Schondorf

Researchers are evaluating Trastuzumab deruxtecan (T-DXd) in adult patients with unresectable or metastatic HER2-low expressing breast cancer. This non-interventional study aims to assess the effectiveness of T-DXd, patients' demographic and clinical characteristics, treatment patterns, tolerability, management of adverse drug reactions, and patient experience. The study also collects data on conventional chemotherapy treatments in a disease registry to better understand treatment outcomes in this population. Participants will receive treatment with Trastuzumab deruxtecan or conventional chemotherapy drugs such as capecitabine, eribulin, gemcitabine, paclitaxel, or nab-paclitaxel according to the Summary of Product Characteristics and routine clinical practice. No study drug will be administered by the researchers, as treatments follow physicians' standard care decisions. This approach allows observation of real-world treatment use and outcomes. During the study, patients' treatment timelines and responses will be followed, focusing on the time to next treatment up to 31 months. Researchers will monitor tolerability, adverse drug reactions, and patient-reported experiences. Data collection includes clinical and demographic information, treatment patterns, and outcomes to provide a comprehensive understanding of T-DXd and conventional chemotherapy use in this patient group.

Researchers are monitoring the long-term safety and effectiveness of Increlex4, a treatment for children and adolescents with Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD). This global registry study is observational and non-interventional, designed to collect safety data during treatment and for at least five years after treatment ends. The study includes participants who have already started Increlex4 therapy as well as those beginning treatment, across various countries including the USA and several European nations. Participants receive Increlex4, a mecasermin injection given twice daily at doses ranging from 40 to 120 mcg/kg or 0.04 to 0.12 mg/kg, as prescribed by their physician. The study does not assign treatments but records data from patients undergoing routine care. This registry captures real-world use of Increlex4 according to local approved guidelines for managing SPIGFD. Throughout the study, researchers collect information on serious adverse events, any adverse events, deaths, and withdrawals related to treatment. Data collection continues during the treatment period and up to 30 days after the last dose. Safety monitoring is the primary focus, with long-term follow-up planned for at least five years post-treatment to assess ongoing health outcomes in children and adolescents receiving Increlex4 therapy.

Researchers are evaluating the effect of balcinrenone/dapagliflozin compared with dapagliflozin alone on cardiovascular death and heart failure events in patients with chronic heart failure and impaired kidney function who recently experienced a heart failure event. This is a Phase III, international, randomized, double-blind, parallel-group, active-controlled study involving approximately 700 sites in about 40 countries. Participants will be randomly assigned in a 1:1:1 ratio to receive one of three treatments once daily: a capsule of balcinrenone/dapagliflozin 15 mg/10 mg with a placebo tablet, a capsule of balcinrenone/dapagliflozin 40 mg/10 mg with a placebo tablet, or a dapagliflozin 10 mg tablet with a placebo capsule. The study is event-driven, with an estimated average duration of 22 months that includes a screening period, a 20-month blinded treatment phase, and a one-month follow-up on open-label dapagliflozin. During the study, participants will be monitored for the time to first occurrence of cardiovascular death, heart failure hospitalization, or heart failure events without hospitalization over approximately 38 months. Assessments include clinical evaluations, laboratory tests, and safety monitoring throughout the study and follow-up period to track treatment effects and patient outcomes.