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Maralixibat in Patients With Cystic Fibrosis and Constipation, A Within-Subjects Pilot Study
Chronic constipation is common in children with cystic fibrosis (CF), likely due to impaired chloride channel function that reduces intestinal secretions. Stan…
A Phase 1 Study of EP31670, a Dual BET and CBP/p300 Inhibitor in Patients With Targeted Advanced Solid Tumors and Hematological Malignancies
A Phase 1, first-in-human study of EP31670, a dual BET and CBP/p300 inhibitor in patients with targeted advanced solid tumors and Hematological Malignancies
CGM Dynamic Index (CDI) for Predicting Prediabetes in People With Cystic Fibrosis
The primary objective of this pilot study is to develop a CGM-based model to predict the progression from prediabetes to diabetes in individuals with cystic fi…
A Dose-Escalation Study Evaluating the Safety and Tolerability of Artesunate in Participants With Idiopathic Pulmonary Fibrosis (SAFE-IPF)
Idiopathic Pulmonary Fibrosis (IPF) is a chronic progressive fibrotic lung disease resulting in increasing shortness of breath, cough, and low oxygen levels as…
Treating Anemia in Myelofibrosis With Repurposed Drugs (Nelfinavir) That Restore Iron Delivery to the Bone Marrow
This is a phase I/II protocol investigating whether Nelfinavir can improve anemia and lower serum fibrosis biomarkers in Myelofibrosis patients.
Sinonasal Therapies and Histologic Correlations for Patients With Cystic Fibrosis in the Era of Highly Effective Modulator Therapy
The investigators are doing this study to discover if it is appropriate for people with Cystic Fibrosis (CF) on highly effective modulator therapy (HEMT) to st…
Phase I Study Accessing the Safety of Pacritinib in Combination With Talazoparib in Patients With Myeloproliferative Neoplasms Unresponsive to Frontline JAK2 (Janus Kinase 2) Inhibition
This is a prospective phase I dose-escalation study, with the primary objective to access the MTD and find the RP2D of talazoparib, given in combination with s…
Losartan for Diffuse Myocardial Fibrosis in Sickle Cell Disease: A Prospective, Phase II Study.
This study is a pilot, phase II, open-label study of the angiotensin II receptor blocker, losartan, in patients with Sickle Cell Disease (SCD) 6 years or older…
Skin-interfaced Colorimetric Bifluidic Sweat Sensor Device for the Diagnosis of Cystic Fibrosis (CF)
Cystic fibrosis (CF) is a multisystem autosomal recessive inherited disease affecting approximately 75,000 individuals in USA. The sweat chloride (Cl) test rem…
Novel Point-of-care Sweat Chloride Testing Device for Monitoring CFTR Function
This study is being done to test a device called micro Sweat Test Patch (mSTP or µSTP). The study team will compare the standard of care (SOC) method of sweat…
A Phase 2, Double-blind, Randomized Study to Compare the Effect of Curcumin Versus Placebo on Inflammatory Cytokines, Symptoms and Disease Parameters in Clonal Cytopenia of Undetermined Significance (CCUS), Low-Risk Myelodysplastic Syndrome (LR-MDS), and Myeloproliferative Neoplasms (MPNs)
This phase II trial evaluates how a curcumin supplement (C3 complex/Bioperine) changes the inflammatory response and symptomatology in patients with clonal cyt…
Clinical Correlation Evaluation of the LIVERFAStTM Test for Diagnosing Important Liver Lesions of Fibrosis and Steatosis Against MR-based Liver Assessment, Magnetic Resonance Elastography (MRE) for Liver Fibrosis and MR-based Assessment of Steatosis, in Adult US Population.
This is a retrospective cross-sectional research intended to explore the utility of LIVERFASt in the clinical pathways for the detection of liver fibrosis and …