Cheektowaga

This research investigates the effects of AP301, a new iron-based phosphate binder, in patients with chronic kidney disease who are receiving maintenance dialysis and have high blood phosphate levels. The study aims to find out if AP301 can lower blood phosphate and how it influences serum calcium, calcium times phosphate levels, and parathyroid hormone levels. Researchers also want to understand what side effects or discomfort patients might experience and whether AP301 improves quality of life in Chinese patients. The trial is a Phase 3, randomized, double-blind, multi-regional study comparing AP301 with a low-dose version of the same drug that acts as a placebo-like comparator. Participants will first stop all phosphate-lowering drugs. Then, they will take either AP301 or the low-dose comparator three times daily with meals for 8 weeks. After that, all participants will take AP301 three times daily for 24 weeks. Finally, they will take either AP301 or the comparator for 3 weeks. During the first 32 weeks, the dose of AP301 may be adjusted up or down based on blood phosphate levels and the study doctor's judgment. Additional treatment may be given if phosphate levels become too high or too low. Throughout the study, patients will be monitored for changes in serum phosphate levels, calcium levels, and parathyroid hormone levels. Researchers will also assess side effects and quality of life. The main outcome measured is the change in serum phosphate concentration from the start of the study to the end of week 8. Participation requires patients to be on dialysis for at least 3 months and to meet certain blood phosphate and calcium criteria, and the study will last at least 35 weeks with various assessments.

Researchers are evaluating the safety and effectiveness of intravesical nadofaragene firadenovec alone or combined with chemotherapy or immunotherapy in people with high-grade Bacillus Calmette-Guerin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC) that includes carcinoma in situ (CIS) with or without high-grade Ta/T1 disease. This phase 3 trial builds on earlier findings where over half of participants achieved a complete response after treatment with nadofaragene firadenovec alone at 3 months. The study aims to better understand treatment options for patients who have not responded to BCG therapy. Participants will receive intravesical nadofaragene firadenovec, a gene therapy designed to boost immune response by delivering the human interferon alfa-2b gene directly into the bladder. Some participants may also receive chemotherapy drugs gemcitabine and docetaxel, or the immunotherapy pembrolizumab administered through intravenous infusion. The treatments are given inside the bladder or by IV infusion as appropriate, and the study will assess these approaches alone or in combination. During the study, participants will be closely monitored for response to treatment, particularly looking for complete response up to 6 months after starting therapy. Researchers will regularly evaluate participants’ health status and tumor response, including safety assessments and follow-up visits over the course of the trial. The study requires participants to be available for the entire duration and includes various clinical assessments to track effectiveness and side effects.

Researchers are evaluating semen quality in men aged 22 to 65 who have an anterior urethral stricture treatable with the Optilume Urethral Drug Coated Balloon (DCB). This single-arm, prospective Phase 4 study aims to assess andrology parameters following treatment with this device. The study will enroll 34 men at up to ten sites across the United States to monitor urinary, sexual, and voiding functions over time. Participants will be treated with the Optilume Urethral DCB, a catheter with a balloon coated in paclitaxel, a drug designed to transfer to the urethral wall when the balloon is inflated. Clinical follow-up visits are planned at 30 days, 3 months, 6 months, and 12 months after treatment to evaluate symptoms and functions related to the lower urinary tract and sexual health. Semen quality will be assessed at baseline, 3 months, and 6 months post-treatment, with further assessments at 12 months and beyond if abnormalities are detected at 6 months. During the study, participants will provide semen samples and undergo evaluations of urinary and sexual function. Researchers will monitor semen quality parameters such as sperm count, concentration, motility, and morphology. Safety will also be tracked, focusing on a primary safety endpoint at 3 months. The total follow-up period extends to at least 12 months, with additional monitoring if needed to ensure semen quality returns to normal.

A phase 3b, Randomized, Controlled Trial of Nadofaragene Firadenovec vs. Observation in Participants with Intermediate Risk Non-Muscle Invasive Bladder Cancer (IR NMIBC)