A Phase 3 interventional study of Rapamycin and Placebo in Tuberous Sclerosis Complex, sponsored by Katarzyna Kotulska. Recruiting at 2 sites in Poland. Open to participants aged 3 Months to 50 Years. Per ClinicalTrials.gov, last updated 2026-10-22.
Sponsored by Katarzyna Kotulska · Phase 3, Interventional, and Treatment
The purpose of the RaRE-TS study is to determine safety, tolerability and efficacy of rapamycin versus placebo in a drug resistant epilepsy associated with tuberous sclerosis complex (TSC).
This is a two-arm, randomized, double-blind, placebo controlled study to evaluate the efficacy, tolerability, and safety of rapamycin versus placebo in a drug resistant epilepsy associated with TSC. The study consists of 3 phases for each patient: screening, dose adjustment blinded phase, core blinded phase, followed by open-label observation. Patients who meet the eligibility criteria will be randomized to receive rapamycin or placebo. The randomization ratio is 1:1. Randomization will be stratified by age, sex and and the number of antiepileptic drugs ever used in the patient's history (up to 3 drugs / more than 3 drugs).
115 studies on the registry are indexed under Tuberous Sclerosis; 27 are open to participants now.
This study's planned enrollment of 200 is above the median of 52 across 73 interventional studies indexed under Tuberous Sclerosis.
Browse Tuberous Sclerosis studies →Katarzyna Kotulska is the lead sponsor of 2 studies on the registry; 2 are open to participants now.
Counted across the registry records on this site, refreshed daily.
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.
Exclusion Criteria:
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.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Each patient randomized to the rapamycin arm will receive rapamycin in liquid. The rapamycin will be administered in individually calculated doses depending on the body surface of participants
Other: Rapamycin
The patients assigned to the placebo arm will receive placebo in liquid, analogically to the rapamycin group.
Other: Placebo
Rapamycin in liquid administered orally
Placebo in liquid administered orally
comparison of the number of patients with at least 50% reduction of seizures per week in the last month of the core blinded phase in comparison to screening phase in the rapamycin vs placebo group
Time frame: final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
number of adverse events (according to CTCAE classification) in the rapamycin vs placebo group during the double-blind core phase
Time frame: final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
comparison of the number of seizures per week and the number of days free of seizures in the rapamycin vs placebo group, during 12-week treatment in double-blind core phase
Time frame: final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
severity of adverse events (according to CTCAE) and the number of patients withdrawn from the study due to adverse events in the rapamycin vs placebo group
Time frame: final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
Started Jan 2023 (actual). Primary completion is estimated for Sep 2029, 80 months after the start.
Since we started tracking on Sep 25, 2026, the dates moved once, on Oct 22, 2026: primary completion Jun 2027 → Sep 2029 and completion Jun 2027 → Sep 2029.
Earlier changes are on the record history on ClinicalTrials.gov ↗
How these figures are worked out, and which studies are left out of them.
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 ↗
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Katarzyna Kotulska