This study is for patients who have been diagnosed with prostate cancer that is prostate-specific membrane antigen (PSMA)-positive and has spread despite treatment with another androgen receptor pathway inhibitor (ARPI). This study is testing an investigational drug called AAA817. "Investigational" means it has not been approved by the United States Food and Drug Administration (FDA). In this study, participants will be randomly assigned (like flipping a coin) to receive AAA817 alone, AAA817 with an androgen receptor pathway inhibitor (ARPI), or standard of care treatment. The primary purpose of this study is to determine whether AAA817, given alone or in combination with an ARPI, is safe and effective compared to standard of care treatments. This drug is given to participants as a radioligand therapy infusion. Participants in this study can expect to be in the study for up to 6.1 years, including two visits before starting treatment, visits every 4 weeks during treatment, and visits every 12 weeks during long-term follow-up for up to 5 years after treatment ends.. There will be a total of 9 patients enrolled locally.

This study is for patients who have been diagnosed with metastatic hormone-sensitive prostate cancer (mHSPC).This study is testing an investigational drug called Tulmimetostat, which will be given in combination with standard prostate cancer treatments: Darolutamide or Abiraterone. "Investigational" means that Tulmimetostat has not been approved by the United States Food and Drug Administration (FDA). The primary purpose of this study is to determine the safety and potential benefits of Tulmimetostat when given with Darolutamide or Abiraterone. The study will also evaluate whether adding Tulmimetostat can delay the progression of prostate cancer compared to treatment with Darolutamide alone. Participants will receive the study drug by mouth, along with standard androgen deprivation therapy (ADT). Those receiving abiraterone will also take a low-dose corticosteroid (prednisone or prednisolone) to help prevent side effects. Participants can expect to be in the study as long as they are benefiting from treatment or until the study ends. During the first 4 weeks, study visits will occur on Days 1, 2, 8, 15, and 22. Beginning with Cycle 2, participants will have visits on Day 1 of every 4-week cycle. At study visits, participants will undergo health assessments such as physical exams, blood and urine tests, heart monitoring (ECG), imaging scans (CT or MRI), and health questionnaires. Some participants may also have an optional biopsy. A total of 8 patients will be enrolled locally over the course of 32 months.

This Phase III trial aims to investigate the efficacy and safety of survodutide (BI 456906) once weekly versus placebo on defined as resolution of steatohepatitis, reduction of liver fibrosis in liver biopsy and long-term improvement in clinical liver outcomes observed in trial participants with MASH and fibrosis stage F2-F3.

This study is for patients who have been diagnosed with low grade, intermediate risk non-muscle invasive bladder cancer. It is testing an investigational drug called TYRA-300. "Investigational" means that the drug has not been approved by the United States Food and Drug Administration (FDA).

The purpose of the study is to see whether TYRA-300 can shrink or eliminate bladder tumors without surgery. Normally, standard care involves a surgery called transurethral resection of bladder tumor (TURBT), but in this study at least one tumor will remain in place to see if the study drug works. The study will also evaluate the genetics of the cancer to see if certain mutations or changes in proteins affect how the drug works.

TYRA-300 is taken by mouth once daily. The dose level will be assigned by the study doctor. Participants may be in the study for about 36 months, which includes a screening period (up to 28 days), a study treatment phase (with visits every 4 weeks and more frequent visits during the first 8 weeks), an end-of-treatment visit, a safety follow-up about 28 days later, and long-term follow-up every 12 weeks. There will be a total of 3 patients enrolled locally over the course of 12 months.

This study aims to evaluate the clinical development program for icotrokinra in the treatment of adult participants with moderately to severely active CD. Icotrokinra may offer additional advantages beyond injectable antibody therapies and available
oral therapies for the treatment of patients with moderate to severely active CD due to its oral route of administration, high local exposure to GI tissues, and systemic activity. Overall, the program will evaluate icotrokinra treatment in a target of 1092 participants through at least 52 weeks but participants can participate in a long-term extension for 4 years (total duration approximately 5 years).

Study 812P310 is a multicenter, open-label extension study aimed to assess long-term safety and efficacy of SPN-812 in the treatment of ADHD in pediatric subjects who have participated in a previous blinded study of SPN-812.

All subjects who complete a blinded study of SPN-812 will have the option to participate in the OLE study in which all subjects receive SPN-812 (Study Medication, SM). Starting dose, dose range, and dose adjustments are all based on which double blind study the subject completed and, if applicable, the current age of the subject (Table 1). A subject who completed Study 812P202, 812P301 or 812P303, enters this OLE study at age 11 years, and then turns 12 while still in the study, their upper potential dose limit will then be raised from 400 mg to 600 mg and titration may occur at either 100 or 200 mg/week, as specified for the 12-17 year-old age group.

Feeding requires babies to coordinate sucking and swallowing, which depends on healthy brain development. Some newborns who experience brain injury or serious illness cannot safely practice feeding by mouth, which disrupts the development of these important brain circuits. As a result, many of these infants require a feeding tube placed in the stomach (called a G-tube) when they leave the hospital. Unfortunately, long-term feeding tube dependence is linked to poorer growth, delayed development, and breathing problems. Despite how common and serious these feeding difficulties are, there are currently no effective therapies for infants with the most severe swallowing problems. This study will test a non-invasive therapy called transcutaneous auricular vagus nerve stimulation (taVNS), which gently stimulates a nerve in the ear that connects to the brain. When paired with structured oral motor exercises, this approach may help strengthen the brain circuits needed for feeding. We will study 12 infants with severe feeding impairments to evaluate whether this therapy is safe, feasible, and shows early signs of benefit. This project will provide early evidence about whether a targeted brain-based therapy can improve feeding outcomes during a key window of early development.

This study is being conducted to evaluate the drug AZD0292, including how safe it is, how long it stays in the blood, and if it may help in reducing the number of exacerbations in people with Pseudomonas aeruginosa in their lungs. These patients have more frequent lung exacerbations and reduced quality of life. Pseudomonas aeruginosa is a bacteria that can make the symptoms of bronchiectasis worse. The study drug (AZD0292) works by attaching to Pseudomonas aeruginosa, thereby reducing its effects on the lungs and improving symptoms.
This study aims not only to test AZD0292 but is also being done to better understand bronchiectasis disease and its associated health problems.
Study drug (AZD0292) or placebo, will be given to participants as an intravenous (IV) infusion. The study is double-blinded.
About 435 participants with bronchiectasis 12 years of age and older, weighing at least 35kg will take part in this study. This study will be conducted globally in approximately 25 countries.

This study is designed to evaluate a new therapy formulation for Alpha-1 Antitrypsin Deficiency (AATD). AATD is an inherited condition in which a person has low blood levels of a protein known as alpha-1 protease inhibitor (called Alpha1-PI). AATD causes an increased risk of chronic obstructive pulmonary disease (COPD) in the form of emphysema (long term lung disease) and, less frequently, other diseases.

This study is being conducted to evaluate the safety and tolerability of 2 different doses of Alpha-1 drugs (Alpha-1 15% and Liquid Alpha1-PI) in participants with AATD. Participants will be placed into one of two groups. Each group will receive both drugs, but different doses of each, at different points in the treatment period (8 Weekly IV infusions of Liquid Alpha1-PI 60 mg/kg followed by 8 Weekly SC (subcutaneous) infusions Alpha1-PI 90 mg/kg OR 8 Weekly IV infusions of Liquid Alpha1-PI 120 mg/kg followed by 8 Weekly SC (subcutaneous) infusions Alpha1-PI 180 mg/kg).

This is an "open label" study, meaning participants and the study staff know which dose of study drug participants receive. The study will last up to 161 days (23 weeks). Many visits are able to be conducted through home health care, lessening the need to come into the clinic.

Alpha-1 15% is an investigational product, meaning it is not approved by the U.S. Food and Drug Administration (FDA). The other drug in this study is Liquid Alpha1-PI (licensed as Prolastin®-C Liquid) and is an FDA approved treatment for adults with emphysema due to AATD. However, it is only approved for the recommended dose of 60 mg/kg. This study includes both the FDA approved 60mg/kg of Liquid Alpha1-PI and an experimental dose of 120 mg/kg that is not FDA approved. Alpha-1 15% is given as an injection under the skin and Liquid Alpha1-PI is given as an infusion into the veins.

Optic neuritis (ON) is a rare inflammatory disorder of the optic nerve that results in vision loss. It is characterized by rapid and variable vision loss, pain in, around and behind the eye, and changes in color vision. Subjects in this study will be followed for 12 months total and randomized in a 2:1 ratio to receive either an IV infusion medication or a placebo infusion. Low Contrast Visual Acuity will be tracked at 3 months. After 3 months, participants will continue the study
through Month 12 in the safety follow-up part of the study intended to provide longer
term safety information.