Clinical and Demographic Characteristics of Adult Patients With Neurofibromatosis in Russia

What this Trial Is About

This research aims to evaluate the clinical and demographic characteristics of adult patients with Neurofibromatosis type 1 (NF1) in Russia. It is an open-label, single-arm, non-interventional, multi-center cohort study that focuses on collecting patient-reported outcomes and clinical data during routine care. The study is designed to understand the various symptoms, complications, and comorbidities associated with NF1 in adult patients. Participants are adult patients diagnosed with NF1 and confirmed plexiform neurofibromas (PN) through clinical assessment or imaging methods such as MRI or ultrasound. Patients may be newly diagnosed or have established PN but must not have received MEK-inhibitor therapy for PN recently. The study does not involve any investigational treatments but observes patients during their usual care. The research collects data at the time of inclusion, including demographic information, disease characteristics, and medical history. During the study, participants undergo assessments including recording age at diagnosis, body measurements, symptom duration, disability degree, and prior medical examinations. Researchers also review hospitalization history related to NF1 and monitor changes in disability over time. The main outcome measures are collected at the initial visit, providing a comprehensive overview of the condition's impact on adult patients. The study will continue through routine follow-up visits without additional interventions, lasting until the study end date in March 2028.

Clinical and Demographic Characteristics of Adult p Atients With NEurofibromatosis in RUSsia