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Showing 1-6 of 6 trials
NCT03128996
This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.
NCT05398783
Background: Scientists have long used simple measures (such as height and weight) to estimate how much a person s body uses food (calories) as energy, as commonly called the metabolic rate. But metabolism varies among people with similar body sizes. Scientists now believe the old formulas for estimating metabolic rates may not work well for all people. Researchers want to find more accurate ways to measure a person s metabolism. Objective: This natural history study will examine the relationships between metabolism, body composition, and body surface area in a wide range of people. Eligibility: Healthy children and adults aged 2 years or older. Also, people aged 2 years or older with conditions that may alter metabolism. These may include diabetes, obesity, renal disease, or cancer. Design: Participants will spend 2 days and 1 night in the hospital. They will provide a medical history and answer questions about their activity levels, the foods they eat, and their lifestyle. They will also eat a special diet. Participants will undergo many tests: They will lie in a bed with a clear hood covering their head for 30 to 45 minutes to measure the gases in their breath. They will lie on a padded table for about 15 minutes while their body is scanned. They will stand on a platform while a 3D scanner measures their body. They will have a test to measure how fast an electric signal moves through their body. They will grip an instrument to measure the strength of their hands. They will drink salty water and provide blood and urine samples. Participants may be invited to return for these 2-day visits up to 8 times per year. Return visits must be at least 2 weeks apart.
NCT07161557
The goal of this observational study is to evaluate Metabolic Health by fit analysis between calculated Nutrilite Metabolic Health Index and the result of Metabolomics analysis from blood plasma samples in healthy adult participants aged between 18 and 60 years. The main question it aims to answer: \- Does the Nutrilite Metabolic Health Index fit well with the result of Metabolomics analysis from blood plasma samples? 200 eligible participants will be enrolled in a single center, one site visit will be made to finish a health questionnaire, a lifestyle questionnaire, a routine physical checkup. Blood samples will be collected for routine blood test (blood sugar, total triglycerides, total cholesterol, low-density lipoprotein cholesterol, high-density lipoprotein cholesterol, uric acid), and for Metabolomics testing. Researchers will calculate the Nutrilite Metabolic Health Index based on participant data, and do the fit analysis against the result from Metabolomics analysis.
NCT03406962
This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
NCT02354443
The purpose of this study is to describe the safety profile of ProHema-CB as part of a single cord blood unit transplant after a myeloablative conditioning regimen in pediatric patients with inherited metabolic disorders. The safety profile will primarily be assessed by neutrophil engraftment.
NCT02107872
This is a phase 1, randomized, double-blind, placebo-controlled, multiple ascending dose study to assess the safety, tolerability, pharmacokinetic (PK), immunogenicity, and pharmacodynamic (PD) effects of REGN1500 in patients with a metabolic disorder.