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

Study Purpose

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).

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	3 Months - 50 Years
Gender	All

More Inclusion & Exclusion Criteria

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

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT05534672
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 3
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	Katarzyna Kotulska
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	N/A
Principal Investigator Affiliation	N/A
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Other
Overall Status	Recruiting
Countries	Poland
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Tuberous Sclerosis Complex

Additional Details

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).

Arms & Interventions

Arms

Experimental: Rapamycin arm

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

Placebo Comparator: Placebo arm

The patients assigned to the placebo arm will receive placebo in liquid, analogically to the rapamycin group.

Interventions

Other: - Rapamycin

Rapamycin in liquid administered orally

Other: - Placebo

Placebo in liquid administered orally

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

International Sites

Medical University of Lodz, Lodz, Poland

Status

Not yet recruiting

Address

Medical University of Lodz

Lodz, , 90-419

Site Contact

Wojciech Mlynarski

[email protected]

+48 42 617-77-91

Warsaw, Poland

Status

Recruiting

Address

Children's Memorial Health Institute, Neurology and Epileptology

Warsaw, , 04-730

Site Contact

Katarzyna Kotulska-Jozwiak

[email protected]

+48 22 8157404

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