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

Description

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

Tuberous Sclerosis Complex, Epilepsy

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Study Overview

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Study Details

Study overview

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).

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

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

Condition
Tuberous Sclerosis Complex
Intervention / Treatment

-

Contacts and Locations

Birmingham

University of Alabama at Birmingham, Birmingham, Alabama, United States, 35294

Los Angeles

University of California at Los Angeles, Los Angeles, California, United States, 90095

Palo Alto

Stanford University, Palo Alto, California, United States, 94304

Boston

Boston Children's Hospital, Boston, Massachusetts, United States, 02115

Saint Louis

Washington University -- St. Louis, Saint Louis, Missouri, United States, 63110

Chapel Hill

University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States, 27510

Cincinnati

Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States, 45229

Houston

University of Texas HSC at Houston, Houston, Texas, United States, 77030

Seattle

Seattle Children's Hospital, Seattle, Washington, United States, 98105

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 1. 0-6 months of age at the time of enrollment (subject must be \<7 months of chronological age at time of randomization and treatment initiation). Corrected age must be at least 39 weeks (calculated by subtracting the number of weeks born before 40 weeks gestation from the chronological age).
  • 2. Has a confirmed diagnosis of TSC based on established clinical or genetic criteria
  • 1. Prior history of seizures (clinical or electrographic) at the time of enrollment or identified on baseline EEG.
  • 2. Has been treated in the past or is currently being treated at the time of enrollment with conventional anticonvulsant medications (AEDs), systemic (oral) mTOR inhibitors (such as rapamycin, sirolimus, or everolimus), ketogenic-related special diet, or another anti-seizure therapeutic agent, device, or procedure.
  • 3. Has taken any other investigational drug as part of another research study, within 30 days prior to the baseline screening visit.
  • 4. Has a significant illness or active infection at the time of the baseline screening visit
  • 5. Has a history of significant prematurity, defined as gestational age \<30 weeks at the time of delivery, or other significant medical complications at birth or during the neonatal period that other than TSC would convey additional risk of seizures or neurodevelopmental delay (i.e. HIE, severe neonatal infection, major surgery, prolonged ventilatory or other life-saving supportive care or procedures).
  • 6. Abnormal laboratory values at baseline (i.e., renal function, liver function, or bone marrow production) that are in the opinion of the investigator clinically significant and may jeopardize the safety of the study subject.
  • 7. Prior, planned or anticipated neurosurgery within 3 months of the baseline visit
  • 8. Has a TSC-associated condition for which mTOR treatment is clinically indicated (i.e. SEGA or AML).
  • 9. Subjects who are, in the opinion of the investigator, unable to comply with the requirements of the study.

Ages Eligible for Study

1 Day to 6 Months

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Darcy Krueger,

Darcy A Krueger, MD, PhD, PRINCIPAL_INVESTIGATOR, Children's Hospital Medical Center, Cincinnati

Martina Bebin, MD, MPA, PRINCIPAL_INVESTIGATOR, University of Alabama at Birmingham

Study Record Dates

2026-06-30