The complement system is an important component of the innate immune system. Abnormal activation, inadequate regulation and control of the complement system, as well as impaired and dysfunctional effector functions, underlie complement mediated diseases including PNH. VSA012 targeting complement system has the potential to treat a variety of diseases associated with abnormal activation of the complement system.The purpose of VSA012-1002 is to evaluate the safety, tolerability, pharmacokinetic, pharmacodynamics and efficacy of VSA012 Injection in subjects with PNH.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
50
VSA012 injection
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China
The First Affiliated Hospital of Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
Number of Participants with Treatment-Emergent Adverse Events (AEs) and/or Serious Adverse Events (SAEs)
Time frame: up to Day 540
preliminary efficacy
Percentage change from baseline in lactate dehydrogenase (LDH) by visit Change from baseline in hemoglobin (Hb) level by visit
Time frame: up to Day 540
Pharmacokinetics (PK) of VSA012 (First dose): Maximum Observed Plasma Concentration (Cmax)
Time frame: Up to 48 hours post-dose
PK of VSA012(First dose): Time to Maximum Observed Plasma Concentration (Tmax)
Time frame: Up to 48 hours post-dose
PK of VSA012(First dose):Area under the concentration-time curve during the dosing interval (AUC 0-tau)
Time frame: Up to 48 hours post-dose
PK of VSA012 (Multiple dose):Trough concentration (C min)
Time frame: Up to 48 hours post-dose
PK of VSA012 (Multiple dose):Accumulation ratio of C max
Time frame: Up to 48 hours post-dose
PK of VSA012 (Multiple dose):AUC 0-tau
Time frame: Up to 48 hours post-dose
PK of VSA012 (Multiple dose):T max
Time frame: Up to 48 hours post-dose
PK of VSA012 (Multiple dose):C max (RacC max)
Time frame: Up to 48 hours post-dose
PK of VSA012 (Multiple dose):Accumulation ratio of AUC 0-tau (RacAUC 0-tau)
Time frame: Up to 48 hours post-dose
Pharmacodynamic (PD) profile of VSA012:Change from baseline in complement factor B (CFB) and complement bypass pathway (CAP) activities
Time frame: up to Day 540
PD of VSA012:Change from baseline in PNH clones, including number of PNH clones in erythrocytes, number of PNH clones in granulocytes, and number of PNH clones in monocytes
Time frame: up to Day 540
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