NCT05104983 · Darcy Krueger
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
(TSC-STEPS)
What this study is about
This trial is a Phase II randomly assigned, where neither patients nor doctors know which treatment is given, placebo controlled multi-site study to evaluate the safety and effectiveness 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).
View original scientific 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).
Interventions
DRUG
Sirolimus
The investigational drug product to be used in this study is sirolimus, provided in oral suspension.
DRUG
Placebo
Matching placebo
Primary outcome measures
Efficacy -- time to seizure onset
Time frame: 12 months of age
Time to seizure onset, comparing sirolimus with placebo
Safety -- adverse events
Time frame: 12 months of age
Percentage of subjects reporting severe (CTCAE v5.0 grade \>= 3) adverse event (AE) or serious adverse event (SAE), comparing sirolimus with placebo.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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
Exclusion criteria
- 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 si
Where
- Birmingham, Alabama
- Los Angeles, California
- Palo Alto, California
- Aurora, Colorado
- Chicago, Illinois
- Boston, Massachusetts
- St Louis, Missouri
- Chapel Hill, North Carolina
- Cincinnati, Ohio
- Houston, Texas
- Seattle, Washington
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jan 22, 2026 · Source of record for eligibility and locations