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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 Single Ascending Dose, Phase 1/2 Trial to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of SP-101 Via Nebulizer for the Treatment of Cystic Fibrosis (CF)
This is a Phase 1/2 multicenter, open-label, single dose trial of SP-101 investigational gene therapy in adults with CF who are ineligible for or intolerant to…
A Prospective, Multicenter, Randomized, Pivotal Study of the May Health System in Transvaginal Ablation of Ovarian Tissue Under Ultrasound Guidance in Women With Infertility Due to Polycystic Ovary Syndrome
A Prospective, Multicenter, Randomized, Pivotal Study of the May Health System in Transvaginal Ablation of Ovarian Tissue under Ultrasound Guidance in Women wi…
Phase II Trial of Enfortumab Vedotin in Recurrent and/or Metastatic Adenoid Cystic Carcinoma
The purpose of this study is to find out whether enfortumab vedotin is an effective and safe treatment for people with adenoid cystic carcinoma (ACC).
International Pleuropulmonary Blastoma/DICER1 Registry (for PPB, DICER1 and Associated Conditions)
Pleuropulmonary blastoma (PPB) is a rare malignant neoplasm of the lung presenting in early childhood. Type I PPB is a purely cystic lesion, Type II is a parti…
Pharmacokinetics of CFTR Modulators in Pregnant Individuals and in Postpartum Breastfeeding Mothers
This study aims to evaluate the pharmacokinetic changes during pregnancy, postpartum, and in breast milk in cystic fibrosis patients receiving a cystic fibrosi…
Advancing ADPKD Treatment With GLP-1RA: A Study of Glucagon-Like Peptide-1 Receptor Agonists' Efficacy, Safety, and Mechanism
The proposed clinical trial aims to assess if a year of treatment with a glucagon-like peptide 1 receptor agonist, a medication approved for weight management …
Imaging Assessments of ARPKD Kidney Disease Progression
Autosomal Recessive Polycystic Kidney Disease (ARPKD) is a potentially lethal genetic disorder for which there are currently no disease specific treatments. Cl…
A Phase 1/2, Multicenter, Open-label Study of REM-422, a MYB mRNA Degrader, in Patients With Recurrent, Metastatic, or Unresectable Adenoid Cystic Carcinoma
The goal of this study is to determine the safety and antitumor effects of REM-422, a MYB mRNA degrader, in people with advanced Adenoid Cystic Carcinoma (ACC)
Pharmacokinetic and Pharmacodynamic Effects of Dorzagliatin in Pancreatic Insufficient-Cystic Fibrosis: A Randomized Double-blind, Cross-over Trial
This study is designed to determine the pharmacokinetic and pharmacodynamic response of dorzagliatin 50 mg twice daily following 7-day administration in indivi…
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…
Skeletal Health and Bone Marrow Composition in Adolescents With Cystic Fibrosis
The investigators will be evaluating bone marrow composition via magnetic resonance imaging in adolescents diagnosed with cystic fibrosis (CF) compared to heal…