CTIS2022-502332-39-00
Recruiting
Phase 1
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 sites60 target enrollmentJune 22, 2023
ConditionsTuberous sclerosis complex (TSC)MedDRA version: 21.0Level: LLTClassification code: 10045138Term: Tuberous sclerosis Class: 10010331Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Tuberous sclerosis complex (TSC)
- Sponsor
- Heidelberg University Hospital AöR
- Enrollment
- 60
- Status
- Recruiting
- Last Updated
- last year
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •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.
Exclusion Criteria
- •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
Outcomes
Primary Outcomes
Not specified
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