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
298 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Baby is younger than 7 months old at the time of treatment start
- Corrected age is at least 39 weeks
- Confirmed diagnosis of Tuberous Sclerosis Complex (TSC) by clinical or genetic criteria
You will not qualify if you...
- Baby has had seizures or shows seizure activity on baseline EEG
- Baby has used or is using anti-seizure medications, systemic mTOR inhibitors, ketogenic diet, or other anti-seizure treatments
- Baby has participated in another investigational drug study within 30 days before screening
- Baby has a significant illness or active infection at screening
- History of severe prematurity (born before 30 weeks gestation) or serious medical problems at birth or neonatal period unrelated to TSC
- Abnormal kidney, liver, or bone marrow labs that pose safety risks
- Neurosurgery planned or done within 3 months before baseline
- Baby has TSC-related conditions needing mTOR treatment like SEGA or AML
- Investigator believes baby cannot comply with study requirements
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
J
Jessica Krefting, RN
CONTACT
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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