RecruitingPhase 2

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
1 Day to 6 Months
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests whether starting sirolimus early is safe and can prevent or delay seizures in babies with tuberous sclerosis complex (TSC). It is for babies ages 0–6 months with confirmed TSC who have not had seizures. Babies will receive either sirolimus or a placebo, a treatment with no active drug.

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).

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Age 0–6 months when joining, and younger than 7 months when assigned treatment and starting it.
  • Corrected age must be at least 39 weeks, using the study’s calculation to adjust for being born early.
  • TSC confirmed by medical signs or genetic testing.

Who can't join

  • Any past seizures, including seizures seen only on an EEG (a test of brain activity), or seizures found on the first EEG.
  • Past or current use of seizure medicines, oral mTOR drugs such as sirolimus or everolimus, a ketogenic-type diet, or another seizure treatment, device, or procedure.
  • Use of another study drug within 30 days before the first screening visit.
  • A serious illness or active infection at screening, or blood test results that the study doctor thinks could make treatment unsafe.
  • Born before 30 weeks of pregnancy, or serious problems at birth or soon after that add a risk of seizures or delayed development beyond the risk from TSC.
  • Past, planned, or expected brain or nerve surgery within 3 months of the first study visit.
  • A TSC-related condition that needs treatment with an mTOR drug.
  • The study doctor believes the baby cannot meet the study requirements.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

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: 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.

Study sites (11)

AL

  • University of Alabama at Birmingham · Birmingham, AL 35294Jessica Krefting, RN · Study contactE. Martina Bebin, MD, MPA · Principal investigatorMore trials in Birmingham

CA

CO

IL

MA

  • Boston Children's Hospital · Boston, MA 02115Sahana Raman · Study contactMustafa Sahin, MD, PhD · Principal investigatorMore trials in Boston

MO

  • Washington University -- St. Louis · St Louis, MO 63110Olga Novak · Study contactMichael Wong, MD, PhD · Principal investigatorMore trials in St Louis

NC

OH

TX

  • University of Texas HSC at Houston · Houston, TX 77030Mariam Lugo Noguera · Study contactHope Northrup, MD · Principal investigatorMore trials in Houston

WA

  • Seattle Children's Hospital · Seattle, WA 98105Mikaela Morales · Study contactStephanie Randle, MD · Principal investigatorMore trials in Seattle