A Multicenter, Randomized, Open-Label, Positive Controlled Phase III Study to Evaluate the Efficacy and Safety of VSA012 Injection in Participants With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy

A study of the efficacy and safety of VSA012 Injection compared to eculizumab for 24 weeks in patients with PNH.

Study Overview

• Status: Not yet recruiting
• Conditions: Paroxysmal Nocturnal Hemoglobinuria, PNH
• Intervention / Treatment: Drug: VSA012; Drug: Eculizumab (Soliris®)

• Study Type: Interventional
• Enrollment (Estimated): 70
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Charlene Si; Phone Number: +86-021-6107 5030; Email: charlene.si@bisirna.com

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China
      • Peking Union Medical College Hospital
      • Contact: Bing Han; Phone Number: 010-69151188; Email: Hanbing-li@sina.com.cn
  • Changchun
    • Jilin City, Changchun, China
      • Jilin
  • Fujian
    • Quanzhou, Fujian, China
      • Quanzhou
  • Guangdong
    • Guangzhou, Guangdong, China
      • Guangzhou
  • Guangxi
    • Nanning, Guangxi, China
      • Nanning
  • Heilongjiang
    • Harbin, Heilongjiang, China
      • Ha'erbin
  • Henan
    • Zhengzhou, Henan, China
      • Zhengzhou
  • Hubei
    • Wuhan, Hubei, China
      • Wuhan
  • Hunan
    • Changsha, Hunan, China
      • Changsha
  • Jiangsu
    • Nantong, Jiangsu, China
      • Nantong
  • Shanxi
    • Xi’an, Shanxi, China
      • Xi'an
  • Sichuan
    • Chengdu, Sichuan, China
      • Chengdu
  • Tianjin Municipality
    • Tianjin, Tianjin Municipality, China
      • Institute of Hematology, Chinese Academy of Medical Sciences
  • Zhejiang
    • Hangzhou, Zhejiang, China
      • The First Affiliated Hospital of Zhejiang University school of medicine
      • Contact: Hongyan Tong; Phone Number: 0571-87236114; Email: hongyantong@aliyun.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort1	Drug: VSA012; VSA012 Injection
Active Comparator: Cohort2	Drug: Eculizumab (Soliris®); Eculizumab injection

What is the study measuring?

Primary Outcome Measures

Outcome 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 Outcome Measures

Outcome 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

Other Outcome Measures

Outcome Measure	Time Frame
Incidence and severity of AEs	between Day 1 and Week 26

Collaborators and Investigators

• Sponsor: Bisirna Therapeutics (Suzhou) Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Estimated): October 29, 2026
• Primary Completion (Estimated): September 30, 2027
• Study Completion (Estimated): December 31, 2029

Study Registration Dates

• First Submitted: September 4, 2026
• First Submitted That Met QC Criteria: September 9, 2026
• First Posted (Actual): September 11, 2026

Study Record Updates

• Last Update Posted (Actual): September 11, 2026
• Last Update Submitted That Met QC Criteria: September 9, 2026
• Last Verified: September 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Anemia, Hemolytic; Anemia; Myelodysplastic Syndromes; Hemic and Lymphatic Diseases; Hemoglobinuria, Paroxysmal; eculizumab
• Other Study ID Numbers: VSA012-3001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No