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

Official title

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.

Overview

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.

Detailed description

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.

Interventions

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

Primary outcome measures

• 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) [Time frame: between Day 126 and Day 168]

Secondary outcome measures (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) [Time frame: between Day 126 and Day 168]
• Change From Baseline in Hemoglobin [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%.
• 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.

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

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

China · 1 center

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

Identifiers

NCT: NCT06932471 · MY008211A-PNH-3-01

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗