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A Phase 2a, Open-label, Single-arm Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VX-407 in Subjects With Autosomal Dominant Polycystic Kidney Disease Who Have a Subset of PKD1 Gene Variants
The purpose of the study is to evaluate the effect of VX-407 on height-adjusted total kidney volume (htTKV), safety, tolerability, and pharmacokinetics (PK) 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…
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 …
DICER1-Related Pleuropulmonary Blastoma Cancer Predisposition Syndrome: A Natural History Study
Background: \- Pleuropulmonary blastoma (PPB) is a rare fast-growing lung tumor that is associated with other, rare tumor types. Most cases of PPB appear in c…
A Double-blind, Phase II Randomized Study of Brain-directed Stereotactic Radiation With or Without AGuIX Gadolinium-based Nanoparticles in the Management of Brain Metastases at Higher Risk of Local Recurrence With Radiation Alone
The purpose of this study is to determine whether AGuIX (Activation and Guidance of Irradiation by X-ray) gadolinium-based nanoparticles make radiation work mo…
Core A: The Hepato/Renal Fibrocystic Diseases Translational Resource (ARPKD Database Study)
Hepato-renal fibrocystic diseases (HRFD) is a term developed that encompasses rare diseases such as Autosomal Recessive Polycystic Kidney Disease (ARPKD), and …
A Randomized, Double-blinded, Placebo-controlled, Parallel Group, Phase 2a Study to Assess the Activity, Safety, and Tolerability of SAR445399 in Adult Participants With Non-Cystic Fibrosis Bronchiectasis (NCFB)
This is a randomized, double-blind, placebo-controlled study to measure the reduction in mucus plug score at 24 weeks of treatment with SAR445399 compared with…
Feasibility and Efficacy of a Well-Formulated Ketogenic Diet in Delaying Progression of Polycystic Kidney Disease in Patients at Risk for Rapid Progression
This is a prospective study to determine ketogenic diet effect on htTKV, GFR, microalbuminuria. This is a single-center study of 20 patients with ADPKD and dee…
A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of ABBV-CLS-628 in Adult Subjects With Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common genetic cause of kidney disease that causes fluid-filled cysts to develop in the kidney…
Resistant Starch Usage in Polycystic Ovary Syndrome: Impact on Cardiometabolic Dysfunction and the Gut Microbiome (CORS-PCOS)
This study will enroll women with PCOS to study the effects of first line therapy, oral contraceptive pills, and then either 12 weeks of resistant starch or 12…
Effect of GLP-1 Agonist Therapy on Insulin Secretion in Adults With Pancreatic Insufficient Cystic Fibrosis and Abnormal Glucose Tolerance: a Randomized, Open-label, Cross-over Trial
Diabetes is a major co-morbidity in pancreatic insufficient cystic fibrosis (PI-CF) and associated with worse outcomes. While reduced β-cell mass contributes t…
Outcomes Related to Body Composition in Teens and Adults With Cystic Fibrosis (ORBIT-CF)
Nutrition and body composition, the amount of muscle and fat in the body, has a role in overall health. This study wants to learn more about how nutrition and …