A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Interventions
- Drug: MY008211A tablets
- Registration Number
- NCT06050226
- Lead Sponsor
- Wuhan Createrna Science and Technology Co., Ltd
- Brief Summary
The main purpose of this study is to evaluate the efficacy of MY008211A in adult patients with PNH , showing signs of active hemolysis, in China.
- Detailed Description
The purpose of this study is to determine whether MY008211A is efficacious and safe for the treatment of PNH patients who are naive to complement inhibitor therapy, including anti-C5 antibody.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 40
- Male and female participants ≥ 18 years of age, BMI≥18 kg/m2，with a diagnosis of PNH confirmed by laboratory tests, according to the PNH diagnostic criteria in the Chinese Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 edition) , and flow cytometry with clone size ≥ 10%.
- Mean hemoglobin level <100 g/L.
- LDH > 1.5 x Upper Limit of Normal (ULN)
- 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.
- Patients with reticulocytes <100x10^9/L; platelets <30x10^9/L; neutrophils <0.5x10^9/L.
- Were using a complement inhibitor before the first administration of MY008211A tablets or had discontinued a previous complement inhibitor for less than five half-lives or 120 days, whichever was the longest.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Arm1：low MY008211A dose MY008211A tablets Participants will receive low MY008211A dose orally b.i.d Arm2：high MY008211A dose MY008211A tablets Participants will receive high MY008211A dose orally b.i.d
- Primary Outcome Measures
Name Time Method Proportion of participants achieving a sustained increase in hemoglobin levels of ≥ 20 g/L in the absence of red blood cell transfusion. up to 84 days Proportion of participants achieving a sustained increase from baseline in hemoglobin levels of ≥ 20 g/L assessed , in the absence of red blood cell transfusions
- Secondary Outcome Measures
Name Time Method Change from baseline in hemoglobin concentration. up to 84 days Change from baseline in hemoglobin concentration (g/L) in absence of red blood cell transfusion
Change from baseline in serum LDH levels. up to 84 days Change from baseline in serum LDH levels (U/L)
Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in the absence of red blood cell transfusions. up to 84 days Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in absence of red blood cell transfusion
Change from baseline in Reticulocyte count. up to 84 days Change from baseline in Reticulocyte count (×10\^9/L)
Changes from baseline in transfusion volume. up to 84 days The average number of red blood cells transfused per week
Change in the level of PNH red cell clones. up to 84 days Change from baseline in the level of PNH red cell clones.
Occurrences of AEs occurring between Day 1 and Day 84. up to 84 days Adverse Events (AEs)
Trial Locations
- Locations (1)
Nstitute of Hematology & Blood Disease Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
🇨🇳Tianjin, Tianjin, China