Tuberous Sclerosis ComplexPhase 3RaRE-TSNCT05534672
Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex
A Phase 3 study testing Rapamycin in people with Tuberous Sclerosis Complex, run by Katarzyna Kotulska at 2 sites in Poland.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 3 months to 50 years
- Conditions the study is for
- Tuberous Sclerosis Complex
- What is being tested?
- Rapamycin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 200 participants
- Where
- 2 sitesPoland
- Study dates
- 23 Jan 2023to Sep 2029
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 months to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Poland2 sites, 2 recruiting
- Not yet recruiting
Medical University of Lodz
Lodz, 90-419
Wojciech Mlynarski, +48 42 617-77-91, wojciech.mlynarski@umed.lodz.pl
- Recruiting
Children's Memorial Health Institute, Neurology and Epileptology
Warsaw, 04-730
Katarzyna Kotulska-Jozwiak, +48 22 8157404, k.kotulska@ipczd.pl
Monika Szkop, +48 22 815 74 04, m.szkop@ipczd.pl
About this study
Official title: Multicenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With TSC
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).
Read the detailed description
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).
- Conditions
- Tuberous Sclerosis Complex
- Being tested
- Rapamycin, Placebo
- Keywords
- tuberous sclerosis complex, epilepsy, rapamycin, drug resistant epilepsy
- Medical subject headings
- Tuberous Sclerosis, Epilepsy, Drug Resistant Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RapamycinOther
Rapamycin in liquid administered orally
PlaceboOther
Placebo in liquid administered orally
| Study group | Role | What they receive |
|---|---|---|
| Rapamycin arm | Experimental | 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 Receives: Other: Rapamycin |
| Placebo arm | Placebo comparator | The patients assigned to the placebo arm will receive placebo in liquid, analogically to the rapamycin group. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
Secondary outcomes (2)
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
Measured: 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
Measured: final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2022
- Study start23 Jan 2023
- Primary completionSep 2029 (estimated)
- Study completionSep 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Tuberous Sclerosis ComplexStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Katarzyna Kotulska
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Katarzyna Kotulska, Children's Memorial Health Institute, Poland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05534672
- Sponsor's protocol ID
- RaRE-TS
- Acronym
- RaRE-TS
- Status last verified
- Oct 2026
- First submitted
- 6 Sep 2022
- Last updated
- 22 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With TSC
- ClinicalTrials.gov ID
- NCT05534672
- Acronym / sponsor ID
- RaRE-TS / RaRE-TS
- Conditions
- Tuberous Sclerosis Complex
- Keywords
- tuberous sclerosis complex, epilepsy, rapamycin, drug resistant epilepsy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Katarzyna KotulskaUniversity, hospital or other
- Study start
- 23 Jan 2023 (actual)
- Primary completion
- Sep 2029 (estimated)
- Study completion
- Sep 2029 (estimated)
- First posted
- 9 Sep 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Rapamycin arm | Experimental | Other: RapamycinEach 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 |
| Placebo arm | Placebo comparator | Other: PlaceboThe patients assigned to the placebo arm will receive placebo in liquid, analogically to the rapamycin group. |
Who may qualify
- Ages
- 3 months to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RapamycinRapamycin in liquid administered orally | Other | Rapamycin arm |
| PlaceboPlacebo in liquid administered orally | Other | Placebo arm |
Primary outcome
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
2 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Oct 2026.
Related trials for Tuberous Sclerosis Complex
RecruitingObservational study
Study of the Disease Process of Lymphangioleiomyomatosis
Lung Disease, Pneumothorax
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
Active, not recruitingPhase 2
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Tuberous Sclerosis Complex, Epilepsy
RecruitingObservational study
Study of Skin Tumors in Tuberous Sclerosis
Tuberous Sclerosis
RecruitingPhase 2/3
Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants
Tuberous Sclerosis Complex
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Rapamycin in people with Tuberous Sclerosis Complex, run by Katarzyna Kotulska at 2 sites in Poland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 22 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Poland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Katarzyna Kotulska-Jozwiak (+48 22 8157404, k.kotulska@ipczd.pl), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Oct 2026.