Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
TSC-STEPS
2 other identifiers
interventional
64
1 country
11
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Oct 2021
Longer than P75 for phase_2
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
October 13, 2021
CompletedFirst Submitted
Initial submission to the registry
October 22, 2021
CompletedFirst Posted
Study publicly available on registry
November 3, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 30, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
June 30, 2027
January 22, 2026
January 1, 2026
4.7 years
October 22, 2021
January 20, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Efficacy -- time to seizure onset
Time to seizure onset, comparing sirolimus with placebo
12 months of age
Safety -- adverse events
Percentage of subjects reporting severe (CTCAE v5.0 grade \>= 3) adverse event (AE) or serious adverse event (SAE), comparing sirolimus with placebo.
12 months of age
Secondary Outcomes (5)
Neurodevelopmental Outcomes
12 and 24 months of age
Quality of Life Outcomes
12 and 24 months of age
EEG Biomarkers
12 and 24 months of age
MRI Biomarkers
12 and 24 months of age
Sirolimus Precision Dosing
12 months of age
Study Arms (2)
Sirolimus
EXPERIMENTALSirolimus
Placebo
PLACEBO COMPARATORPlacebo
Interventions
Eligibility Criteria
You may qualify if:
- 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).
- Has a confirmed diagnosis of TSC based on established clinical or genetic criteria
You may not qualify if:
- Prior history of seizures (clinical or electrographic) at the time of enrollment or identified on baseline EEG.
- 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.
- Has taken any other investigational drug as part of another research study, within 30 days prior to the baseline screening visit.
- Has a significant illness or active infection at the time of the baseline screening visit
- 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).
- 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.
- Prior, planned or anticipated neurosurgery within 3 months of the baseline visit
- Has a TSC-associated condition for which mTOR treatment is clinically indicated (i.e. SEGA or AML).
- Subjects who are, in the opinion of the investigator, unable to comply with the requirements of the study.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Darcy Kruegerlead
Study Sites (11)
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
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Darcy A Krueger, MD, PhD
Children's Hospital Medical Center, Cincinnati
- PRINCIPAL INVESTIGATOR
Martina Bebin, MD, MPA
University of Alabama at Birmingham
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR
- Purpose
- PREVENTION
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR INVESTIGATOR
- PI Title
- IND Sponsor/Lead Principal Investigator
Study Record Dates
First Submitted
October 22, 2021
First Posted
November 3, 2021
Study Start
October 13, 2021
Primary Completion (Estimated)
June 30, 2026
Study Completion (Estimated)
June 30, 2027
Last Updated
January 22, 2026
Record last verified: 2026-01