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Prevention study for Tuberous Sclerosis Complex
Official title Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
ClinicalTrials.gov ID: NCT05104983
What this study is testing
What is Sirolimus?
Sirolimus is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for tuberous sclerosis complex.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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).
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 6
You may be able to join if
- 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)...
- Has a confirmed diagnosis of TSC based on established clinical or genetic criteria
You likely can't join 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...
- 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...
- Abnormal laboratory values at baseline (i.e., renal function, liver function, or bone marrow production) that are in the opinion of the investigator...
See the full eligibility 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...
- Has a confirmed diagnosis of TSC based on established clinical or genetic criteria
- 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)...
- 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...
- 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...
- 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).
- people who are, in the opinion of the investigator, unable to comply with the requirements of the study.
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- Los Angeles, California, United States
- Palo Alto, California, United States
- Aurora, Colorado, United States
- Chicago, Illinois, United States
- Boston, Massachusetts, United States
- St Louis, Missouri, United States
- Chapel Hill, North Carolina, United States
- Cincinnati, Ohio, United States
- Houston, Texas, United States
- Seattle, Washington, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 day to 6 months. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; Los Angeles, California, United States; Palo Alto, California, United States; Aurora, Colorado, United States; Chicago, Illinois, United States; Boston, Massachusetts, United States and 5 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.