Меню
Идёт набор NCT05432466

Clinical Trial to Compare the Efficacy of Celiprolol to Placebo in Patients With Vascular Ehlers-Danlos Syndrome

Фаза III С лечением Vascular Ehlers-Danlos Syndrome

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: ACER-002 (celiprolol) 200 mg BID, Placebo BID.
Кому может быть актуально
Состояния в реестре: Vascular Ehlers-Danlos Syndrome. Базовые параметры: 15 лет — 64 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
США
Следующий шаг
Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

Не всё понятно в терминах? Прочитайте наш гид для пациентов →

Официальное название

A Phase 3 Randomized, Double-Blind, Decentralized Clinical Trial to Compare the Efficacy of Celiprolol to Placebo in the Treatment of Patients With COL3A1-Positive Vascular Ehlers-Danlos Syndrome

Обзор

This is a prospective, Phase 3, randomized, double-blind, placebo-controlled efficacy study to evaluate celiprolol in patients genetically confirmed as COL3A1-positive vEDS using a decentralized clinical trial design.

Подробное описание

This is a prospective, Phase 3, randomized, double-blind, placebo-controlled efficacy study to evaluate celiprolol in patients genetically confirmed as COL3A1-positive vEDS using a decentralized clinical trial design.

The double-blind portion of this study is intended to end if statistical significance is reached at the interim analysis (accrual of 28 vEDS-related events requiring medical attention; estimated to take 24 months) or after accrual of 46 vEDS related clinical events requiring medical attention (estimated to take 40 months).

A total of approximately 150 patients who meet all the inclusion and none of the exclusion criteria will be enrolled and randomized 2:1 to receive either celiprolol or placebo, respectively.

Following the double-blind treatment period or occurrence of vEDS-related clinical event, patients have the option to participate in an open label extension period.

Вмешательства

  • Препарат ACER-002 (celiprolol) 200 mg BID
    ACER-002 (celiprolol) 200 mg BID
  • Препарат Placebo BID
    placebo for ACER-002

Первичные конечные точки

  • Time to first occurrence of a vEDS-related clinical event requiring medical attention: Fatal/nonfatal cardiac or arterial events [including dissection or rupture], uterine rupture, intestinal rupture, and/or unexplained sudden death [Срок оценки: Over the double-blind period (estimated to be 40 months)]
Вторичные конечные точки (5)
  • Number and proportion of patients reporting a vEDS related clinical event requiring medical attention: Fatal/nonfatal cardiac or arterial events [including dissection or rupture], uterine rupture, intestinal rupture, and/or unexplained sudden death [Срок оценки: Over the double-blind period (estimated to be 40 months)]
  • Number and percentage of patients with adverse events [Срок оценки: Over the double-blind period (estimated to be 40 months)]
  • Number and percentage of Serious Adverse Events (SAE) [Срок оценки: Over the double-blind period (estimated to be 40 months)]
  • Number and percentage of patient deaths [Срок оценки: Over the double-blind period (estimated to be 40 months)]
  • Number and percentage of patient discontinuations [Срок оценки: Over the double-blind period (estimated to be 40 months)]

Критерии участия

Критерии включения

  • Willingness to obtain magnetic resonance angiogram (MRA) image at local imaging facility.
  • A genetic test confirming the presence of a pathogenic COL3A1 variant (classified as likely pathogenic or pathogenic according to ACMG/AMP Guidelines.
  • Patients must be ≥ 15 years of age at the time of randomization.
  • Able and willing to discontinue use of β-blockers prior to randomization.

Критерии исключения

  • Lack of a COL3A1-positive test at screening (e.g., COL3A1 benign, likely benign, variant of unknown significance \[VUS\] or no variant) or presence of a COL3A1 variant but demonstration of a COL3A1 variant reported to be a haploinsufficiency variant.
  • Arterial rupture or dissection, uterine rupture, and/or intestinal rupture within 6 months prior to Screening.
  • Patients unable to discontinue β-blocker treatment prior to randomization.
  • Unable or unwilling to complete the study procedures.
  • Breastfeeding, pregnancy, or planned pregnancy during the trial.
  • Any medical condition that in the opinion of the Investigator may pose a safety risk to the patient in this study, which may confound efficacy or safety assessment, or may interfere with study participation.
  • Use of any prohibited medications

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
Четверное слепое
Основная цель
Лечение

Центры проведения

США · 1 центр
  • Science 37 — Culver City

Идентификаторы

NCT: NCT05432466 · ACER-002-301

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗