17 results
Primary:• To evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria (PKU) as measured by mean change in blood Phe levels from baseline to Weeks 5 and 6 (ie, the average of each respective…
This study has been transitioned to CTIS with ID 2024-513041-37-00 check the CTIS register for the current data. Primary objective of this study is to significantly reduce relevant (moderate/severe) parenchymatous brain injury on postoperative MRI…
This study has been transitioned to CTIS with ID 2024-516324-34-01 check the CTIS register for the current data. To evaluate (reduction of) intraoperative blood-loss with and without pre-operative TXA administration, in children undergoing a…
This study has been transitioned to CTIS with ID 2023-505643-39-00 check the CTIS register for the current data. Part A: PrimaryTo evaluate the safety and tolerability of single ascending dose of BIIB115 administered via IT bolus injection to…
This study has been transitioned to CTIS with ID 2023-504884-17-00 check the CTIS register for the current data. The main goal of this study is to find out whether finerenone in combination with either an ACE inhibitor or an ARB can reduce the…
This randomised double-blind placebo-controlled study will investigate the effects of a oligofructose vs a placebo on (change in) stool consistency in healthy children with hard stools. Other parameters to be investigated include (change in) stool…
To evaluate the influence of DSA monitoring, provided by the NarcotrendTM monitor, on the speed of emergence and recovery from GA.
In the present randomised double blind controlled study, we will study the effects of GOS or FOS vs a placebo on stool consistency. Other parameters to be investigated include stool frequency, stool consistency in number of cases (%), painful…
The primary objective is to demonstrate the superiority versus placebo of a 4 weeks intranasal OT administration on oral skills assessed by the Neonatal Oral-Motor Assessment Scale (NOMAS) in infants with PWS aged less than or equal to 3 months at…
Primary: To determine whether three months of prophylactic treatment with co-trimoxazole causes a reduction in the number of days a child experiences at least two RTI symptoms in children aged 6 months to <=10 years with recurrent RTIs, when…
This study has been transitioned to CTIS with ID 2024-510603-11-00 check the CTIS register for the current data. Primary Objective: To evaluate the efficacy of ION373 in improving or stabilizing gross motor function in patients with Alexander…
Primary Objective:To investigate change in fat mass index between 6 and 12 months of age in infants receiving Nuturis® formula feeding versus standard formula feeding during the first 6 months of life.Secondary Objectives:- To investigate change in…
This study has been transitioned to CTIS with ID 2024-512321-84-00 check the CTIS register for the current data. Primary objectives:Evaluation of efficacy of multispecific T-cell transfer in patients with chemo-refractory viral infections after…
In the present randomised double blind controlled study, we will study the effects of GOS vs a placebo on stool consistency in children with hard or lumpy stools. Other parameters to be investigated include stool frequency, stool consistency in…
The main objective is to study the effect of the nutritional intervention versus the control product on white matter development (as assessed using tract based spatial statistics of fractional anisotropy) using a DTI scan at the corrected age of…
This study has been transitioned to CTIS with ID 2023-506269-78-00 check the CTIS register for the current data. Objectives for Part 1: Primary (Efficacy): To demonstrate that the efficacy of fenfluramine (ZX008) 0.8 mg/kg/day is superior to placebo…
The aim of this study is to evaluate if a PPT can significantly decrease - or possibly prevent - the collapse of the trachea in newborns with EA and moderate or severe TM. Additionally, the study aims to determine whether the observed effect of PPT…