MY008211A tablets: MY008211A tablets Participants will receive MY008211A at a dose of 400 mg orally b.i.d
Study summary
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.
Eligibility
Sex
ALL
Min age
18 Years
Max age
75 Years
Healthy volunteers
No
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. Stable regimen of anti-C5 antibody treatment for at least 6 months before treatment, and Hb was still \< 100 g/L.
3. The average hemoglobin level of at least two tests in 4 months before screening \< 100 g/L.
4. The average hemoglobin level of two tests in the central laboratory during screening \< 100 g/L.
5. 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.
Primary outcome measure(s)
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) — between Day 126 and Day 168 The proportion of patients with sustained hemoglobin levels ≥ 120 g/L among those without RBC transfusion.
Trial sites (1)
Facility
City
Region
Status
Peking Union Medical College Hospital (PUMCH).
Beijing
Beijing Municipality
Recruiting
More Wuhan Createrna Science and Technology Co., Ltd trials in China
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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