The clinical trials landscape of hydrocephalus: a scoping review of therapeutic priorities, innovation trends, and research gaps.

Hydrocephalus includes a diverse group of disorders with different causes, symptoms, and treatment options. Despite progress in surgery and diagnostics, the overall understanding of clinical research in hydrocephalus remains limited. We performed a scoping review to systematically examine registered clinical trials, aiming to identify trends in treatment approaches, research areas, and gaps in the current evidence. A comprehensive search of ClinicalTrials.gov was conducted to identify all registered clinical trials related to hydrocephalus. Trials were included regardless of study design, phase, or geographic location. Extracted variables included study characteristics, hydrocephalus subtype, intervention type, research domain, recruitment status, and geographic distribution. Trials were categorized into interventional, device-focused, and biomarker/imaging domains, and analyzed descriptively to assess temporal trends, global distribution, and research priorities. A total of 90 trial entries were identified. The majority of studies focused on adult populations (84.4%), with normal pressure hydrocephalus (NPH) representing the predominant subtype (71.1%). Interventional trials accounted for 60.0% of studies, followed by biomarker and imaging investigations (25.6%) and device-focused trials (14.4%). Surgical and procedural interventions were most common (43.3%), while pharmacologic therapies were limited (7.8%). Clinical trial activity has increased substantially over time, with nearly half of all trials (47.8%) registered between 2020 and 2026. Geographic distribution was heavily concentrated in high-income regions, particularly Europe (46.5%) and North America (32.3%), with limited representation from other regions. Most studies were conducted within a single country (91.1%), with minimal international collaboration. At the time of analysis, 30% of trials were reported as completed status, while 31.1% remained active. The overall trial completion status was 67.5%. The landscape of hydrocephalus clinical trials is rapidly growing but still uneven, mainly focusing on NPH studies and device- and procedure-based treatments, with little development in non-surgical therapies, reflecting the predominantly structural nature of hydrocephalus. Research efforts are mostly in high-income areas with limited global collaboration. These results reveal important gaps in treatment development, trial design, and geographic diversity, emphasizing the need for a more balanced and worldwide research approach to improve hydrocephalus management.