AstraZeneca ((AZN)), AstraZeneca plc ((GB:AZN)), AstraZeneca ((DE:ZEGA)), AstraZeneca plc US ((AZNCF)) announced an update on their ongoing clinical study.
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AstraZeneca is conducting an observational study titled ‘Observational Study of the Effectiveness and Safety of the MEK 1/2 Inhibitor (MEKi) Koselugo (Selumetinib) in Pediatric Patients With Symptomatic, Inoperable, Neurofibromatosis Type 1 (NF1)-Associated Plexiform Neurofibromas (PN).’ The study aims to evaluate the real-world effectiveness and safety of selumetinib in Chinese pediatric patients with NF1-PN, a rare genetic disorder. This research is significant as it seeks to fill the data gap regarding the treatment’s impact in this specific population.
The intervention being tested is selumetinib, an oral selective inhibitor of MEK 1 and 2. It is intended to treat symptomatic, inoperable plexiform neurofibromas in pediatric patients with NF1, offering a targeted therapy option where surgical and conventional treatments have limited efficacy.
The study is designed as a prospective, multicenter, observational study involving approximately 80-100 pediatric patients across 12 centers in China. It follows a case-only observational model with a prospective time perspective, focusing on real-world data collection without any masking or allocation interventions.
The study began on December 20, 2023, with an enrollment period of 16 months. Patients will be observed for 24 months following their first dose of selumetinib. The last update was submitted on July 23, 2025, indicating the study’s ongoing status and relevance.
The market implications of this study are significant for AstraZeneca, as positive outcomes could enhance investor confidence and potentially boost stock performance. The study’s findings may also impact the broader industry by highlighting the effectiveness of targeted therapies in rare genetic disorders, setting a precedent for competitors.
The study is ongoing, with further details available on the ClinicalTrials portal.