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Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Recruiting
Trial ID: NCT05104983
Purpose
This trial is a Phase II randomized, double-blind, placebo controlled multi-site study to
evaluate the safety and efficacy of early sirolimus to prevent or delay seizure onset in TSC
infants.
This study is supported by research funding from the Office of Orphan Products Division
(OOPD) of the US Food and Drug Administration (FDA).
Official Title
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Stanford Investigator(s)
Eligibility
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.
Intervention(s):
drug: Sirolimus
drug: Placebo
Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Jennifer Winterbottom