Norditropin® long-term use for Noonan Syndrome short stature monitored for safety and effectiveness

Noonan Syndrome (NS) is a relatively common genetic disorder characterized by distinctive facial features, heart defects, and often short stature. Growth hormone deficiency or insensitivity is a frequent cause of poor growth in NS patients, leading to the use of recombinant human growth hormone (rhGH) therapies like Norditropin® (somatropin). While rhGH is approved for treating short stature in NS, comprehensive long-term real-world data on its sustained effectiveness, safety profile, and impact on Health-Related Quality of Life (HRQoL) in this specific population remain crucial for optimizing clinical practice. This post-marketing surveillance study aims to systematically collect such data, addressing a key knowledge gap regarding the long-term management of growth in NS.

This is a non-interventional, post-marketing surveillance registry (NCT05308927) designed to collect real-world data on children with short stature due to Noonan Syndrome. Participants, estimated to be 221 individuals, will receive Norditropin® (somatropin) according to their usual clinical practice, meaning no specific study-mandated dose, route, or frequency. The study commenced on 2022-03-16 and is projected to complete by 2028-03-17. Primary endpoints include long-term growth evolution, comprehensive safety assessments, and evaluation of Health Related Quality of Life (HRQoL). Data collection will occur during routine medical visits, reflecting standard-of-care management.

This study is currently in the "ENROLLING_BY_INVITATION" phase, with an estimated completion date of 2028-03-17. Consequently, no results or findings are available from this specific registry at this time. The abstract describes the study's purpose and design rather than presenting any data on the long-term use of Norditropin® in children with Noonan Syndrome. Therefore, no specific numbers, p-values, or fold-changes can be reported from this ongoing surveillance.