Summary: This (DEEp OCEAN Study) is a double-blind, randomized, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of LP352 in the treatment of seizures in children and adults with DEE. The study consists of 3 main phases: Screening, Titration period, Maintenance period, followed by a Taper period and Follow-Up. The total duration of the study will be approximately ...

Summary: This is a research study to find out if adding a new study drug called revumenib to commonly used chemotherapy drugs is safe and if they have beneficial effects in treating patients with acute myeloid leukemia (AML) or acute leukemia of ambiguous lineage (ALAL) that did not go into remission after treatment (refractory) or has come back after treatment (relapsed), and to determine the total dose o...

Summary: The long-term goal of this project is to develop a therapy to assist pre-term and term infants with brain injury overcome difficulties in learning to feed so that infants may be discharged home with their families and avoid the burdens of of a gastrostomy tube (G-tube) or prolonged home nasogastric feeding. Few other therapies exist for infants who are not making progress with feeding volumes at t...

Summary: This (DEEp SEA Study) is a double-blind, randomized, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of LP352 in the treatment of seizures in children and adults with DS. The study consists of 3 main phases: Screening, Titration period, and Maintenance period, followed by a Taper period and Follow-Up. Participants will be randomized to LP352 or placebo. ...

Summary: Researchers are looking for a way to prevent cytomegalovirus (CMV) in children and adolescents who receive a kidney transplant (KT) and weigh less than 40 kilograms (88.2 pounds). The goals of the study are to: * Learn what happens to letermovir in the body over time * Learn about the safety of letermovir and if participants tolerate it

Summary: To compare the symptoms of patients who have a MIPG to the symptoms of patients who have a MITG.

Summary: A 12 months mono-center, descriptive study, to evaluate pain and predictive factors Following placement of percutaneous Radiologic gastrostomy (PRG). The primary objective is to evaluate the abdominal pain associated with percutaneous radiologic gastrostomy (PRG) placement.

Summary: This randomized controlled trial will evaluate the efficacy of ultrasound-guided external oblique intercostal fascial plane (EOI) block in reducing intraoperative and postoperative pain in pediatric patients undergoing laparoscopic gastrostomy under general anesthesia. Forty patients aged 3-18 years will be randomly allocated to receive either bilateral EOI block with 0.25% ropivacaine or sham blo...

Summary: This is a single-center prospective study evaluating the efficacy of a prototype gastrostomy anchor dressing for use with button gastrostomy tubes. The anchor dressing has been previously evaluated for usability by parents, patients, and nurses. This study compares several outcomes related to complications from gastrostomy tube placement using a prospective cohort of patients and a retrospective a...

Summary: Gastroscopy is a commonly used, direct, and reliable method for screening and diagnosing digestive tract diseases. However, as an invasive examination, it can cause adverse reactions such as pain, nausea, vomiting, and choking cough in patients. Compared with ordinary gastroscopy, painless gastroscopy offers higher comfort and satisfaction for patients and greater convenience for endoscopists duri...

Summary: The study was planned to evaluate the effects of discharge training on PEG use given to palliative care patients' relatives on the caregiver burden and quality of life of the relatives. The main questions that the study aims to answer are as follows: * How does PEG training given before discharge affect the knowledge level of caregivers on PEG use? * How does PEG training given before discharge af...

Summary: This is an open-label, multi-center, Phase 1/2 study of the brain-penetrant MEK inhibitor, mirdametinib (PD-0325901), in patients with pediatric low-grade glioma (pLGG).