This is an international, Phase IIb/III multi-center, double-blind, placebo-controlled, randomised trial assessing the efficacy and safety of spesolimab versus placebo in patients with moderate to severe HS. Approximately 200 trial participants in Part 1 (Phase IIb) and 260 trial participants in Part 2 (Phase III) will be randomised. For Part 1, after signing the informed consent, trial participants will enter the screening period for up to 28 days, and if all eligibility criteria are met, trial participants will be randomised in a 1:1:1:1 ratio to either active group, including high dose, medium dose, and low dose group, or placebo group. Once randomised, trial participants will start a treatment period of 50 weeks. Administration of treatment will be up to Week 48.

In this research study a study drug named XEN1101 is being tested for the treatment of seizures. The main purpose of this study is to determine if XEN1101 can reduce the number of seizures and if it is safe to use. XEN1101 has been tested in other studies in epilepsy patients and it was considered to be well-tolerated. It had side effects that are similar to other anti-seizure medications commonly used to help patients with epilepsy.

This research is being done to better understand how anti-cancer drugs affect cells within a tumor. Injecting very small amounts (microdoses) of pembrolizumab alone or in combination with MK-0482 or MK-4830 above may help the developer of these drugs to understand more about how these drugs work and what changes they make on the tumor. Subjects in this study will have their tumor injected with anti-cancer drugs using a CIVO device. The CIVO device delivers microdoses of multiple drugs or drug combinations directly into the tumor. Some of the procedures in this study include ultrasound assisted placement for tumor injection, a mucrodose injection of anti-cancer drugs into the tumor, and surgery to remove the tumor. Participation in the study will take 5 visits over a period if 56 days. The screening portion will be 28 days and the treatment portion will be 28 days from the time of the injection to the time of follow-up contact by the study team.

This is a multi-center, randomized, double-blind, placebo-controlled, phase II/III study to evaluate the efficacy and safety of spesolimab compared to placebo in the treatment of Netherton syndrome. This study will last up to 72 weeks with 16 clinic visits. Eligible patients will be randomized 2:1 to receive either spesolimab i.v. loading doses at Week 0 plus spesolimab subcutaneous doses every 4 weeks, or to receive placebo i.v. loading dose at Week 0 plus placebo subcutaneous doses every 4 weeks. At Week 20, all patients will enter the open label period to receive spesolimab subcutaneous dose every 4 weeks up to Week 52. There will be a safety follow up visit 16 weeks after the last dose.

Pimavanserin is an antipsychotic may have some beneficial effects on core autism symptoms and co-morbid conditions such as irritability, anxiety, sleep disorders, mood instability due to epilepsy, etc.). These potential benefits stem from pimavanserin's impact on the serotonin system in the body. This study will investigate pimavanserin in the treatment of irritability, core autism symptoms, and co-morbid conditions in children ages 6-17 years old.

The Southeastern Consortium for Lung Cancer Health Equity (SC3), led by Dr. Robert A. Winn, assembles an outstanding interdisciplinary team of translational researchers positioned in the heart of the historical and current tobacco-producing region within the southeast. Collectively, SC3's investigative team has unparalleled experience in lung cancer screening, translational research in lung cancer health disparities, community outreach and engagement, and recruiting and retaining racial and ethnic minorities and individuals from other medically underserved groups using evidence-based strategies. As NCI-designated cancer centers, all three centers report high enrollment of underserved minorities onto interventional trials and are committed to reducing the substantial disparities found in lung cancer outcomes in their collective Black/African American and rural communities.

The purpose of this pilot study is to evaluate safety and efficacy of a novel treatment, low intensity focused ultrasound pulsation (LIFUP) for treatment resistant depression (TRD). The initial visit will involve consent and an MRI scan, followed by two more treatment visits over the course of one week. During the first treatment day, participants will receive either focused ultrasound or sham stimulation. On the second treatment day, all participants will receive focused ultrasound. Response and potential side effects will be monitored pre- and post- each treatment along with one week and one month follow-up assessments. Follow-up assessments will also involve an MRI scan.

Social anxiety, which broadly consists of fears related to being negatively evaluated and avoidance of social situations, is highly prevalent among Veterans. Social anxiety is associated with significant deficits in social and occupational functioning. This study aims to evaluate a brief text-based intervention for decreasing social anxiety related safety behaviors among Veterans attempting to re-integrate into the workforce. Findings from this study will examine the efficacy of the text based intervention with aims at improving functional outcomes and quality of life among Veterans. Additional data from participating Veterans will provide valuable feedback on the intervention and assist in future refinement.

This is a Phase 3, placebo-controlled, randomized, double-blind research study evaluating the efficacy, safety, and PK of baricitinib in children from 6 years to less than 18 years of age with severe alopecia areata. The study is divided into 4 periods: a 5-week screening period, a 36-week double-blind treatment period, an approximately 2-year long-term extension period, and a 4-week posttreatment follow-up period. If the subject meets all eligibility criteria they will be randomized to receive either baricitinib high dose, baricitinib low dose, or placebo for 36 weeks. Participants will then be transitioned into the long-term extension treatment period. Subjects will attend 18 clinic visits for up to 145 weeks.

Individuals with Alpha-1 Antitrypsin Deficiency Associated Liver Disease who were enrolled in and completed the AROAAT2001 or AROAAT2002 study will be invited to participate in this study. This is a 2 year open label extension study of the drug Fazirsiran. Participants will receive 200mg of Fazirsiran every 12 weeks at the study site. Study visits will include blood work, pulmonary function test, questionnaires and liver evaluation testing. A liver biopsy will be performed at end of study, week 102. Potential risk include but are not limited to, allergic reaction to study medication or risk of infection or bleeding from biopsy. Safety and side effects of all assessments and therapies will be monitored throughout the study.