14 results
At least 90% of the subjects, with 95% confidence, will tolerate the test formula.
The test product supports normal physical growth in healthy term infants
The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia…
Assess the potential efficay of L-serine dietary supplementation in patients children with a GRIN2B LoS mutation.
Primary objective of this study is to significantly reduce relevant (moderate/severe) parenchymatous brain injury on postoperative MRI for the total group (both prenatal and postnatal diagnosis) and the prenatal diagnosis group, which is needed to…
The primary objective of this study is to evaluate the feasibility of antenatal paracetamol/placebo administration to pregnant women 0.5-2 hours prior to birth.The secondary objective of this study is to compare the effect of antenatal acetaminophen…
In a randomized control trial (RCT) with mothers with postpartum depressive symptoms the following main hypotheses will be tested:• Oxytocin will promote more sensitive caregiving during mother-infant interactions and while interacting with a life-…
To evaluate whether in newborns with asphyxia and early clinical signs of hypoxic ischemic encephalopathy, early postnatal allopurinol compared to placebo administered in addition to standard of care (including therapeutic hypothermia if indicated)…
Primary Objective: To evaluate the efficacy of ION373 in improving or stabilizing gross motor function in patients with Alexander disease Secondary Objectives: To further evaluate the efficacy of ION373 in improving or stabilizing disease…
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…
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…
To perform a double-blind randomized placebo controlled multicenter study with darbepoetin in infants with MRI confirmed PAIS and to investigate whether darbepoetin can reduce brain injury in neonates who suffered from perinatal arterial ischemic…
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…
Primary Endpoint: Feasibility of including patients in the study, randomization and delivery of the intervention. To assess the primary endpoint, we will examine the probability that: (1) a screened patient is eligible; (2) an eligible patient…