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A Phase 2, Open-label, Fixed-dose Study to Assess the Efficacy of Sodium Phenylbutyrate (ACER-001) in Treating Pediatric and Adult Patients With Medium Chain Acyl-CoA Dehydrogenase (MCAD) Deficiency Resulting From the Prevalent ACADM c.985 A>G (K304E) Mutation
This is a medical research study to test a medication in patients 4 years of age and older with a disease called medium-chain acyl-CoA dehydrogenase deficiency…
A Phase 2a Trial of Immune Modulation in Combination With Ultrasound-mediated Blood Brain Barrier Opening in Patients With Newly Diagnosed Glioblastoma
Brain tumor treatment is hampered by the blood-brain barrier (BBB). This barrier prevents drugs carried in the bloodstream from getting into the brain. If the …
Interleukin-4Ralpha Blockade by Dupilumab Decreases Staphylococcus Aureus Colonization and Increases Microbial Diversity in Chronic Rhinosinusitis With Nasal Polyposis (CRSwNP)
Hypothesis: The investigators hypothesize that in patients with CRSwNP who demonstrate sinus colonization with staphylococcus aureus, the administration of dup…
A Phase II, Open-Label, Multicenter Study With a Safety Run-In to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of MT027 Administrated Intracerebroventricularly in Patients With Recurrent or Progressive Glioblastoma (WHO Grade 4)
This is a Phase II trial to evaluate the safety, tolerability, efficacy, and PK/ pharmacodynamic profiles of MT027 injected via ICV in participants with recurr…
Efficacy and Safety of Cryopreserved Autologous Mobilized Peripheral Blood CD34+ HSPCs Transduced Ex Vivo With the EFS-ADA Lentiviral Vector in Patients With Severe Combined Immune Deficiency Due To Adenosine Deaminase Deficiency
The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral bl…
Clinical Correlation Evaluation of the LIVERFAStTM Test for Diagnosing Important Liver Lesions of Fibrosis and Steatosis Against MR-based Liver Assessment, Magnetic Resonance Elastography (MRE) for Liver Fibrosis and MR-based Assessment of Steatosis, in Adult US Population.
This is a retrospective cross-sectional research intended to explore the utility of LIVERFASt in the clinical pathways for the detection of liver fibrosis and …
A Double-Blind, Active-Controlled, Multiple-Ascending Dose, Phase 1b/2a Study of Aerosolized RSP-1502 Delivered Via the PARI LC Plus® Nebulizer in Subjects With Cystic Fibrosis and Chronic Pseudomonas Aeruginosa Lung Infection
A double-blind, active-controlled, multiple-ascending dose, safety study of aerosolized RSP-1502 in subjects with cystic fibrosis Pseudomonas aeruginosa lung i…
A Phase 1, Open-Label Study Evaluating the Effect of Miricorilant on Hepatic Lipids in Patients With Presumed Metabolic Dysfunction-Associated Steatohepatitis (MASH)
A Phase 1, Open-Label Study Evaluating the Effect of Miricorilant on Hepatic Lipids in Patients with Presumed Metabolic Dysfunction-Associated Steatohepatitis …
Philips FAST picoSAT Convenience Sampling for Clinical Performance in Neonate, Infant, and Pediatric Patients
The main goal of this study is to look at the performance of the neonatal, infant, and pediatric Philips SpO2 sensors with the Philips FAST Pulse Oximetry tech…
An Open Label, Exploratory, Proof-of Concept Study of Triheptanoin as Treatment for Patients With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency
This is a medical research study to test a medication in patients with a disease called Pyruvate Dehydrogenase Complex (PDC) Deficiency. The medication is trih…
A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Long-Term Clinical Benefit of Govorestat in Patients With Charcot-Marie-Tooth-Sorbitol Dehydrogenase Deficiency (CMT-SORD)
This study is designed to assess the long-term efficacy and clinical benefit of AT-007 in patients with CMT-SORD. This randomized, double-blind study will asse…
Cartilage Remodeling Using Low-Temperature Radiofrequency for Facial Reconstruction
The goal of this study is to evaluate the efficacy of using the Vivaer low-temperature radio-frequency (LTRF) device to reshape ex-vivo human nasal or auricula…