Bilirubin Neurotoxicity (BN) and Neurodevelopmental Impairment (NDI) in Extremely Preterm (EP) Infants:...
Bilirubin EncephalopathyThe purpose of this study is to evaluate the effect of usual versus reduced lipid intake on unbound bilirubin levels, brainstem auditory evoked responses, and neurodevelopmental outcome at 2 years in extremely preterm infants.
A Clinical Trial of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and...
SCN2A-DEEEpilepsyThe goal of this trial is to learn about the effect of PRAX-222 in pediatric participants with early onset SCN2A developmental and epileptic encephalopathy (DEE), aged 2 to 18 years.
PROSPECTIVE, MULTI-CENTER, OPEN-LABEL, SINGLE-ARM REGISTRATION TRIAL OF THE TUBRIDGE FOR THE TREATMENT...
Brain DiseasesAneurysm2 moreA trial to evaluate the safety and efficacy of the Tubridge flow-diverter stent for the treatment of intracranial wide-necked, small to medium-sized aneurysms.
Upper Limb Rehabilitation Using Virtual Reality in Children With Cerebral Palsy (RV-REEDUC)
Cerebral PalsyBrain DiseasesThis study aims to evaluate the effect of a virtual reality rehabilitation protocol on visuo-motor coordination and upper limb functional abilities in children with unilateral or bilateral cerebral palsy.
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like...
AdrenoleukodystrophyBatten Disease14 moreThe primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of metabolism who have evidence of early demyelinating disease in the central nervous system (CNS) who are undergoing standard treatment with unrelated umbilical cord blood transplantation (UCBT). The secondary objective of the study is to describe the efficacy of UCBT with intrathecal administration of DUOC-01 in these patients.
Neuroprotective Effect of Autologous Cord Blood Combined With Therapeutic Hypothermia Following...
Hypoxic Ischemic EncephalopathyCerebral InfarctionThis study examines the effect of cord blood in the treatment of newborn infants with neonatal encephalopathy in combination with hypothermia, which is the standard treatment for this condition. The hypothesis is that the cord blood + hypothermia combination will produce better neuroprotection than the standard treatment of hypothermia alone.
Cerebral Pulsatility Index Compared To Mean Arterial Blood Pressure Guided Protocol In Sepsis Induced...
SepsisNorepinephrine1 moreThe aim of our study is to compare between transcranial doppler pulsatility index and mean arterial blood pressure in guiding management of sepsis induced encephalopathy.
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Mucopolysaccharidosis DisordersHurler Syndrome27 moreThis single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).
Fenfluramine for the Treatment of Different Types of Developmental and Epileptic Encephalopathies:...
Refractory EpilepsySYNGAP1 Encephalopathy4 moreThis study is a pilot non-controlled clinical trial with adjunctive fenfluramine for the treatment of five different types of developmental and epileptic encephalopathies (DEEs) focused on epileptic and "non-epileptic outcomes": SYNGAP1 and STXBP1 encephalopathies, inv-dup(15) encephalopathy, multifocal or bilateral malformations of cortical development, and continuous spikes and waves during sleep. The main goal is to assess changes in seizure frequency comparing before and after treatment with fenfluramine in five specific types of developmental and epileptic encephalopathies (DEEs). Secondary objectives of this study are the analysis of changes in seizure intensity and duration, and "non-epileptic outcomes" such as variations in cognitive activity, level of alertness, impulsivity/self-control, gait stability and other alterations that might be detected during the interview and physical examination.
Registration Trial of the Intracranial Visualized Stent for the Wide-necked Intracranial Aneurysms...
Brain DiseasesAneurysm2 moreA registration trial of the Intracranial Visualized stent in the treatment of wide-necked intracranial aneurysms