Abstracts

Rationale: Infantile spasms (IS) are a rare and severe epileptic encephalopathy of infancy, typically presenting within the first year of life and often associated with developmental regression and poor neurological outcomes. While standard treatments such as adrenocorticotropic hormone (ACTH), vigabatrin, and corticosteroids are available, treatment response remains highly variable across patients. To better understand current therapeutic patterns and identify areas for improvement, we analyzed completed interventional trials listed on ClinicalTrials.gov. This database offers a centralized, publicly accessible source of clinical research activity. Investigating these trials helps clarify which therapies show the most promise, where evidence is lacking, and how future studies can better address the unmet clinical needs in IS management.

Methods: A structured search of ClinicalTrials.gov was conducted on March 7, 2025, using the term “infantile spasms.” The search was limited to completed interventional studies evaluating pharmacologic treatments. Trials with observational or expanded access designs, or those not meeting completion criteria, were excluded. Thirteen eligible studies were identified and systematically reviewed. Key elements extracted included therapeutic agents, study design, primary endpoints, and reported efficacy and safety outcomes.

Results: The included trials assessed a range of pharmacologic agents, spanning both established therapies (ACTH, vigabatrin, prednisolone) and investigational compounds (cannabidiol, ganaxolone, TAK-935, tricaprilin, pyridoxine, lithium carbonate). Efficacy outcomes most commonly focused on seizure reduction, EEG normalization, and developmental progression. Vigabatrin showed continued benefit in tuberous sclerosis complex–related IS, while cannabidiol and TAK-935 demonstrated potential in broader developmental epileptic encephalopathies. Adverse effects varied by agent, with notable concerns including vigabatrin-associated visual field defects and gastrointestinal disturbances linked to cannabidiol and tricaprilin. The clinical heterogeneity of IS was reflected in the variable outcomes reported, underscoring the need for individualized therapeutic strategies.

Conclusions: This review highlights the diversity of pharmacologic interventions under investigation for IS and reinforces the role of both established and emerging therapies. While completed trials provide valuable insight into evolving treatment strategies, important gaps remain—particularly the lack of patient-level outcomes and long-term neurodevelopmental data. Future research should focus on stratified study designs, biomarker-informed approaches, and extended follow-up to optimize treatment selection and improve clinical outcomes in this high-risk pediatric population.

Funding: The author declares that no funding was received for this study.