Spots Global Cancer Trial Database for Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
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Trial Identification
Brief Title: Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Official Title: Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Study ID: NCT05104983
Study Description
Brief 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
Minimum Age: 1 Day
Eligible Ages: CHILD
Sex: ALL
Healthy Volunteers: No
Locations
University of Alabama at Birmingham, Birmingham, Alabama, United States
University of California at Los Angeles, Los Angeles, California, United States
Stanford University, Palo Alto, California, United States
Boston Children's Hospital, Boston, Massachusetts, United States
Washington University -- St. Louis, Saint Louis, Missouri, United States
University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States
Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States
University of Texas HSC at Houston, Houston, Texas, United States
Seattle Children's Hospital, Seattle, Washington, United States
Contact Details
Name: Darcy A Krueger, MD, PhD
Affiliation: Children's Hospital Medical Center, Cincinnati
Role: PRINCIPAL_INVESTIGATOR
Name: Martina Bebin, MD, MPA
Affiliation: University of Alabama at Birmingham
Role: PRINCIPAL_INVESTIGATOR
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