CI

At a glance

ClinicalIndex Comparison Record
Phase 2Recruiting· 64 target
Drug / intervention
Sirolimus +1 moredrug
Likely dose
Not stated in record
Key inclusion· 2
  • Age 0-6 months at enrollment with corrected age ≥39 weeks
  • Confirmed diagnosis of TSC based on clinical or genetic criteria
Key exclusion· 9
  • Prior history of seizures (clinical or electrographic) or seizures on baseline EEG
  • Prior or current treatment with conventional AEDs, systemic mTOR inhibitors, ketogenic diet, or other anti-seizure therapeutic agent/device/procedure
  • Investigational drug use within 30 days prior to baseline
  • Significant illness or active infection at baseline

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT05104983
NCT05104983Phase 2RecruitingOn TrackUpdated 5mo ago
Long Recruiting

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

Darcy Krueger·interventional·Posted Nov 3, 2021·Updated Jan 22, 2026

In Brief

A Phase 2 clinical trial evaluating Sirolimus and Placebo for Tuberous Sclerosis Complex and Epilepsy. Currently recruiting, targeting 64 participants across 11 sites.

Detailed 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).

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
CountriesUnited States
Collaborators--

Timeline

Phase 2Recruiting
202220232024202520262027
First PostedNov 3, 2021
Enrollment StartOct 13, 2021
Primary CompletionJun 30, 2026
Study CompletionJun 30, 2027
TodayJul 2, 2026
Enrollment to primary: 4.7 yearsPosted 4.7 years ago

Interventions

Sirolimusdrug

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

Placebodrug

Matching placebo