Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
NCT ID: NCT05104983
Last Updated: 2026-01-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
64 participants
INTERVENTIONAL
2021-10-13
2027-06-30
Brief Summary
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This study is supported by research funding from the Office of Orphan Products Division (OOPD) of the US Food and Drug Administration (FDA).
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
PREVENTION
TRIPLE
Study Groups
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Sirolimus
Sirolimus
Sirolimus
The investigational drug product to be used in this study is sirolimus, provided in oral suspension.
Placebo
Placebo
Placebo
Matching placebo
Interventions
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Sirolimus
The investigational drug product to be used in this study is sirolimus, provided in oral suspension.
Placebo
Matching placebo
Eligibility Criteria
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Inclusion Criteria
2. Has a confirmed diagnosis of TSC based on established clinical or genetic criteria
Exclusion Criteria
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.
1 Day
6 Months
ALL
No
Sponsors
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Darcy Krueger
OTHER
Responsible Party
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Darcy Krueger
IND Sponsor/Lead Principal Investigator
Principal Investigators
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Darcy A Krueger, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Children's Hospital Medical Center, Cincinnati
Martina Bebin, MD, MPA
Role: PRINCIPAL_INVESTIGATOR
University of Alabama at Birmingham
Locations
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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
Countries
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Central Contacts
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Facility Contacts
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Jessica Krefting, RN
Role: primary
Angela Martinez
Role: primary
Sahana Raman
Role: primary
Olga Novak
Role: primary
Mariam Lugo Noguera
Role: primary
Mikaela Morales
Role: primary
Other Identifiers
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2021-0438
Identifier Type: -
Identifier Source: org_study_id
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