This study is for subjects that have been diagnosed with AML (acute myeloid leukemia) and MDS (myelodysplastic syndrome). This study is testing an "investigational" (not yet FDA approved) study drug called Sea-CD70. This study will find out if the drug is safe and tolerable, and find out the maximum tolerated dose (highest dose of a drug or treatment that does not cause unacceptable side effects) or recommended dose for the next phase of clinical study. The subject will be given the study drug in 28 day cycles. At any given visit, the subject may undergo procedures, such as a physical exam, blood samples, ECG (electrocardiogram), or a bone marrow exam. The subject may remain in the study for up to approximately 3 years.
This study is for patients who have been diagnosed with relapsed (the disease has gotten worse after a period of improvement) or refractory (the disease does not respond to treatment) Epstein-Barr Virus-Positive (EBV+) lymphomas.
The investigational drug in this study is Nanatinostat. Investigational means it is not approved by the United States' Food and Drug Administration (US FDA). Nanatinostat is a tablet that will be taken by mouth once a day.
The purpose of this research study is to determine the effectiveness and safety of the combination therapy, nanatinostat and valganciclovir, on treating lymphoma cancers. Valganciclovir is an approved anti-viral drug (a drug that fights against virus in your body), which has been used in the prevention and treatment of infection caused by a virus called cytomegalovirus (CMV), which is similar to Epstein-Barr virus (EBV). The safety and effectiveness of nanatinostat on its own will also be reviewed in a small subset of patients.
Participants can expect to receive treatment until disease progression, unacceptable toxicity/ side effects, or participants withdraw. Participants can expect to be in follow up for up to 5 years.
This study is for participants that have been diagnosed with Medulloblastoma. The purpose of this study is to test the effectiveness of using an investigational agent called DFMO for Medulloblastoma. An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA). This study will look at the ability of this study drug to either keep participants tumor in remission or if they have active tumor, for their tumor to respond to the treatment and will also look at the safety and tolerability of DFMO. After this first day participants will be seen in clinic once every 30 days for the first 6 months of the study, after that they will be seen once every 90 days for the remainder of the study. These visits will last about 2 hours.
This study is for participants with tumors from pediatric cancers and genomic/molecular testing was done as part of standard of care treatment. This is an observational study; therefore, only information about the disease and medical treatment will be collected and participants will not receive any treatments or additional medications. The sponsor, Beat Childhood Cancer, will collect and store personal health information and molecular/genomic test results, tissue samples, and bodily fluids (examples: additional tube(s) of blood, urine, bone marrow or cerebral spinal fluid) that are left over after testing or treatment is completed in a data registry and a specimen bank, and make these available for future research. Database personnel will continue to collect and store participant information from future visits, as long as they do not withdraw from participation in this study.
The purpose of this study is to test whether a drug called PRA023/MK7240 (the study drug) is a good treatment for patients with Systemic Sclerosis associated with Interstitial Lung Disease (SSc-ILD). The study drug PRA023/7240 is an investigational drug that is given by infusion every 4 weeks. An investigational drug is not approved by The US Food and Drug Administration. It can only be used in a research study like this one. In this study, PRA023/MK7240 will be compared with a placebo (dummy drug). The placebo will be a saline solution that does not have any study drug in it. The comparison with the placebo helps to determine whether the effects seen in your body is because of the PRA023/MK7240 or not. This is a randomized study meaning that you will be assigned by chance (like flipping a coin) to receive either the study drug or placebo. This will be done with the help of a computer-based program and you will have 50% chance of receiving either the study drug or placebo. The study is double-blinded study and 50 weeks long, meaning you and your study doctor will not know what you are receiving, the study drug or placebo.
The study is sponsored by Prometheus Biosciences, Inc., a subsidiary of Merck & Co., Inc. The study is being done at approximately 25 sites across the United States. The main portion of the study will require 15 visits to the MUSC main campus and will have the following procedures completed over the course of your participation: blood draw, physician-led assessments of your disease (for example physical exam and skin thickness testing), tests to assess your pulmonary function and health (Pulmonary Function Test (PFT) and High Resolution Computed Tomography (HRCT)), electrocardiogram, as well as asked to complete surveys. If you complete the initial blinded treatment period of 50 weeks, the study doctor will discuss whether you are eligible to enter the open label period of the study, meaning no placebo. If you are eligible and agree, you will receive 500 mg of study drug once every 4 weeks for an additional 52 weeks. Compensation is available for participation.
The purpose of this research is to find out if a drug called ganciclovir can prevent cytomegalovirus (CMV) reactivation, in which the virus wakes up from an inactive state, in patients with respiratory failure associated with severe sepsis. In order for you to take part in this study, we verified that you also have previously had an infection with a virus called Cytomegalovirus (CMV). Ganciclovir is an FDA approved drug that has been widely used for the prevention and treatment of CMV in patients with weakened immune systems. A total of 482 patients will be enrolled in this study at sites throughout the US.
This study is for subjects that have been newly diagnosed with diffuse large B cell lymphoma (DLBCL). This study is testing an "investigational" (not yet approved by the Food and Drug Administration (FDA)) combination of the drug zanubrutinib (BRUKINSAâ„¢) and a standard chemotherapy treatment called R-CHOP. This study will test the how well the drug combination works and how safe it is. The subject will be given the study drug combination in 21 day cycles. At any given visit, the subject may undergo procedures, such as a physical exam, blood samples, bone marrow biopsies, and imaging scans. The subject may remain in the study for up to approximately 2 years.
This randomized, double-blind, placebo-controlled Phase 2/3 adaptive study involves an initial investigational blood test to determine if you have a specific variation related to kidney disease. The investigational blood test is to see if you have changes in your DNA of a gene called APOL1. People who have this gene variation may be at risk of losing their kidney function faster than others. If you have the variants (changes in DNA) you may be eligible to continue participation in the study. If you do not have the variants, you will not be eligible, and the study doctor will discuss your other options with you. If you decide to participate, there will be no cost to you and you will be compensated. This study will start by comparing two doses of VX-147 against placebo in subjects with APOL1-mediated kidney disease for 12 weeks. Subjects in Phase 2 will continue to Phase 3 once a dose for Phase 3 is selected. Then the Phase 3 dose of VX-147 will be evaluated for safety and effectiveness. If you meet the requirements and choose to take part in the study, you will be randomly assigned to a treatment group. You will not know which study treatment group you are assigned to and it is possible that you will receive placebo instead of VX-147. The study includes a screening, treatment, and follow-up period. The study will end after the last patient enrolled has completed 2 years in the study. This means some patients enrolling earlier could be in the study for up to 4 years.
The purpose of this research is to compare the risks and benefits of two different procedures used to help patients with mitral valve regurgitation (also known as MR). MR is a condition where the valve does not close fully when it is supposed to, and some blood can then leak back into the left atrium instead of circulating to the rest of the body. The treatment options this study will compare are: (1) transcatheter edge-to-edge repair (abbreviated as TEER; which is a catheter procedure for repairing the mitral valve that doesn't require surgery to open up the heart) and (2) mitral valve repair surgery, which is an open-heart surgical procedure. There are no new or "experimental" procedures being tested in this study: both treatment options are well-established treatments and are regularly performed in patients who have MR.
The purpose of this study is to identify domains in which stroke suvivors may have struggled since experiencing a stroke. These include things like emotional, family and work function. This study entails an interview and questionnaires that ask about functioning in these areas.
The longer-term goal of this work is to identify areas these areas so that we can develop a psychotherapy that could be helpful for individuals recovering from and living with the aftermath of stroke.