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

NCT07816237 | Not Yet Recruiting Phase 3

• 1 other identifier: VSA012-3001
• Study Type: interventional
• Target: 70
• Locations: 1 country
• Sites: 14

Brief Summary

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

Conditions

Paroxysmal Nocturnal Hemoglobinuria, PNH

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (10)

• 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

Other Outcomes (1)

• Incidence and severity of AEs

  between Day 1 and Week 26

Study Arms (2)

Cohort1

EXPERIMENTAL

Drug: VSA012

Cohort2

ACTIVE COMPARATOR

Drug: Eculizumab (Soliris®)

Interventions

VSA012 DRUG

VSA012 Injection

Cohort1

Eculizumab (Soliris®) DRUG

Eculizumab injection

Cohort2

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

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

Study Sites (14)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

Location

Jilin

Jilin City, Changchun, China

Location

Quanzhou

Quanzhou, Fujian, China

Location

Guangzhou

Guangzhou, Guangdong, China

Location

Nanning

Nanning, Guangxi, China

Location

Ha'erbin

Harbin, Heilongjiang, China

Location

Zhengzhou

Zhengzhou, Henan, China

Location

Wuhan

Wuhan, Hubei, China

Location

Changsha

Changsha, Hunan, China

Location

Nantong

Nantong, Jiangsu, China

Location

Xi'an

Xi’an, Shanxi, China

Location

Chengdu

Chengdu, Sichuan, China

Location

Institute of Hematology, Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, China

Location

The First Affiliated Hospital of Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Interventions

eculizumab

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Central Study Contacts

Charlene Si

CONTACT

+86-021-6107 5030; charlene.si@bisirna.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 4, 2026
• First Posted: September 11, 2026
• Study Start (Estimated): October 29, 2026
• Primary Completion (Estimated): September 30, 2027
• Study Completion (Estimated): December 31, 2029
• Last Updated: September 11, 2026

Record last verified: 2026-09

Locations

CN (14)