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.
This research study aims to learn more about children and adolescents who have a shiga toxin-producing E. coli (STEC) infection. E. coli is a type of bacteria found in the intestines. Although most types of E. coli are harmless, some produce toxins that can make children sick. This study will assess what type of treatment is best for this infection.
The purpose of this study is to see if taking depemokimab is safe and effective in treating Hypereosinophilic syndrome (HES) in adults (≥18 years) with uncontrolled HES receiving standard of care (SoC) therapy. The study will last approximately 52 weeks and is a placebo-controlled, double blind, multicentre study.
This study will compare WATCHMAN FLXTM ("DEVICE") to a category of OAC called non-vitamin K antagonist oral anticoagulation, also known as NOACs ("CONTROL"). NOAC medications include Pradaxa® (dabigatran), Eliquis® (apixaban), Xarelto® (rivaroxaban), or Savaysa® (edoxaban).The study will research the safety and effectiveness of the WATCHMAN FLXTM device compared to treatment with NOAC therapy to determine if it works for patients with your type of atrial fibrillation who require treatment for potential blood clot formation (thrombus) in the LAA.
People who experience a stroke often have problems performing many different tasks during daily life. Most studies investigating stroke recovery and rehabilitation focus only on one type of task. The purpose of this research study is to measure individuals' function in many areas that can be affected by a stroke, such as their balance, mood, memory, reaching, and speech. This study will also track changes that can occur with task performance over time.
The purpose of this study is to examine the relationship between reflexes in the leg and the presence of neuropathic pain. The researchers are recruiting 30 individuals with spinal cord injury (SCI) total, 15 individuals with neuropathic pain due to SCI and 15 individuals without neuropathic pain. For this portion of the study, there are 2 visits. The first visit will examine cutaneous reflexes in the leg. During the second visit, the study team will assess sensation in the leg and administer questionnaires about pain, functioning, and quality of life.
The purpose of the second part of the study is to examine the effect of reflex training in the leg to decrease neuropathic pain. For this, the researchers are recruiting 15 individuals with neuropathic pain due to spinal cord injury to participate in the reflex training procedure. The study involves approximately 50 visits with a total study duration of about 6.5 months (3 months for baseline and training phases followed by 1 month and 3 month follow-up visits).
Sensory stimulation has been shown to enhance rehabilitation outcomes. However, most sensory stimulation devices interfere with natural hand tasks. Thus, a new wearable stimulation device has been developed to deliver imperceptible vibration to wrist skin. This study is to evaluate the community use of the device for patients with neurologic movement disorders. Participation will include wearing the provided device and charging the device every night. The knowledge regarding community use of the device may contribute to improving the device functionality and usability for future users of the device.
This study is for patients who have had surgery for pancreatic neuroendocrine tumor (pNET). This study is being done to find out if giving chemotherapy after surgery is better or worse than the usual approach for patients that have had surgery for pNET.
The purpose of this study is to create a de-identified, public use,
repository of data of Chronic Obstructive Pulmonary Disease (COPD)
patients with by Alpha-1 antitrypsin deficiency (AATD), a rare genetic
condition that can cause COPD and emphysema.
This study is for patients that have been diagnosed with leukemia. This study is called a screening study and we are doing this study to find better ways to diagnose and treat leukemia in children, adolescents and young adults. Bone marrow, blood, and medical information about participant's cancer and treatment will be collected. Participants can expect to be on this study for 5 years.