Kromeriz 1

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.

This research aims to evaluate the long-term safety and tolerability of pelacarsen (TQJ230) in adults with established cardiovascular disease and elevated Lipoprotein(a) who have completed the parent trial CTQJ230A12301. The study is an open-label extension following the phase 3 parent study, providing participants continued access to pelacarsen after the initial trial. Participants will receive pelacarsen 80 mg by subcutaneous injection once a month during this open-label extension. The study is single-arm and multicenter, focusing on continued treatment with pelacarsen for up to 36 months after completion of the parent study. Throughout the study, participants will be monitored regularly to assess safety and tolerability, with particular attention to adverse events occurring up to 36 months. Researchers will collect data on health status throughout this period to understand the long-term effects of pelacarsen in this patient population.