Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment

Официальное название

A Multicenter, Single-arm, Open-label Phase III Study to Evaluate Efficacy and Safety of MY008211A Tablets in Patients With PNH and Residual Anemia, Despite Treatment With Anti-C5 Antibody.

Обзор

The main purpose of this study is to evaluate the efficacy of MY008211A in PNH patients with residual anemia despite treatment with anti-C5 antibody.

Подробное описание

This is a multicenter, single-arm, open-label phase III clinical trial. The purpose of this study is to determine whether MY008211A is efficacious and safe for the treatment of PNH patients Who Are Still Anemia After Anti-C5 Antibody Treatment.

Вмешательства

• Препарат MY008211A tablets
  MY008211A tablets Participants will receive MY008211A at a dose of 400 mg orally b.i.d

Первичные конечные точки

• The proportion of Participants With Sustained Hemoglobin Levels of ≥ 120 g/L in the Absence of Red Blood Cell Transfusions (defined as no red blood cell infusion after D14 to D168) [Срок оценки: between Day 126 and Day 168]

Вторичные конечные точки (9)

• The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion (defined as no red blood cell infusion after D14 to D168) [Срок оценки: between Day 126 and Day 168]
• Change From Baseline in Hemoglobin [Срок оценки: between Day 126 and Day 168]
• The proportion of patients with LDH < 1.5 ULN among those without RBC transfusion. [Срок оценки: between Day 126 and Day 168]
• Change (Expressed as Percentages) in LDH level from baseline [Срок оценки: between Day 126 and Day 168]
• Change in reticulocyte count from baseline [Срок оценки: between Day 126 and Day 168]
• The proportion of patients without RBC transfusion [Срок оценки: between Day 14 and Day 168]
• Change in FACIT-F score from baseline [Срок оценки: between Day 126 and Day 168]
• The Clinical BTH Rate [Срок оценки: between Day 1 and Day 168]
• The Major Adverse Vascular Events Rate [Срок оценки: between Day 1 and Day 168]

Критерии участия

Критерии включения

• Male and female participants ≥ 18 years of age and BMI ≥ 18.0 kg/m2 with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with clone size ≥ 10%.
• Stable regimen of anti-C5 antibody treatment for at least 6 months before treatment, and Hb was still < 100 g/L.
• The average hemoglobin level of at least two tests in 4 months before screening < 100 g/L.
• The average hemoglobin level of two tests in the central laboratory during screening < 100 g/L.
• Vaccination against Neisseria meningitidis infection is required prior to the start of study treatment. If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given.

Критерии исключения

• Patients with reticulocytes <100x10\^9/L; platelets <30x10\^9/L; neutrophils <0.5x10\^9/L.
• History of recurrent invasive infections caused by encapsulated organisms,e.g. meningococcus or pneumococcus.
• Known or suspected hereditary complement deficiency.
• Previous bone marrow or hematopoietic stem cell transplantation.
• Previous splenectomy.
• A history of malignancy within 5 years before screening, except cured local basal cell carcinoma of the skin and carcinoma in situ of the cervix.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Peking Union Medical College Hospital (PUMCH). — Пекин

Идентификаторы

NCT: NCT06932471 · MY008211A-PNH-3-01

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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