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Active, not recruitingNCT05104983TSC-STEPSUpdated Oct 29, 2026

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

A Phase 2 interventional study of Sirolimus and Placebo in Tuberous Sclerosis Complex and Epilepsy, sponsored by Darcy Krueger. Active, not recruiting at 8 sites in United States. Open to participants aged 1 Day to 6 Months. Per ClinicalTrials.gov, last updated 2026-10-29.

Sponsored by Darcy Krueger · Phase 2, Interventional, and Prevention

Updated Oct 29, 2026Now Active, not recruitingPrimary completion moved+2 moreGo to Updates ↓
Phase
Phase 2
Study type
Interventional
Enrollment
64
Allocation
Randomized
Ages
1 Day to 6 Months
Sex
All
01

Study 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).

Read the detailed description

Tuberous Sclerosis Complex (TSC) is caused by genetic mutation in TSC1 or TSC2, resulting in dysregulation of the mechanistic target of rapamycin (mTOR) signaling pathway. Age at time of seizure onset in TSC infants has been linked to long-term neurodevelopmental outcome in this high-risk population. Sirolimus is an mTOR inhibitor used to treat many of the symptoms of TSC, including epilepsy. This will be the first study to truly evaluate a targeted, disease-modifying drug therapy for preventing or delaying seizure onset in TSC using a rational, mechanism-based therapeutic approach.

02

Conditions studied

  • Tuberous Sclerosis Complex
  • Epilepsy

Keywords

  • Tuberous Sclerosis Complex
  • TSC
  • epilepsy
  • prevention
  • mTOR
  • sirolimus
  • infant
03

In context

Tuberous Sclerosis

115 studies on the registry are indexed under Tuberous Sclerosis; 27 are open to participants now.

This study's planned enrollment of 64 is above the median of 52 across 73 interventional studies indexed under Tuberous Sclerosis.

Browse Tuberous Sclerosis studies →

Lead sponsor

This is the only study on the registry with Darcy Krueger as lead sponsor.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
1 Day to 6 Months
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion 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

Exclusion criteria

Exclusion 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 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 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.
Terms in these criteria, in plain language(2)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

05

Study design

Phase
Phase 2
Primary purpose
Prevention
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Triple (Participant, Care provider, Investigator)
Enrollment
64 participants (estimated)

Study arms

  • Experimental
    Sirolimus

    Sirolimus

    Drug: Sirolimus

  • Placebo comparator
    Placebo

    Placebo

    Drug: Placebo

Interventions

  • DrugSirolimus

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

  • DrugPlacebo

    Matching placebo

06

What researchers measure

Primary outcomes

  1. Efficacy -- time to seizure onset

    Time to seizure onset, comparing sirolimus with placebo

    Time frame: 12 months of age

  2. 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.

    Time frame: 12 months of age

Secondary outcomes

  1. Neurodevelopmental Outcomes

    Neurodevelopmental outcomes at the end of treatment, comparing sirolimus with placebo.

    Time frame: 12 and 24 months of age

  2. Quality of Life Outcomes

    Patient and caregiver quality of life, comparing sirolimus with placebo.

    Time frame: 12 and 24 months of age

  3. EEG Biomarkers

    EEG measures of neuronal connectivity, comparing sirolimus with placebo.

    Time frame: 12 and 24 months of age

  4. MRI Biomarkers

    MRI measures of neuronal connectivity, comparing sirolimus with placebo.

    Time frame: 12 and 24 months of age

  5. Sirolimus Precision Dosing

    Validate the feasibility and effectiveness of sirolimus precision dosing in infants with TSC

    Time frame: 12 months of age

07

Study locations

8 sites
08

Timeline

2 registry updates since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedDate before it movedWhere the date was before it moved

Started Oct 2021 (actual). Primary completion is estimated for Jun 2027, 68 months after the start.

Since we started tracking on Sep 25, 2026, the dates moved once, on Oct 2, 2026: primary completion Jun 2026 → Jun 2027 and completion Jun 2027 → Jun 2028.

Earlier changes are on the record history on ClinicalTrials.gov ↗

How these figures are worked out, and which studies are left out of them.

Registered
Oct 22, 2021
First posted
Nov 3, 2021
StartActual
Oct 13, 2021
Primary completionEst.
Jun 30, 2027was Jun 2026
CompletionEst.
Jun 30, 2028was Jun 2027
Last update
Oct 29, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Status
Recruiting→Active, not recruiting
changed Oct 29, 2026
Sites
3 sites removed
Show 3 removed
  • Children's Hospital Colorado · Aurora, United States
  • Lurie Children's Hospital of Chicago · Chicago, United States
  • Washington University -- St. Louis · St Louis, United States
Oct 29, 2026
Primary completion
Jun 30, 2026→Jun 30, 2027
Oct 2, 2026
Study completion
Jun 30, 2027→Jun 30, 2028
Oct 2, 2026
Show all 2 updates
  1. Oct 29, 2026
    Recruiting→Active, not recruiting
    3 sites removed
    Show 3 removed
    • Children's Hospital Colorado · Aurora, United States
    • Lurie Children's Hospital of Chicago · Chicago, United States
    • Washington University -- St. Louis · St Louis, United States
    + 2 other changes: verification date and contact details
  2. Oct 2, 2026
    Primary completion Jun 30, 2026→Jun 30, 2027
    Study completion Jun 30, 2027→Jun 30, 2028
    + 1 other change: verification date

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

09

Registry details

Key details

Study ID
NCT05104983
Lead sponsor
Darcy Krueger
Responsible party
Darcy Krueger (IND Sponsor/Lead Principal Investigator, Children's Hospital Medical Center, Cincinnati) — Sponsor-investigator
First posted
Nov 3, 2021
Start date
Oct 13, 2021
Primary completion
Jun 30, 2027 (estimated)
Completion
Jun 30, 2028 (estimated)
Last update
Oct 29, 2026

Study contacts

Darcy A Krueger, MD, PhD
principal investigator · Children's Hospital Medical Center, Cincinnati
Martina Bebin, MD, MPA
principal investigator · University of Alabama at Birmingham

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.

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Discussion

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