Nashville, TN Paid Clinical Trials

This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001). Read Less

This phase II trial tests how well canakinumab works to prevent progression to cancer in patients with clonal cytopenias of unknown significance (CCUS). CCUS is a blood condition defined by a decrease in blood cells. Blood cells are composed of either red blood cells, white blood cells, or platelets. In patients with CCUS, blood counts have been low for a long period of time. Patients with CCUS also have a mutation in one of the genes that are responsible for helping blood cells develop. The combination of genetic mutations and low blood cell counts puts patients with CCUS at a higher risk to develop blood cancers in the future. This transformation from low blood cell counts to cancer may be caused by inflammation in the body. Canakinumab is a monoclonal antibody that may block inflammation in the body by targeting a specific antibody called the anti-human interleukin-1beta (IL-1beta). Read Less

This study is focused on males who have Hemophilia B and who need regular preventive treatment with factor IX protein (FIX) replacement therapy to prevent and also to control their bleeding events. The aim of the study is to gather at least 6 months of information on bleeding events for each individual participant while they continue to use their usual FIX replacement therapy. There is no experimental treatment being tested in this study. The study is informational, and part of a larger program to understand and treat Hemophilia B with a potential experimental new therapy in the future. There is no obligation to agree to taking part in this future study. The study is looking to answer several other research questions to help understand each participant's individual disease characteristics, including: * How often to use FIX replacement therapy, both on a regular basis (prophylaxis) and as needed to treat bleeding events * Measurement of FIX activity (factor IX is a clotting factor) by different laboratories using different types of tests in Hemophilia B participants * Possible complications from the FIX replacement therapy the patient receives (usual standard of care will continue to be used) * How quality of life is affected by Hemophilia B * How joint health is affected by Hemophilia B * How often the participant visits the emergency room, urgent care center, physician's office, hospital, or has a telemedicine visit as a result of bleeding events * Whether the body makes antibodies (a protein produced by the body's immune system) against the FIX replacement therapy you receive, which could make the drug less effective or could lead to side effects Read Less

The aim of this study is to identify the optimal dose for Vosilasarm (EP0062) as monotherapy and in combination with standard-of-care therapies to assess its Safety, Tolerability, Pharmacokinetics, and Efficacy in Patients with Relapsed Locally Advanced or Metastatic AR+/HER-2-/ER+ Breast Cancer Read Less

The goal of this randomized clinical trial is to study the best treatment for open lower leg fractures to prevent infection. The main questions it aims to answer is if treating tibia fracture patients with a calcium sulfate antibiotic depot is better at preventing infection that the standard of care. Read Less

The goal of this interventional clinical trial is to learn about TNG260, a CoREST inhibitor, in combination with pembrolizumab in patients with advanced solid tumors with a known STK11 mutation. The main question\[s\] it aims to answer are: * the recommended dose for Phase 2 * to evaluate the safety and tolerability of the combination therapy * to determine the pharmacokinetics of TNG260 * to evaluate the initial antineoplastic activity Participants will receive study treatment until they experience an undesirable side effect, their disease progresses or until they withdraw consent. Read Less

This is a randomized placebo-controlled trial of semaglutide, an FDA-approved therapy for the treatment of type 2 diabetes mellitus and obesity, in adults with symptomatic asthma despite the use of inhaled steroids and with excess body weight. This study will test the central hypothesis that semaglutide will improve asthma control and reduce airway inflammation due to direct effects on the respiratory tract in adult asthma associated with obesity. Read Less

This randomized controlled trial will evaluate the effect of intravitreal faricimab or fluocinolone acetonide (FAc) intravitreal implant compared with observation on long-term visual acuity following treatment of choroidal melanoma with iodine-125 plaque brachytherapy. Read Less

This study will be a prospective randomized control trial to evaluate the effects of subcutaneously administered TXA among patients undergoing nasal Mohs reconstruction with local flaps at VUMC. Read Less

This is a phase IV post-marketing study for MagnetOs Easypack Putty. MagnetOs Easypack Putty is a synthetic bone graft extender product that is routinely used by surgeons as a treatment for patients with degenerative disc disease or spinal trauma undergoing spinal fusion surgery. In this study, MagnetOs Easypack Putty will be used according to the latest U.S. Instructions For Use, standalone in the posterolateral spine. Read Less

This Phase 1/2, open-label, multicenter study is conducted in patients with previously treated selected solid tumors, including non-small cell lung cancer (NSCLC), small cell lung cancer (SCLC), high-grade neuroendocrine cancer of any primary site, diffuse large B-cell lymphoma (DLBCL), and tumors with L-MYC or N-MYC amplification. Patients receive escalating doses of a GSPT1 molecular glue degrader MRT-2359 to determine safety, tolerability, maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D) of MRT-2359. Once the MTD and/or RP2D is identified, additional patients enroll to Phase 2 study, which includes molecular biomarkers stratification or selection, namely expression or amplification of L-MYC and N-MYC genes, hormone receptor positive (HR)-positive, human epidermal growth factor 2 (HER2)-negative breast cancer and prostate cancer. Read Less

Pulmonary arterial hypertension (PAH) is a severe disease with a delayed diagnosis and markedly elevated mortality. High-risk populations, such as those with known genetic defects, provide a unique opportunity to determine the features of susceptibility and resilience to PAH. This proposal will fundamentally overturn the prevailing understanding of PAH by creating molecularly-driven signatures of susceptibility and resilience, provide novel insight into disease severity, and potentially identify new therapeutic targets. Funding Source - FDA OOPD Read Less