Paroxysmal Nocturnal Hemoglobinuria, PNH
Conditions
Brief summary
A study of the efficacy and safety of VSA012 Injection compared to eculizumab for 24 weeks in patients with PNH.
Interventions
DRUGVSA012
VSA012 Injection
Eculizumab injection
Sponsors
Bisirna Therapeutics (Suzhou) Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
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
* Known or suspected hereditary or acquired complement deficiency; * Presence or suspicion of a systemic active bacterial, viral, or fungal infection * History of infection with capsular bacteria (e.g., meningococcus, pneumococcus, etc.) * Patients with reticulocytes \<100x10\^9/L; platelets \<30x10\^9/L; neutrophils \<0.5x10\^9/L. * Positive of HIV, HBsAg or HCVAb. * History of recurrent invasive infections caused by encapsulated organisms,e.g. meningococcus or pneumococcus. * Previous splenectomy. * A history of malignancy within 5 years before screening
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The proportion of patients with sustained hemoglobin levels ≥ 120 g/L among those without RBC transfusion (defined as no red blood cell infusion after W2 to W26). | between Week 2 and Week 26 |
Secondary
| Measure | Time frame |
|---|---|
| Proportion of participants achieving a sustained increase from baseline in hemoglobin levels of ≥ 20 g/L assessed among those without RBC transfusion (defined as no red blood cell infusion after W2 to W26). | between Week 20 and Week 26 |
| The proportion of patients with hemolysis controlled (defined as LDH < 1.5 ULN) among those without RBC transfusion | between Week 20 and Week 26 |
| Change (Expressed as Percentages) in Hb level from baseline | between Week 20 and Week 26 |
| The proportion of patients without RBC transfusion | between Week 2 and Week 26 |
| Change in reticulocyte count from baseline | between Week 20 and Week 26 |
| Change in Total bilirubin count from baseline | between Week 20 and Week 26 |
| Change in FACIT-F score from baseline | between Week 20 and Week 26 |
| The Clinical BTH Rate | between Day 1 and Week 26 |
| The Major Adverse Vascular Events Rate | between Day 1 and Week 26 |
| Incidence and severity of AEs | between Day 1 and Week 26 |
Countries
China
Contacts
CONTACTCharlene Si
Outcome results
None listed