A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
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.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Arm1：low MY008211A dose Participants will receive low MY008211A dose orally b.i.d |
Drug: MY008211A tablets
The first 10 participants will be received low-dose MY008211A tablets, and the next 30 participants will be randomized to low-dose or high-dose treatment arms in a 1:2 ratio.
Other Names:
|
| Experimental: Arm2：high MY008211A dose Participants will receive high MY008211A dose orally b.i.d |
Drug: MY008211A tablets
The first 10 participants will be received low-dose MY008211A tablets, and the next 30 participants will be randomized to low-dose or high-dose treatment arms in a 1:2 ratio.
Other Names:
|
Outcome Measures
Primary Outcome Measures
- 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
- 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 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)
- 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)
Other Outcome Measures
- Changes from baseline in alternative complement pathway activity. [up to 84 days]
Alternative complement pathway activity measured by the WIESLAB® kit.
- Change from baseline in plasma levels of the Bb fragment. [up to 84 days]
Bb fragment cleaved by factor B of complement.
- Maximum Plasma Concentration (Cmax) Of MY008211A tablets [up to 84 days]
PK parameters
- Area Under The Concentration Versus Time Curve (AUC) Of MY008211A [up to 84 days]
PK parameters
Eligibility Criteria
Criteria
Inclusion Criteria:
-
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%.
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Mean hemoglobin level <100 g/L.
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LDH > 1.5 x Upper Limit of Normal (ULN)
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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:
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Patients with reticulocytes <100x109/L; platelets <30x109/L; neutrophils <0.5x10^9/L.
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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.
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History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
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Known or suspected hereditary complement deficiency
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Previous bone marrow or hematopoietic stem cell transplantation.
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Previous splenectomy.
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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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Nstitute of Hematology & Blood Disease Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College | Tianjin | Tianjin | China |
Sponsors and Collaborators
- Wuhan Createrna Science and Technology Co., Ltd
Investigators
- Principal Investigator: Fengkui Zhang, Ph.D, Blood Disease Hospital, Chinese Academy of Medical Sciences
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- MY008211A-PNH-2-01