Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study

Launched by DARCY KRUEGER · Oct 22, 2021

Keywords

ClinConnect Summary

The Stopping TSC Onset and Progression 2B trial is a research study looking at whether a medication called sirolimus can help prevent or delay seizures in infants with Tuberous Sclerosis Complex (TSC). This study is currently recruiting participants, specifically infants who are between 0 to 6 months old and have a confirmed diagnosis of TSC. To be eligible, the baby must not have had any seizures before joining the study, and they should not be on any other anti-seizure medications or treatments that could interfere with the study.

If your baby participates, they will be randomly assigned to receive either sirolimus or a placebo (a pill that looks the same but has no active medicine). This means that neither you nor the doctors will know which treatment your baby is getting, which helps ensure the results are fair. Throughout the study, researchers will monitor your baby’s health to see how well the medication works and if it is safe. It's important to note that this study is supported by the FDA, which shows it has a significant backing in the medical community.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. 0-6 months of age at the time of enrollment (subject must be \<7 months of chronological age at time of randomization and treatment initiation). Corrected age must be at least 39 weeks (calculated by subtracting the number of weeks born before 40 weeks gestation from the chronological age).
• • 2. Has a confirmed diagnosis of TSC based on established clinical or genetic criteria
• Exclusion Criteria:
• • 1. Prior history of seizures (clinical or electrographic) at the time of enrollment or identified on baseline EEG.
• • 2. Has been treated in the past or is currently being treated at the time of enrollment with conventional anticonvulsant medications (AEDs), systemic (oral) mTOR inhibitors (such as rapamycin, sirolimus, or everolimus), ketogenic-related special diet, or another anti-seizure therapeutic agent, device, or procedure.
• • 3. Has taken any other investigational drug as part of another research study, within 30 days prior to the baseline screening visit.
• • 4. Has a significant illness or active infection at the time of the baseline screening visit
• • 5. Has a 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 other than TSC would convey additional risk of seizures or neurodevelopmental delay (i.e. HIE, severe neonatal infection, major surgery, prolonged ventilatory or other life-saving supportive care or procedures).
• • 6. Abnormal laboratory values at baseline (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.
• • 7. Prior, planned or anticipated neurosurgery within 3 months of the baseline visit
• • 8. Has a TSC-associated condition for which mTOR treatment is clinically indicated (i.e. SEGA or AML).
• • 9. Subjects who are, in the opinion of the investigator, unable to comply with the requirements of the study.