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Phase I/II Trial of Docetaxel and SX-682 in Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma, Salivary Gland Carcinoma, and Advanced Prostate Cancer
Background: Head and neck cancers (HNCs) account for about 5% of all cancers worldwide. They grow in the mouth, throat, nasal cavity, or salivary glands. Pros…
Comparison of Continous Glucose Monitor and OGTT as a Screen for Cystic Fibrosis Related Diabetes and Impaired Glucose Tolerance
Cystic Fibrosis (CF) related diabetes (CFRD) is a unique form of diabetes mellitus, different from type 1 diabetes and type 2 diabetes. The diagnosis of CFRD i…
Natural History of Noncirrhotic Portal Hypertension
Background: \- Noncirrhotic Portal Hypertension (NCPH) is caused by liver diseases that increase pressure in the blood vessels of the liver. It seems to start…
Natural History of Treated Neurocysticercosis and Long-Term Outcomes
Neurocysticercosis is a brain disease due to the larval stage of the pork tapeworm (Taenia solium). The most common symptoms patient experience from infection …
A Phase I Randomised, Single-blind, Placebo-controlled Study to Assess the Safety, Tolerability, and Pharmacokinetics of AZD1613 Following Multiple Ascending Dose Administration in Participants With Autosomal Dominant Polycystic Kidney Disease
A study to investigate safety, tolerability, and pharmacokinetics of AZD1613 following subcutaneous or intravenous administration in participants with autosoma…
A Sequential, Phase 1a/1b, Double-blind, 2-part Study to Investigate the Safety, Tolerability, Microbiological Response, and Pharmacokinetics of Rev-56 for Inhalation Compared With Placebo in Healthy Adult Participants and Adult Non-cystic Fibrosis Bronchiectasis (NCFB) Patients With Chronic Pseudomonas Aeruginosa
This is a Phase 1a/1b study to test the safety, tolerability, microbiological response, and pharmacokinetics of inhaled Rev-56 compared to placebo in adult hea…
Bempedoic Acid Therapy for Polycystic Kidney Disease (BEAT-PKD) Randomized Clinical Trial
Autosomal dominant polycystic kidney disease (ADPKD), the most commonly inherited kidney disease, is characterized by the development of cysts in the kidney th…
A Phase 2, Multicenter, Randomized, Active-controlled Study to Assess the Safety and Efficacy of ANG003 at Two Different Dose Levels in Subjects With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
In this study, ANG003, a pancreatic enzyme replacement therapy (PERT; commonly called "enzymes"), is being investigated as a potential treatment for exocrine p…
Streamlined Treatment of Pulmonary Exacerbations in Pediatrics Randomized Controlled Trial (STOP PEDS RCT)
The STOP PEDS RCT is a multicenter, parallel, open label randomized controlled trial evaluating the long-term (one year) and short-term safety and efficacy of …
Role of Genetic Factors in the Pathogenesis of Lung Disease
This study is designed to evaluate the genetics involved in the development of lung disease by surveying genes involved in the process of breathing and examini…
The Physiological Responses and Adaptation of Brown Adipose Tissue to Chronic Treatment With Beta3-Adrenergic Receptor Agonists
Background: Brown adipose tissue (BAT) is a type of fat in the body. It may prevent weight gain, improve insulin sensitivity, and reduce fatty liver. Research…
A Phase 1, Randomized, Double-blind, Placebo-controlled, Dose Escalation Study Evaluating Safety and Pharmacokinetics of VX-581 in Healthy Subjects
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of single dose of VX-581 and multiple ascending doses of VX-581 alone a…