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Идёт набор NCT05534672

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

Фаза III С лечением Tuberous Sclerosis Complex

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают
В протоколе указаны: Rapamycin, Placebo.
Кому может быть актуально
Состояния в реестре: Tuberous Sclerosis Complex. Базовые параметры: 3 мес. — 50 лет · Все.
Что важно проверить
Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится
Польша
Следующий шаг
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Официальное название

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

Обзор

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).

Подробное описание

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).

Вмешательства

  • Другое Rapamycin
    Rapamycin in liquid administered orally
  • Другое Placebo
    Placebo in liquid administered orally

Первичные конечные точки

  • 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 [Срок оценки: 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 [Срок оценки: final analyses after the formal final database lock, planned within one month after the last patient last visit in the study]
Вторичные конечные точки (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 [Срок оценки: 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 [Срок оценки: final analyses after the formal final database lock, planned within one month after the last patient last visit in the study]

Критерии участия

Критерии включения

  • 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

Критерии исключения

  • 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение
Рандомизированное
Модель
Параллельные группы
Маскирование
Двойное слепое
Основная цель
Лечение

Центры проведения

Польша · 2 центра
  • Medical University of Lodz — Lodz
  • Children's Memorial Health Institute, Neurology and Epileptology — Warsaw

Идентификаторы

NCT: NCT05534672 · RaRE-TS

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗