NCT05104983Active, not recruitingPhase 2
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Source: ClinicalTrials.gov · Last updated 29 Oct 2026
Brief summary (registry, in English)
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).
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 64 (estimated)
- First submitted
- 22 Oct 2021
- Start date
- 13 Oct 2021
- Primary completion date
- 30 Jun 2027
- Lead sponsor
- Darcy Krueger
- Diseases
- Epilepsy
- NCT number
- NCT05104983
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 64
- From 13 Oct 2021 to the primary completion date, 30 Jun 2027
- 8 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Prior history of seizures (clinical or electrographic) at the time of enrollment or identified on baseline EEG.
- Has taken any other investigational drug as part of another research study, within 30 days prior to the baseline screening visit.
- Prior, planned or anticipated neurosurgery within 3 months of the baseline visit
Other illness
- 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.
- Has a significant illness or active infection at the time of the baseline screening visit
- Has a TSC-associated condition for which mTOR treatment is clinically indicated (i.e. SEGA or AML).
Lab values
- 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.
Age
- 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).
Other
- Subjects who are, in the opinion of the investigator, unable to comply with the requirements of the study.
Who the study is looking for
- 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).
- Has a confirmed diagnosis of TSC based on established clinical or genetic criteria
Age in the registry: 1 day to 6 months · Sex: All · Does not accept healthy volunteers
Sites (8)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| University of Alabama at Birmingham Coordinator of this site? Confirm its status | Birmingham | United States | Unknown status |
| Boston Children's Hospital Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| University of North Carolina at Chapel Hill Coordinator of this site? Confirm its status | Chapel Hill | United States | Unknown status |
| Cincinnati Children's Hospital Medical Center Coordinator of this site? Confirm its status | Cincinnati | United States | Unknown status |
| University of Texas HSC at Houston Coordinator of this site? Confirm its status | Houston | United States | Unknown status |
| University of California at Los Angeles Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
| Stanford University Coordinator of this site? Confirm its status | Palo Alto | United States | Unknown status |
| Seattle Children's Hospital Coordinator of this site? Confirm its status | Seattle | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.