Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Start Date
10/13/2021
Completion Date
6/30/2027
Summary
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).
Detailed Description
Tuberous Sclerosis Complex (TSC) is caused by genetic mutation in TSC1 or TSC2, resulting in dysregulation of the mechanistic target of rapamycin (mTOR) signaling pathway. Age at time of seizure onset in TSC infants has been linked to long-term neurodevelopmental outcome in this high-risk population. Sirolimus is an mTOR inhibitor used to treat many of the symptoms of TSC, including epilepsy. This will be the first study to truly evaluate a targeted, disease-modifying drug therapy for preventing or delaying seizure onset in TSC using a rational, mechanism-based therapeutic approach.
Eligibility Criteria
Age Range: No minimum to No maximum
Interventions
Sirolimus
Placebo
Conditions
Locations
University of Alabama at Birmingham
Birmingham, Alabama 35294
United States
University of California at Los Angeles
Los Angeles, California 90095
United States
Stanford University
Palo Alto, California 94304
United States
Children's Hospital Colorado
Aurora, Colorado 80045
United States
Lurie Children's Hospital of Chicago
Chicago, Illinois 60611
United States
Boston Children's Hospital
Boston, Massachusetts 02115
United States
Washington University -- St. Louis
St Louis, Missouri 63110
United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina 27510
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229
United States
University of Texas HSC at Houston
Houston, Texas 77030
United States
Seattle Children's Hospital
Seattle, Washington 98105
United States