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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

200 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-01-23

Study Completion Date

2027-06-30

Brief Summary

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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).

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Detailed Description

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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

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Tuberous Sclerosis Complex

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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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

Group Type EXPERIMENTAL

Rapamycin

Intervention Type OTHER

Rapamycin in liquid administered orally

Placebo arm

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

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Placebo in liquid administered orally

Interventions

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Rapamycin

Rapamycin in liquid administered orally

Intervention Type OTHER

Placebo

Placebo in liquid administered orally

Intervention Type OTHER

Eligibility Criteria

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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

Minimum Eligible Age

3 Months

Maximum Eligible Age

50 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No