Long-term neuropsychologic outcome of pre-emptive mTOR inhibitor treatment in children with tuberous sclerosis complex (TSC) under 4 months of age (PROTECT), a two-arm, randomized, observer-blind, controlled phase IIb national multicentre clinical trial: study protocol

CONCLUSION: This study addresses a critical gap in understanding the impact of pre-emptive mTOR inhibitor therapy on neuropsychologic outcomes in young TSC patients, aiming to improve overall patient outcomes and quality of life. EUCT number: 2022-502332-39-00, Registered 22/06/2023, https://euclinicaltrials.eu/search-for-clinical-trials/?lang=en&EUCT=2022-502332-39-00. …