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A Phase 2, Randomized, Open-label, Controlled Study to Evaluate the Efficacy and Safety of Rapcabtagene Autoleucel Versus Comparator in Participants With Severe Refractory Idiopathic Inflammatory Myopathies (IIM)
A Phase 2, randomized, open-label, controlled study to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with sever…
A Phase Ic, Open-label, Multicenter Study to Evaluate the Safety, Tolerability, and Activity of Inhaled GDC-6988 in Patients With Muco-obstructive Disease
This study evaluates the safety, tolerability, and activity of inhaled GDC-6988 in participants with muco-obstructive disease.
The Vision Study: Cerebral/ Cortical Visual Impairment (CVI)
Cerebral/Cortical Visual Impairment (CVI) is the leading cause of childhood visual impairment in the United States and other industrialized countries. CVI is a…
Bronchiectasis and NTM Research Registry
The Bronchiectasis Research Registry is a consolidated database of non-cystic fibrosis (non-CF) Bronchiectasis and Nontuberculosis Mycobacteria (NTM) patients …
A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime
The hypothesis for this study is that a preparative regimen that maximizes host immunosuppression without myeloablation will be well tolerated and sufficient f…
Immunoinflammatory and Metabolic Responses in Post Cardiac Arrest Syndrome (PCAS)
This is a prospective, observational study to investigate molecular mechanisms mediating the systemic inflammatory process, and changes to metabolism, and thei…
Pediatric Cardiomyopathy Mutation Analysis
The goal of this protocol is to obtain information from individuals with cardiomyopathy and from their families in order to elucidate the molecular genetics of…
Home-based, Digitally Delivered Breathing Training in People With Non-cystic Fibrosis Bronchiectasis: A Randomized Control Trial
The purpose of this research is to investigate whether a home-based and health coach supported specific breathing and respiratory muscle training program impro…
Application of Lung Ultrasound Artefact Analysis for Exclusion of Acute Rejection in Asymptomatic Lung Transplant Recipients
This study is being done to study a safe and non-invasive way to diagnose lung rejection and infection.
Oscillating Lung Expansion (OLE) Therapy in the Bronchiectasis Patients (BE) Home Care Study Protocol
The study will examine how the performance of Oscillating Lung Expansion (OLE) therapy affects the respiratory health of patients with Bronchiectasis (BE). Th…
Center Without Walls for Imaging Proteinopathies With PET (CW2IP2): Phase I Pilot Study of Biodistribution, Metabolism, Excretion and Brain Uptake of 11C-M503
The current protocol is to determine the biodistribution, metabolism, excretion and brain uptake of 11C-M503. The goal of this radiotracer is to quantify alpha…
The Validation of Minimally Invasive Oxygen Nanosensor Technology to Quantify Mitochondrial Function in Human Muscle
Past mitochondrial disease treatment studies have been unsuccessful in determining treatment efficacy, and a major factor has been the lack of validated biomar…