Меню
Идёт набор NCT06772766

A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia

Фаза I С лечением Beta-Thalassemia

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: 9MW3011, 9MW3011 placebo.
Кому может быть актуально
Состояния в реестре: Beta-Thalassemia. Базовые параметры: 18 лет — 65 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
Китай
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 Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of 9MW3011 in Patients With Non-transfusion-dependent β- Thalassemia

Обзор

This is a phase Ib, randomized, double-blind, placebo-controlled, multiple ascending dose study . The objectives of the study are to evaluate the safety , tolerability, pharmacokinetics(PK), pharmacodynamics(PD), and immunogenicity of 9MW3011 in patients with non-transfusion-dependent β- thalassemia .

Подробное описание

A total of 40 subjects diagnosed with non-transfusion-dependent β-thalassemia will be enrolled in this study and assigned into four dosage cohorts. In each cohort, subjects will be randomized in a 4:1 ratio to receive 9MW3011 or placebo via intravenous infusion.

Вмешательства

  • Препарат 9MW3011
    Ascending IV doses administered per protocol
  • Препарат 9MW3011 placebo
    Ascending IV doses administered per protocol

Первичные конечные точки

  • Adverse Event(including serious adverse event) [Срок оценки: up to day 169]
  • Number of subjects with abnormal vital signs [Срок оценки: up to day 169]
  • Number of subjects with abnormal clinically significant results from physical examination [Срок оценки: up to day 169]
  • Number of subjects with abnormal clinically significant 12-lead electrocardiogram (ECG) parameters [Срок оценки: up to day 169]
  • Number of subjects with abnormal clinically significant clinical laboratory results [Срок оценки: up to day 169]
Вторичные конечные точки (5)
  • Concentration of 9MW3011 in serum [Срок оценки: up to day 169]
  • PD-parameters-hepcidin [Срок оценки: up to day 169]
  • PD-parameters-serum iron [Срок оценки: up to day 169]
  • Anti-drug antibody(ADA) [Срок оценки: up to day 169]
  • Liver iron concentration(LIC) [Срок оценки: up to day 169]

Критерии участия

Критерии включения

  • Male and female subjects aged 18 to 65 years (inclusive)
  • Subject must have a documented genetic diagnosis of β-thalassemia or hemoglobin E/ β-thalassemia
  • Subjects must meet the criteria for non-transfusion-dependent thalassemia
  • Subjects must have a baseline hemoglobin level between 70-100 g/L(inclusive), based on 2 consecutive measurements taken at least 1 week apart within 4 weeks before randomization
  • Subjects must have evidence of iron overload during screening
  • Subject must have performance status: Eastern Cooperative Oncology Group (ECOG) performance score of 0 to 1
  • Subjects must fully understand the study procedures and methods, voluntarily participate in the trial, and sign an informed consent form

Критерии исключения

  • Subjects diagnosed with alpha-thalassemia
  • Subjects diagnosed with HbS/beta-thalassemia or transfusion-dependent beta-thalassemia
  • Subjects exhibit severe iron overload at the time of screening
  • In addition to thalassemia, subjects have any other forms of anemia and hematological disorders that the investigator assesses may compromise safety or influence study outcomes
  • Combined with any significant systemic diseases or psychiatric disorders
  • Subjects have New York Heart Association (NYHA) Class III-IV heart failure and other cardiovascular diseases within 6 months prior to screening or currently present
  • During the screening or baseline period, subjects exhibiting a QTcF interval of ≥450ms for males and ≥470ms for females on a 12-lead electrocardiogram (ECG), or presenting an abnormal 12-lead ECG with clinical significance
  • Uncontrolled hypertension before screening
  • A history of malignant neoplasm occurring within the last five years
  • Severe infection requiring hospitalization or intravenous antimicrobial therapy, or uncontrolled systemic bacterial, fungal, or viral active infection
  • Subject have received concomitant treatment that was not permitted by the protocol
  • Subjects whose hematological parameters did not meet the inclusion criteria during screening
  • Subjects with a history of substance abuse, as well as those who yield positive results on substance abuse screening
  • Subjects who are unable to undergo MRI scans
  • Pregnant or lactating women
  • Subjects presenting any other factors deemed unsuitable for participation assessed by the investigator

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
Двойное слепое
Основная цель
Лечение

Центры проведения

Китай · 2 центра
  • The first Affiliated Hospital of Guangxi Medical University — Nanning
  • Hainan General Hospital — Haikou

Идентификаторы

NCT: NCT06772766 · 9MW3011-C04

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗