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Phase 1b/2a Trial of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) From an HLA-partially Matched Related or Unrelated Donor After TCRab+ T-cell/CD19+ B-cell Depletion for Patients With Monogenic and/or Early-onset (EO), Medically Refractory (MR) Crohn Disease (CD)
This research study is investigating whether alpha beta T-cell depleted hematopoietic stem cell transplant (HSCT) can be an immune system replacement for Crohn…
Healthcare Organizational Structural Conditions and the Health of People Recently Released From Prison
People released from prison experience two times higher cardiovascular (CV) mortality than the general population, have a high prevalence of poor CV health and…
A Phase 3, Randomized, Open-Label, Multicenter Study to Evaluate the Safety (Compared to Iron Sucrose), Efficacy and Pharmacokinetics of Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects With Chronic Kidney Disease (CKD)
Primary Objectives: To evaluate the safety (compared to iron sucrose) and efficacy of ferumoxytol in pediatric CKD subjects with iron deficiency anemia (IDA) …
BOOST-PD - Better On-time Observations of Motor Fluctuations Using Wearable Sensor Technology: A Naturalistic Study on IPX-203 for Parkinson's Disease
The purpose of this study is to evaluate the effect of IPX203 (Crexont®) - the newest extended-release levodopa formulation - on the duration and quality of go…
Genetic, Metabolic, and Growth Factor Repository for Cerebrovascular Disorders
This study encompasses the maintenance and utilization of a repository of samples from patients with cerebrovascular disease and stroke. The types of tissue ta…
Quantitative Pulmonary Imaging Registry & Biorepository
The goal of this project is to establish a registry and biorepository of images and biological samples from subjects undergoing novel pulmonary imaging methods…
A Phase II Placebo Controlled Study of the Effects of Anakinra (Kineret) in Subjects With Corticosteroid-Resistant/Intolerant Meniere's Disease (CR-MD) and Corticosteroid-Resistant/Intolerant Autoimmune Inner Ear Disease Disease (CR-AIED)
A Phase II randomized, placebo controlled study design of anakinra (Kineret) in corticosteroid-resistant or intolerant Meniere's disease (CR-MD)and corticoster…
Coronary Microvascular Disease and Endothelial Function in Women and Men
Women suffer disproportionately than men from Cardiac Syndrome X ( chest pain in the absence of flow limiting coronary artery stenosis). Coronary microvascular…
The Influence of a Fasting Mimicking Diet on Ulcerative Colitis
The purpose of this study is to see how a diet that mimics fasting effects inflammation in patients with mild to moderate Ulcerative Colitis (UC). The diet may…
Moderately Carbohydrate-restricted Diet as Treatment Targeting Improvement in Hepatic Lipid and Insulin Sensitivity in Adolescents With NAFLD
This will be a 6-month randomized clinical trial with two arms: moderately carbohydrate-restricted diet and a fat-restricted control diet. This 6-month study w…
An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 2 to < 12 Years) With Fabry Disease and Amenable GLA Variants
An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to \< 12…
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 …