RecruitingPhase 2NCT05104983

Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study


Sponsor

Darcy Krueger

Enrollment

64 participants

Start Date

Oct 13, 2021

Study Type

INTERVENTIONAL

Conditions

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).


Eligibility

Min Age: 1 DayMax Age: 6 Months

Plain Language Summary

Simplified for easier understanding

This study is testing whether starting the drug sirolimus (an mTOR inhibitor) in babies with tuberous sclerosis complex (TSC) — before any seizures occur — can prevent epilepsy from ever developing. **You may be eligible if...** - Your baby has a confirmed diagnosis of tuberous sclerosis complex (TSC) based on genetic or clinical criteria - Your baby is 6 months old or younger (no older than 7 months at the start of treatment) - If premature, the corrected gestational age is at least 39 weeks - Your baby has NOT yet had any seizures (including those detectable only on a brain activity monitor) **You may NOT be eligible if...** - Your baby has already had any seizures - Your baby has already been treated with anti-seizure medications or mTOR inhibitors - Your baby has other serious health conditions that might interfere with the study Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGSirolimus

The investigational drug product to be used in this study is sirolimus, provided in oral suspension.

DRUGPlacebo

Matching placebo


Locations(11)

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

Children's Hospital Colorado

Aurora, Colorado, United States

Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Washington University -- St. Louis

St 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

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

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NCT05104983


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