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An Open-label, Phase I Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AZD5492 Following Single-ascending Dose and Step-up Dose Administration to Adult Participants With Systemic Lupus Erythematosus or Idiopathic Inflammatory Myopathies or Rheumatoid Arthritis
The purpose of this study is to measure the safety, tolerability, PK, and PD of AZD5492 administered subcutaneously in adult participants with SLE or IIM or RA…
Electrical Impedance Myography: Natural History Studies in Neuromuscular Disorders and Healthy Volunteers
Background: \- Electrical impedance myography (EIM) is a new technique being studied to see if it is helpful in evaluating muscle disorders and nerve disorder…
Individual Cerebral Hemodynamic Oxygenation Relationships (ICHOR 1)
This is an observational study in patients who require clinical anesthesia. The main purpose of this study is to understand whether there are differences in th…
Ultracompact Hand-Held Swept-Source Optical Coherence Tomography (SS-HH-OCT) as a Novel Diagnostic Modality for Early-Onset Retinal Dystrophies (EORDs)
The goal of this observational study is to utilize a novel imaging system designed for high-resolution retinal imaging of neonates, infants and children to ide…
A Phase 2 Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Narsoplimab in Pediatric Patients (28 Days to ≤ 18 Years of Age.) With High-Risk Hematopoietic Stem Cell Transplant Thrombotic Microangiopathy
The purpose of this study is to evaluate the safety and efficacy of narsoplimab in pediatric patients with thrombotic microangiopathies (TMA) following hematop…
A Pilot Study to Assess the Effects of Simeox 200 Airway Clearance Device in Bronchiectasis Patients With Chronic Mucus Hypersecretion
The purpose of this single arm post-market observational study is to evaluate the short-term effectiveness of the Simeox 200 airway clearance device in improvi…
Transformative Research In DiabEtic NephropaThy
This is a prospective, observational, cohort study of patients with a clinical diagnosis of diabetes who are undergoing clinically indicated kidney biopsy. The…
Center Without Walls for Imaging Proteinopathies With PET (CW2IP2): Phase I Pilot Study of Biodistribution, Metabolism, Excretion and Brain Uptake of 18F-JSS20-183A
The current protocol is to determine the biodistribution, metabolism, excretion and brain uptake of 18F-JSS20-183A. The goal of this radiotracer is to quantify…
Study of the Role of Genetic Modifiers in Hemoglobinopathies
This study will investigate the role of genetic modifiers in hemoglobinopathies through a large-scale, multi-ethnic genome-wide association study (GWAS).
Phase 1 Study of Ketogenic Diet for Prevention of Epileptic Spasms in Infantile Onset Genetic Epilepsies
Epileptic spasms (ES) are a predominantly infantile seizure type observed frequently in certain genetic disorders. Ketogenic diet (high ratio of fat to carbohy…
TRANSPIRE: Lung Injury in a Longitudinal Cohort of Pediatric HSCT Patients
Hematopoietic stem cell transplant (HSCT) is an effective but toxic therapy and pulmonary morbidity affects as many as 25% of children receiving transplant. Ea…
A Double-Blind, Randomized, Pilot Study to Investigate the Efficacy of a Topical Palmitated Formulation of Capsaicin (Capsadyn®) In the Treatment of Diabetic Neuropathic Foot Pain
The purpose of this research is to assess whether the use of a topical cream with an active ingredient can reduce the amount of foot pain experienced in partic…