaluate the safety and tolerability of cobimetinib in children and young adults, including estimation of the maximum-tolerated dose (MTD) or the maximum administered…
To assess the pharmacokinetics and safety of naloxegol in paediatric patients ages > 6 months to < 18 years receiving treatment with opioids.
The proposed study aims to bring neuroimaging and genetics closer to the child/adolescent psychiatric clinic by developing automated classifiers, and obtain associated multimodal biomarker profiles. Such biomarkers are prospected to, aid in clinical…
To further characterize the transport of phenylalanine and other large neutral amino acids across the blood-brain barrier in PKU patients with high and low brain vulnerability to elevated blood phenylalanine concentrations, we will investigate…
With this research project we will investigate the relationship between SSRI concentration in the (pre)term neonate and clinical short and long term effects on the neonate after exposure to SSRI*s during pregnancy and lactation. Furthermore, the…
Primary ObjectivesThe primary objectives are to evaluate in infants who are hospitalized with RSV infection:• the pharmacokinetics of JNJ-53718678 after multiple oral doses;• the safety and tolerability of JNJ-53718678 when administered for 7 days.…
Verification of functional tests with the LoRRca, a next generation ektacytometer, on red blood cells of healthy individuals with or without comorbidities/risk factors.
To evaluate the long-term safety and tolerability of pregabalin in pediatric subjects 1 month through 16 years of age with partial onset seizures and pediatric and adult subjects 5 to 65 years of age with (PGTC) seizures.
Primary Objective: To assess whether early postoperative AED withdrawal improves cognitive function compared to late withdrawal.Secondary objectives: * To confirm safety of earlier AED withdrawal; we will assess eventual seizure freedom, seizure…
To assess in paediatric patients with ASD with and without (first-time) antipsychotic medication• the prevalence and incidence of insulin resistance and other metabolic abnormalities• the risk factors for insulin resistance and other metabolic…
To address the hypothesis that a potato derived protease inhibitor cream significantly limits perianal dermatitis in patients after colorectal surgery.