McGehee

Researchers are evaluating SYX-5219 in a multi-part, first-in-human Phase 1 study involving healthy volunteers and people with moderate to severe atopic dermatitis AD. The study aims to determine the safety, tolerability, and levels of SYX-5219 in blood and urine when given in different dosing schedules. This includes single ascending doses, multiple ascending doses, and multiple dose administrations in participants with AD. The study has three parts Part 1 involves single ascending doses and a food effect evaluation in healthy volunteers, with doses given once on Day 1 and again after a washout period. Part 2 involves multiple ascending doses given once or twice daily to healthy volunteers over a defined period. Part 3 enrolls participants with AD who receive SYX-5219 or placebo twice daily for up to 42 days. Each part includes randomization and placebo control to compare effects. Participants will undergo safety and exploratory efficacy assessments during and after treatment, including monitoring adverse events up to 10 days after dosing in Part 1, 14 days after the last dose in Part 2, and up to Day 56 in Part 3. Blood and urine samples are collected at multiple timepoints to measure SYX-5219 levels. The study includes follow-up visits to monitor participant health and drug effects for several weeks after treatment ends.

Researchers are studying UGN-104, a new formulation of UGN-101 also known as JELMYTO, to evaluate its effectiveness and safety in treating patients with low-grade upper tract urothelial cancer LG-UTUC. This phase 3, single-arm study focuses on patients with this specific type of cancer affecting the upper urinary tract, aiming to assess how well the treatment works and its safety profile. Participants will receive UGN-104 once a week for six weeks, with each dose administered directly into the upper urinary tract via a ureteral catheter or nephrostomy tube. The dose consists of 4 mg mitomycin per 1 mL sterile hydrogel. After the initial treatment period, patients who have no detectable disease at the primary disease evaluation visit about three months after the first dose may enter a follow-up phase where they could receive monthly maintenance doses for up to 11 months, depending on the investigators decision. During the study, participants will have evaluations every three months to check for disease response or recurrence. These assessments include urine cytology, visual inspection via ureteroscopy, and biopsies if needed. Researchers will monitor the complete response rate at three months as the primary outcome and track the duration of response, durable complete response rate, and any treatment-related side effects for up to 15 months. The total participation time varies depending on response and disease status.

This research aims to evaluate the effects of TAK-330 compared to standard four-factor prothrombin complex concentrate 4F-PCC in adults treated with Factor Xa inhibitors who need urgent surgery or invasive procedures. The study focuses on reversing anticoagulation caused by Factor Xa inhibitors and is a Phase 3, randomized, triple-blind trial sponsored by Takeda. Participants will be randomly assigned to receive either TAK-330 or the standard 4F-PCC. TAK-330 is given as a single intravenous infusion dose of 25 IUkg before surgery, with an optional additional 25 IUkg dose during surgery if needed, not exceeding a total of 50 IUkg or 5,000 IU. The standard 4F-PCC dose and infusion speed follow local protocols, with an additional dose allowed during surgery under the same maximum limits. These treatments are administered during hospitalization prior to surgery. During the study, participants will be hospitalized for the surgery and treatment administration, with assessments of hemostasis effectiveness at surgery end and 24 hours after treatment. Researchers will monitor blood product use, adverse events including thrombotic events, and participant survival up to 30 days after surgery. Follow-up will also include contact via telehealth or phone 30 days post-surgery to evaluate safety and outcomes. Total participation duration covers the hospital stay and the 30-day post-surgery monitoring period.