Actively Recruiting
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Led by Darcy Krueger · Updated on 2026-01-22
64
Participants Needed
11
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to evaluate whether early treatment with sirolimus can prevent or delay the start of seizures in infants with Tuberous Sclerosis Complex (TSC), a genetic condition caused by mutations affecting the mTOR signaling pathway. This Phase II randomized, double-blind, placebo-controlled trial investigates the safety and effects of sirolimus in this high-risk infant population. The study is supported by the US FDA's Office of Orphan Products Division. Participants will be randomly assigned to receive either sirolimus oral suspension or a matching placebo. Treatment begins before infants reach seven months of age and continues while researchers monitor for seizure onset. The study compares the timing of seizures and collects data on safety, neurodevelopment, quality of life, and biological markers through EEG and MRI at 12 and 24 months of age. During the trial, infants will undergo regular assessments including neurological exams, EEG and MRI scans, and developmental evaluations to track seizure onset and brain development. Researchers will monitor safety by recording any adverse events and adjust sirolimus dosing as needed. The primary outcomes focus on the time to first seizure and safety at 12 months, with additional follow-ups to 24 months to assess longer-term effects.
CONDITIONS
Brief Title
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 0 to 6 months old at enrollment, with corrected age at least 39 weeks
- Confirmed diagnosis of Tuberous Sclerosis Complex (TSC) by clinical or genetic criteria
You will not qualify if you...
- History of seizures or abnormal EEG at enrollment
- Prior or current treatment with anticonvulsant medications, systemic mTOR inhibitors, ketogenic diets, or other anti-seizure therapies
- Use of investigational drugs within 30 days before screening
- Significant illness or active infection at screening
- History of significant prematurity (gestational age less than 30 weeks) or major medical complications at birth aside from TSC
- Abnormal baseline lab values deemed unsafe by the investigator
- Neurosurgery planned or done within 3 months of baseline
- Presence of TSC-related conditions requiring mTOR treatment (such as SEGA or AML)
- Inability to comply with study requirements as judged by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 12 months of age
Participants receive either sirolimus or placebo to evaluate prevention or delay of seizure onset in TSC.
Regular visits during treatment as scheduled by the study team
Duration - Up to 24 months of age
Participants are monitored for neurodevelopmental outcomes, quality of life, and biomarker assessments after treatment ends.
Visits at 12 and 24 months of age
Trial Site Locations
Total: 11 locations
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35294
Actively Recruiting
2
University of California at Los Angeles
Los Angeles, California, United States, 90095
Actively Recruiting
3
Stanford University
Palo Alto, California, United States, 94304
Actively Recruiting
4
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
5
Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
6
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
7
Washington University -- St. Louis
St Louis, Missouri, United States, 63110
Actively Recruiting
8
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27510
Active, Not Recruiting
9
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
10
University of Texas HSC at Houston
Houston, Texas, United States, 77030
Actively Recruiting
11
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Actively Recruiting
Research Team
M
Molly S Griffith, BA
J
Jessica Krefting, RN
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
PREVENTION
Number of Arms
2
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