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Идёт набор NCT07187401

A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor

Фаза I / Фаза II С лечением Paroxysmal Nocturnal Hemoglobinuria (PNH) Persistent Anemia

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: ALN-CFB, Placebo.
Кому может быть актуально
Состояния в реестре: Paroxysmal Nocturnal Hemoglobinuria (PNH), Persistent Anemia. Базовые параметры: от 18 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
Канада, South Korea, Великобритания
Следующий шаг
Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Официальное название

A Randomized, Double-Blind, Placebo-Controlled, First-In-Human Study of the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of ALN-CFB, an Investigational siRNA Therapeutic Against Complement Factor B, in Participants With Paroxysmal Nocturnal Hemoglobinuria and Persistent Anemia on Approved C5-Inhibitor Therapy

Обзор

This study is researching an experimental drug called ALN-CFB. The study is focused on people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are currently taking a complement component C5 inhibitor ("C5-inhibitor") and continue to have anemia (low red blood cell count). The aim of the study is to see how tolerable ALN-CFB is compared to placebo. A placebo looks like the study drug but does not contain any drug. The study is looking at several other research questions, including: * What side effects may happen from taking ALN-CFB * How much ALN-CFB is in the blood at different times * How much Complement Factor B (CFB) protein levels in the blood are affected by ALN-CFB

Подробное описание

The protocol will be amended to describe Part B of the study after Part A data have been analyzed.

Вмешательства

  • Препарат ALN-CFB
    Administered as defined in the protocol
  • Препарат Placebo
    Administered as defined in the protocol

Первичные конечные точки

  • Occurrence of Treatment-Emergent Adverse Events (TEAEs) [Срок оценки: Through 365 Days]
  • Severity of TEAEs [Срок оценки: Through 365 Days]
Вторичные конечные точки (4)
  • Concentrations of combined ALN-CFB and major metabolites in plasma [Срок оценки: Through 365 Days]
  • Concentrations of combined ALN-CFB and major metabolites in urine [Срок оценки: Through 24 Hours following ALN-CFB administration]
  • Absolute change from baseline in CFB concentration [Срок оценки: Baseline, Through 365 Days]
  • Percentage change from baseline in CFB concentration [Срок оценки: Baseline, Through 365 Days]

Критерии участия

Критерии включения

  • Has been diagnosed with PNH confirmed by a history of high flow cytometry from prior testing
  • Treated with a stable dose of C5 inhibitor (eculizumab or approved eculizumab biosimilar, ravulizumab, or crovalimab) for at least 24 weeks prior to screening visit, as described in the protocol
  • Has hemoglobin ≤10.5 g/dL at screening visit 1, with evidence of anemia prior to this visit, as described in the protocol
  • Has peripheral blood reticulocyte count of ≥100 x 10\^9/L at screening visit 1

Критерии исключения

  • Has history of bone marrow transplantation or receipt of an organ transplant
  • Has history of meningococcal infection or similar recurrent infections by other encapsulated bacterial organisms
  • Has any active, ongoing infection or a recent infection requiring ongoing systemic treatment with antibiotics, antivirals, or antifungals within 2 weeks of screening or during the screening period
  • Has laboratory evidence of bone marrow failure, as described in the protocol
  • Have recent, unstable medical conditions, not related to PNH or PNH-related complications, as described in the protocol

NOTE: Other Protocol Defined Inclusion / Exclusion Criteria Apply

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение
Рандомизированное
Модель
Последовательный дизайн
Маскирование
Четверное слепое
Основная цель
Лечение

Центры проведения

South Korea · 5 центров
  • St. Vincent Hospital - The Catholic University of Korea — Suwon
  • The Catholic University of Korea, Seoul St. Mary's Hospital — Seoul
  • Seoul National University Hospital — Seoul
  • Severance Hospital — Seoul
  • Samsung Medical Center — Seoul
Великобритания · 2 центра
  • St. James's University Hospital — Leeds
  • Kings College Hospital NHS Foundation Trust — London
Канада · 1 центр
  • Toronto General Hospital — Toronto

Идентификаторы

NCT: NCT07187401 · ALN-CFB-PNH-2468 · 2024-519806-11-00

Первоисточники (государственные реестры)

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