This study is examining the use, safety and performance of an investigational, meaning not approved for commercial use or sale by the US Food and Drug Administration (FDA) device called Transcatheter Mitral Valve Replacement (TMVR). The TMVR is a device used to treat mitral valve disease in patients with heart failure (weakened heart muscle). The device is placed by a non surgical approach using a delivery catheter (hollow tube) placed in your blood vessel at the top of your leg and directed up to your heart. This five year study will include a screening process to determine you meet eligibility criteria. If you qualify you will then be seen for a baseline visit, undergo the procedure to place the device, day after procedure, at hospital discharge, 1,3,6, 12 months, then yearly for up to 5 years. Study related testing includes physical exams, medication review, 6 minute hall walk test, questionnaires, blood work, CT scans, electrocardiogram or ECG (recording of your heart's electrical activity) and echocardiogram (ultrasound test of your heart).

The purpose of this two-visit brain imaging (magnetic resonance imaging, MRI) study is to identify brain targets for improving treatment and preventative interventions for individuals at risk for co-occurring bipolar disorder and alcohol use disorder. The preliminary visit(s) for a parent and his/her biological child will include completion of clinical interviews, surveys, and labwork to determine study eligibility. If they are considered eligible for the study, brain imaging visits will occur within 1-2 weeks at which a 1-hour MRI will be completed along with additional clinical interviews, surveys, and labwork. Brief follow-up phone call interviews will be completed with participants every 3 months for 1 year. Study participation is confidential and compensated.

This study aims to improve access of Veterans with epilepsy living in rural areas to the most important diagnostic procedure for the care of patients with epilepsy: the routine electroencephalogram (EEG). We will test a new method for recording EEG which uses a novel dry electrode system headset that does not require an EEG technologist to operate. The headset integrates the EEG electrodes and amplifier into a compact system which is easily placed on the head. This approach could make it possible for a nurse or nurse assistant with minimal training to record an EEG in a rural community based outpatient clinic (CBOC) as part of an epilepsy telemedicine outreach program along with clinical interviews. We will compare performance of this dry electrode system to standard EEG when it is used by EEG technologists in three VA medical centers. This project has the potential to improve access of Veterans to the EEG procedure and decrease cost to the Veterans Health Care System.

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 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.

Subjects in the first group will be enrolled in the GM-CSF for Reversal of Immunoparalysis in Pediatric Sepsis-induced MODS (GRACE-2) trial, comparing GM-CSF versus placebo. Subjects in the second group will be an observational cohort with no intervention, because this group has very low mortality and morbidity. Subjects in the third group will be enrolled in the Targeted Reversal of Inflammation in Pediatric Sepsis-induced MODS (TRIPS) trial, comparing anakinra and placebo. The fourth group, with very severe inflammation,
will be an observational cohort because clinical management of the inflammation
is standard of care, and there is no equipoise about enrolling these children in a placebo controlled trial. The primary outcome of both trials will be duration and severity of organ dysfunction using the cumulative PELOD-2 score, and secondary outcomes will assess health related quality of life and family functioning at 3 and 12 months.

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 study is to obtain long-term diabetes control information after patients' participation in the MSC in T1D trial. Specifically, the goal of this study is to determine if patients receiving an MSC infusion in addition to the standard of care for diabetes have a long-term beneficial effect in slowing disease progression than patients receiving placebo infusion.

This is a 3-part study, with each part having a unique set of objectives for male
adolescents aged 12 to < 18 years with fragile X syndrome (FXS). Part 1 is an openlabel,
single-dose, pharmacokinetics (PK) assessment of BPN14770 25 mg and
50 mg; Part 2 is double-blind (DB) and randomized; and Part 3 is an open-label
extension (OLE) for patients who complete Part 2.