Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy

Phase 3RecruitingNCT06932744

Wuhan Createrna Science and Technology Co., Ltd66 enrolled

Overview

The main purpose of this study is to evaluate the efficacy of MY008211A compared to eculizumab in PNH patients.

This is a multi-center, randomized, open-label, active comparator-controlled, parallel group study. The purpose of this study is to determine whether MY008211A compared to eculizumab is efficacious and safe for the treatment of PNH patients who were naive to complement inhibitor therapy.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Paroxysmal Nocturnal Haemoglobinuria (PNH)

Interventions

MY008211A tabletsDRUG

Participants will receive MY008211A at a dose of 400 mg orally b.i.d for 24 weeks

Eculizumab InjectionDRUG

Eculizumab Injection for 24 weeks

Eligibility

Sex: ALLMin age: 18 YearsMax age: 75 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. 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%. 2. Mean hemoglobin level \<100 g/L at screening. 3. LDH \> 1.5 x Upper Limit of Normal (ULN) at screening. 4. 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. Exclusion Criteria: 1. Patients with reticulocytes \<100x10\^9/L; platelets \<30x10\^9/L; neutrophils \<0.5x10\^9/L. 2. History of recurrent invasive infections caused by encapsulated organisms,e.g. meningococcus or pneumococcus. 3. Known or suspected hereditary complement deficiency. 4. Previous bone marrow or hematopoietic stem cell transplantation. 5. Previous splenectomy. 6. 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.

Locations (2)

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences

Beijing, Beijing Municipality, China

NOT_YET_RECRUITING

Chinese Academy of Medical Sciences Hematology Hospital

Tianjin, Tianjin Municipality, China

RECRUITING

Outcomes

Primary Outcomes

The proportion of Participants With Sustained Hemoglobin Levels of ≥ 120 g/L in the Absence of Red Blood Cell Transfusions

The proportion of patients with sustained hemoglobin levels ≥ 120 g/L among those without RBC transfusion (defined as no red blood cell infusion after D14 to D168).