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Genetic Regulators of Metabolism and Development in Children
This is a prospective, non-randomized, non-blinded observational study. The overarching goal is to discover new disease-associated genes in children, while est…
The Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia
Background: People s bodies need to break down food into the chemicals. These chemicals are used for energy and growth. Some people cannot process all chemica…
Cardiovascular Disease Discovery Protocol
Background: \- Researchers are interested in studying individuals who have known or suspected metabolic, inflammatory or genetic diseases that may put them at…
Study of TCR Alpha Beta T-Cell and CD19 B-Cell Depletion for Hematopoietic Cell Transplantation From Haploidentical Donors in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children
This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is s…
Effect of Endoscopic Sleeve Gastroplasty on Patients With Obesity and Concomitant Metabolic Dysfunction-Associated Steatohepatitis (MASH): A Multicenter, Open-label, Randomized Controlled Trial
Metabolic dysfunction-associated steatotic liver disease (MASLD) is the most common chronic liver disease globally. While weight loss through lifestyle modific…
Evaluation of Children With Endocrine and Metabolic-Related Conditions
Background: Endocrine glands give off hormones. Researchers want to learn more about the disorders that affect these glands in children. These disorders might…
Cardiovascular and Metabolic Sciences Biorepository
The purpose of the Cardiovascular and Metabolic Sciences Biorepository is to collect and store information and biospecimens (blood, urine, stool, and heart tis…
A Phase 1, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AMG 513 in Participants With Obesity
The primary objective of this study is to assess the safety and tolerability of AMG 513 after single and multiple doses.
Theoretically Informed Behavioral Intervention to Prevent HIV-related Comorbidities
The goal of this waitlist control clinical trial is to learn if the tailored LEARN 2 platform can prevent HIV-related comorbidities with shared risk factors in…
A Phase 1 Open-label, Multicenter, First-in-human, Ascending Dose Trial Evaluating the Safety and Tolerability of a Lipid Nanoparticle Formulation of CRISPR-Guide RNA-Cas9 Nuclease (CTX310) for In Vivo Editing of the Angiopoietin-like 3 (ANGPTL3) Gene in Participants With Refractory Dyslipidemias
This is a single-arm, open-label, multicenter, ascending dose Phase 1 trial that will enroll participants 18 to 75 years of age with dyslipidemias that are ref…
Neuroinflammation and Alzheimer's Disease Imaging Biomarkers in Midlife Obesity
High body fat at midlife, as evidenced by overweight or obese body mass index (BMI), is increasingly understood as a risk factor for Alzheimer's disease. Howev…
Exercise Effects on Adipose Tissue Structure and Function
Participants will be randomized into one of two different experimental groups: 1) Exercise group and 2) No exercise (control group). Subject participation in t…