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: 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 double-blind, randomized phase 3 multicenter placebo-controlled study in at least 16 evaluable male participants diagnosed with MCT8 deficiency. Male participants, from 4 years of age (at randomization) and having demonstrated stable maintenance treatment with tiratricol, will be randomized to receive placebo or tiratricol for 30 days or until reaching rescue criterion (serum total triio...

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: Glutamine has the potentials of immunomodulation and adjustment of protein metabolism. The primary objective of this study is to evaluate the efficacy of glutamine on sarcopenia in gastric adenocarcinoma patients undergoing gastrectomy. The secondary endpoints, including the physical activity, weight loss, and nutritional profiles, will be evaluated among these patients.

Summary: The primary objective of this study is to improve the 2-year progression-free survival in children with high-risk solid tumors who are administered a maintenance regimen with continuous sirolimus administered on a backbone of metronomic chemotherapy following the completion of standard therapy, as compared to high-risk solid tumor patients treated with observation alone following completion of sta...

Summary: Obese patients often have fat accumulation in the head and neck, increased soft tissue in the oropharynx, decreased lung compliance, decreased lung volume and residual volume, and some obese patients also suffer from obstructive sleep apnea. Therefore, obese patients may experience hypoxemia during sedative gastroscopy. High-flow nasal cannula oxygen therapy (HFNC) can provide patients with high-f...

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

Summary: In Amyotrophic Lateral Sclerosis, dysphagia has a high incidence. With deterioration of swallowing function, percutaneous endoscopic gastrostomy (PEG) tube is recommended to ensure sufficient and safe oral intake. Dysphagia and PEG placement alter quality of life (QoL). However, QoL and attitudes toward PEG remain largely unexplored. The purpose of this study is to monitor the swallowing function ...

Summary: The goal of this study is to demonstrate safety, feasibility, and efficacy of a novel pharyngo-esophageal stimulation technique in restoring aerodigestive and swallowing functions in select infants at-risk for chronic gavage tube feeding or gastrostomy. The main aims are: * To provide consistent activation of deglutition (the process of swallowing), swallowing-airway interactions, and peristalsis ...

Summary: The goal of this clinical trial is to investigate feasibility and safety of an oral therapy with zinc in patients affected by Guanine nucleotide-binding protein G(o) subunit alpha (GNAO1) associated disorders. The main questions it aims to answer are: * Is a daily oral therapy with zinc in GNAO1 associated disorders a safe and feasible therapy? * Are there potential changes in general motor skills...

Summary: The purpose of this study is to determine if neoadjuvant therapy to increases resection rate for pancreatic adenocarcinoma.