Neuro-oncology anywhere: Preliminary results from a decentralized clinical trial aimed at improving neurocognitive outcomes in patients with CNS malignancy.

Abstract

e13891 Background: Most patients with central nervous system (CNS) malignancy develop cognitive impairment, fatigue, and mood-related symptoms that are impacted by cancer and treatment, including radiation therapy. We currently have limited therapies to mitigate neurocognitive toxicity. It is important to accurately assess neurocognitive function and symptom burden when working to establish therapeutics. The objective of this trial is to assess the effectiveness of therapies aimed at improving neurocognitive function and quality of life by leveraging a decentralized approach incorporating an established digital neurocognitive assessment tool and development of a remote symptom collection/medication log to improve capture of longitudinal neurocognitive data. Methods: This is an open-label decentralized clinical trial (NCT06377696) that randomizes patients who have received intracranial radiation for primary and secondary brain tumors in a 1:1 allocation to health promotion with a wearable device +/- metformin (targeting radiation-related toxicity). Remote symptom collection is reported through digital patient surveys. Medication adherence is being monitored through a digital medication log developed for the study. Remote neurologic examination is performed by clinical neuro-oncologists at telehealth visits. Participants perform serial remote cognitive assessments with an established digital neurocognitive testing platform – Mayo Test Drive (MTD). Cognitive assessments and symptom surveys were designed to be completed on a smartphone, tablet, or computer device. Results: The study started enrollment on May 31, 2024. As of January 27, 2025, 54 of 100 planned participants have enrolled (27 on each arm). 51 participants remain active on trial; 3 withdrew from trial participation (2 prior to treatment, 1 after treatment). The overall completion rate for a quality-of-life questionnaire (EORTC QLQ-C30) is 85.0%, and each dose of metformin logged is 78.5%. The overall completion rate for virtual neurologic examination and short test of mental status is 94.7%. For MTD, the overall completion rate is 97.9%. The average MTD session duration was 16 minutes. Most participants completed MTD testing using a smartphone (90.5%) at home (89.1%). Conclusions: These results provide preliminary support that a decentralized trial assessing neurocognitive outcomes is feasible in patients with CNS malignancy. The completion rates of questionnaires and objective cognitive evaluation are comparable or higher than expected with historic norms. Final study results will provide real-world cognitive and quality-of-life data for patients with CNS tumors while providing preliminary data on the effectiveness of device-based health promotion +/- metformin for treatment related cognitive decline. Clinical trial information: NCT06377696 .