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

Official title

A Randomized, Multicenter, Active-comparator Controlled, Open-label Trial to Evaluate Efficacy and Safety of MY008211A Tablets in PNH Patients Who Are Naive to Complement Inhibitor Therapy

Overview

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

Detailed description

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.

Interventions

• Drug MY008211A tablets
  Participants will receive MY008211A at a dose of 400 mg orally b.i.d for 24 weeks
• Drug Eculizumab Injection
  Eculizumab Injection for 24 weeks

Primary outcome measures

• The proportion of Participants With Sustained Hemoglobin Levels of ≥ 120 g/L in the Absence of Red Blood Cell Transfusions [Time frame: between Day 126 and Day 168]

Secondary outcome measures (8)

• The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion [Time frame: between Day 126 and Day 168]
• The proportion of patients with LDH < 1.5 ULN among those without RBC transfusion. [Time frame: between Day 126 and Day 168]
• Change (Expressed as Percentages) in LDH level from baseline [Time frame: between Day 126 and Day 168]
• Change in reticulocyte count from baseline [Time frame: between Day 126 and Day 168]
• The proportion of patients without RBC transfusion [Time frame: between Day 14 and Day 168]
• Change in FACIT-F score from baseline [Time frame: between Day 126 and Day 168]
• The Clinical BTH Rate [Time frame: between Day 1 and Day 168]
• The Major Adverse Vascular Events Rate [Time frame: between Day 1 and Day 168]

Eligibility criteria

Inclusion criteria

• 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%.
• Mean hemoglobin level <100 g/L at screening.
• LDH > 1.5 x Upper Limit of Normal (ULN) at screening.
• 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

• 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Open label

Primary purpose

Treatment

Study locations

China · 2 centers

• Peking Union Medical College Hospital, Chinese Academy of Medical Sciences — Beijing
• Chinese Academy of Medical Sciences Hematology Hospital — Tianjin

Identifiers

NCT: NCT06932744 · MY008211A-PNH-3-02

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗