ong-term neuropsychologic outcome of pre-emptive mTOR inhibitor treatment in children with tuberous sclerosis complex (TSC) under 4 months of age (PROTECT) - PROTECT

Heidelberg University Hospital AöR0 个研究点目标入组 60 人开始时间: 2023年6月22日最近更新: 2024年7月22日

适应症

试验速览

暂无简介。

•Definite diagnosis of TSC according to the 2021 Updated International Tuberous Sclerosis Complex Diagnostic Criteria, <4 months of age at the time of enrolment (randomization and treatment initiation must occur before 4 months of age; infants born prematurely must have a corrected age of at least 39 weeks, calculated by subtracting the number of weeks born before 40 weeks gestation from the actual chronological age, in weeks), Signed informed consent from legal guardian(s) prior to any study specific procedure.

•Has a TSC-associated condition for which mTOR treatment is clinically indicated, i.e. subependymal giant cell astrocytoma (SEGA)., Has been treated in the past or is currently being treated at the time of enrolment with systemic mTOR inhibitors (such as rapamycin, sirolimus, or everolimus), Contraindication to study medication. Rapamune® oral solution contains soya oil. Patients allergic to peanut or soya must not take this medicine, Current enrolment, or observation period of competing clinical trials at any time during enrolment in the study., History of significant prematurity, defined as gestational age < 30 weeks at the time of delivery, or other significant medical complications at birth or during the neonatal period that, apart from TSC, would convey additional risk of seizures or neurodevelopmental delay (i.e. hypoxic ischemic encephalopathy (HIE), severe neonatal infection, major surgery, prolonged ventilatory or other lifesaving supportive care or procedures), Abnormal laboratory values at screening (i.e., renal function, liver function, or bone marrow production) that are in the opinion of the investigator clinically significant and may jeopardize the safety of the study subject., Parents / caregiver of the child who are, in the opinion of the investigator, unable to comply with the requirements of the study

发起方

Heidelberg University Hospital AöR