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Long-term neuropsychologic outcome of pre-emptive mTOR inhibitor treatment in children with tuberous sclerosis complex (TSC) under 4 months of age (PROTECT)
EU CTIS ID: 2022-502332-39-00
What this study is testing
Neuropsychologic outcome at 24 months of age assessed by rater blinded neuropsychologic testing measured by the cognitive scale on the Bayley Scales of Infant and Toddler Development III (BSID-III) compared with Standard of Care (SOC) alone.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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.
You likely can't join if
- 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.
See the full eligibility 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.
- 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
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.