ClinicalTrials.gov record NCT05534672

Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

Multicenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With TSC

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.The registry record was last updated on Aug 8, 2023, about 3 years ago, so the status shown may be out of date. The study team or the registry can confirm.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
Age3 months old to 50 years old
SexAll
LocationPoland
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 3, randomized, double-blind, placebo-controlled study compares oral rapamycin with matching placebo in people with tuberous sclerosis complex (TSC) and drug-resistant epilepsy. It evaluates seizure outcomes plus safety and tolerability. The study includes screening, a blinded dose-adjustment phase, a 12-week core blinded treatment phase, and an open-label observation phase.

What it is studying. The study is testing whether orally administered rapamycin (given in individually calculated doses) affects seizure frequency compared with placebo and measures safety and tolerability, including adverse events.

What the researchers hope to learn. To determine the safety, tolerability, and efficacy of rapamycin versus placebo in drug-resistant epilepsy associated with TSC; to compare the proportion of patients achieving at least a 50% seizure reduction; to compare seizure frequency, seizure-free days, and adverse event counts and severity.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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).

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Other
  • Rapamycin
  • Placebo

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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).

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

After screening, eligible participants are randomized 1:1 to receive either rapamycin liquid or matching placebo, taken by mouth with dose calculated for each person. The study includes a blinded dose-adjustment phase, a blinded core treatment phase, and then an open‑label observation phase. During the study participants will be monitored for seizure frequency and for adverse events (safety), and receive the study drug or placebo under double-blind conditions. The registry does not list any special invasive procedures (for example, spinal taps or biopsies); routine safety monitoring is performed as part of the trial.

How the study is structured. Participants are randomly assigned 1:1 to receive oral liquid rapamycin or matching placebo. The trial is double-blind (neither participants nor staff know who receives rapamycin) and uses parallel treatment arms. Randomization is stratified by age, sex, and number of antiepileptic drugs ever used. After screening there is a blinded dose-adjustment period, then a 12-week blinded core treatment phase, followed by open-label observation.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
  • The listed sites are in Poland. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on tuberous sclerosis complex

Clinical trials30 trials listed for tuberous sclerosis complexEvery trial the registry lists for tuberous sclerosis complex, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in tuberous sclerosis complex researchLatest monthly update: September 2026. Includes: CBD add-on therapy. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jan 23, 2023
Primary completion
June 2027 (estimated)
Study completion
June 2027 (estimated)
Target enrollment
200 participants (estimated)
Last updated on registry
Aug 8, 2023
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Katarzyna Kotulska

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (2)

Terms used on this page · explanations by MedicalUpdates.ai

placebo
A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
randomization
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
informed consent form
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
prior to screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
contraindications
A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
MRI
A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
double-blind
Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
placebo controlled
A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
blinded
Who does not know which intervention a participant is receiving. It can apply to participants, the people giving the treatment, those assessing results, or those analysing the data.
open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
eligibility criteria
The rules the study team uses to decide who may take part. They are usually split into inclusion criteria and exclusion criteria.
randomization ratio
How many participants go to each group. In a 2:1 ratio, two are assigned to the first group for every one assigned to the second.
stratified
Randomisation done separately within subgroups, such as age bands or disease severity, so that each group contains a similar mix.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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