Evaluating Rapamycin for Drug Resistant Epilepsy in Tuberous Sclerosis Complex

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study aims to assess the safety, tolerability, and efficacy of rapamycin compared to a placebo in patients with drug-resistant epilepsy associated with tuberous sclerosis complex (TSC). It is a randomized, double-blind, placebo-controlled trial that includes three phases: screening, dose adjustment, and a core blinded phase, followed by an open-label observation. Eligible participants will be randomized in a 1:1 ratio to receive either rapamycin or placebo, with stratification based on age, sex, and prior antiepileptic drug use.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* male or female aged from 3 months up to 50 years at the day of randomization
* patients/parents/caregivers are willing to and able to give informed consent form for the participation in the study
* patients/parents/caregivers are willing to and able to comply with all study requirements
* definite diagnosis of TSC according to the Consensus criteria (Northrup, 2013)
* drug-resistant epilepsy associated with TSC with at least 8 seizures during 4 weeks

Exclusion Criteria:

* history of treatment with mTOR inhibitor in the three months prior to screening,
* history of pseudo-epileptic seizures,
* history of progressive CNS disease other than TSC
* recent surgery within 2 weeks prior to the screening
* severe infection within 2 weeks prior to the screening
* use of the cannabis derivatives
* contraindications for MRI or general anesthesia
* occurrence of the serious comorbidities which, in the opinion of the investigator, may either put a patient at significant risk associated with the participation in the study or may influence the results of the study the investigator
* pregnancy

Where it's running

• Children's Memorial Health Institute, Neurology and Epileptology
  Warsaw, Poland Recruiting
• Medical University of Lodz
  Lodz, Poland Not yet recruiting

Study contacts

• Study coordinator: Katarzyna Kotulska-Jozwiak
• Email: k.kotulska@ipczd.pl
• Phone: +48 22 8157404

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Smartphone app to help follow the 2024 KDIGO chronic kidney disease guidelines

  This project will test whether a smartphone app helps young adults (ages 16–29) with chronic kidney disease follow the 2024 KDIGO guideline recommendations.

  Recruiting at 2 sites St. James's Hospital, Ireland · Dublin and 1 other locations · NCT07561957

• Registry for pregnant women with Tuberous Sclerosis Complex and Lymphangioleiomyomatosis

  This study is collecting information from pregnant women with Tuberous Sclerosis Complex and Lymphangioleiomyomatosis to see how their health and their babies' health are affected during pregnancy.

  Recruiting Children's Hospital Medical Center, Cincinnati · Cincinnati, Ohio · NCT06160310

• Comparing Rapamycin and Vigabatrin for Preventing Symptoms of Tuberous Sclerosis in Infants

  This study is testing whether the medications rapamycin or vigabatrin can help prevent symptoms in infants with Tuberous Sclerosis.

  Recruiting Phase 2/3 Children's Memorial Health Institute, Poland · Warsaw and 1 other locations · NCT04987463

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.