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A First-In-Human Clinical Trial of Lentiviral-mediated CSF2RA Gene Transfer/Pulmonary Macrophage Transplantation Therapy of Hereditary Pulmonary Alveolar Proteinosis
The major goal of this study is to evaluate a new type of cell transplantation therapy for individuals with hereditary PAP, study a new treatment that may be u…
Slow-SPEED: Slowing Parkinson's Early Through Exercise Dosage
The goal of this clinical trial is two-fold. First to investigate the feasibility of whether a remotely administered smartphone app can increase the volume and…
The Associations Between EIT and Clinical Measures During Standard of Care Procedures in Patients With Respiratory Disease: A Preliminary Study
The purpose of this study is to evaluate the Genesis Electrical Impedance Tomography (EIT) imaging system for use in pediatric respiratory disease populations …
A Phase 1-2, Double-Blind, Sham-Controlled Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Patients With MECP2 Duplication Syndrome
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
A National Registry For Pulmonary Alveolar Proteinosis
The major goal of Part A of this study is to establish a National PAP Registry to help make reliable new research tests available to doctors to improve the dia…
An Evaluation of the Clinical Usability of the SCD411 Prefilled Syringe as a Proposed Biosimilar to US-licensed Eylea®
This is a single-arm, multicenter study to evaluate clinical usability of a pre-filled syringe containing SCD411 in adult patients with neovascular Wet Age-Rel…
Standing Like a Tree: Effects and Mechanisms of Daoist Zhanzhuang on Human Flourishing
This project investigates the impact of Daoist Zhanzhuang (sometimes spelled as Chan Chuang) on human flourishing, and explores the physiological, psychologica…
Pharmacogenetic Determinants Of Treatment Response In Children
To investigate whether genetic polymorphisms in genes encoding proteins involved in the metabolism or effects of drugs or environmental agents influence the di…
Expanding Veteran Sponsorship Initiative+ to Support High-Risk Transitioning Servicemembers and Veterans: A Precision Medicine Approach (PEC 24-113)
Background Young Veterans in the U.S. face a heightened risk of suicide, particularly during the first year after leaving military service-a period often refer…
A Phase I/II, Dose-escalation and Dose-optimization Study to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of MT-4561 in Patients With Various Advanced Solid Tumors and to Evaluate Effect of MT-4561 on Pharmacokinetics of Oral Midazolam
This is a First In Human (FIH), multicenter, open-label, Phase I/II study to evaluate safety, tolerability, Pharmacokinetics (PK), pharmacodynamics, and effica…
Evaluation of Gut-Related Well-Being and Mood Effects of a Daily Nutritional Food Ingredient From the Co-Fermentation of Fungal Mycelium (Pleurotus Pulmonarius) and Microalgae (Chlorella Vulgaris) in Healthy Adults Experiencing Minor Gastrointestinal Discomfort: A 30-Day, Two-Arm, Open-Label Study
The purpose of the study is to evaluate the gut-related well-being and mood effects of a food ingredient obtained from the co-fermentation of oyster mushroom m…
A Two-part Multicenter Study: a Randomized, Double-blind, Placebo-controlled Dose-escalation Safety Phase (Part 1) Followed by Double-blind, Placebo-controlled, Adaptive Phase (Part 2) Study to Evaluate the Safety and Efficacy of AB-1003 in Adult Subjects With LGMD2I/R9 Mutations in the Gene Encoding Fukutin Related Protein (FKRP)
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular …