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Effectiveness of L-serine dietary supplementation in children with a GRIN2B loss-of-function mutation: n-of-1 series

Assess the potential efficay of L-serine dietary supplementation in patients children with a GRIN2B LoS mutation.

CeRebrUm and CardIac protection with Allopurinol in neonates with critical congenital heart disease requiring cardiac surgery with cardiopulmonary bypass

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…

Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

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…

Oxytocin and reactivity to infant signals in mothers postpartum depression

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-…

The effect of antenatal acetaminophen administration on breathing effort of premature infants at birth: a pilot study

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…

RIC-NEC Phase II Feasibility Randomized Controlled Trial: Remote Ischemic Conditioning in Necrotizing Enterocolitis

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…

Oxytocin treatment in neonates and infants aged from 0 to 3 months with prader-willi syndrome: a study of the safety and efficacy on oral and social skills and, feeding behavior of intranasal administrations of oxytocin vs. placebo (phase iii clinical trial)

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…

Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander Disease

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…

Effect of ALlopurinol in addition to hypothermia for hypoxic-ischemic Brain Injury on Neurocognitive Outcome - a blinded randomized placebo controlled parallel group multicenter trial for superiority (Phase III)

This study has been transitioned to CTIS with ID 2024-511322-31-00 check the CTIS register for the current data. To evaluate whether in newborns with asphyxia and early clinical signs of hypoxic ischemic encephalopathy, early postnatal allopurinol…