ClinicalTrials.gov record NCT07816237

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 16, 2026

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

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
Age18 years old to 75 years old
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is a randomized, open-label Phase III study comparing VSA012 injection with eculizumab (Soliris®) for 24 weeks in people with paroxysmal nocturnal hemoglobinuria (PNH) who are naive to complement inhibitor therapy. Participants are assigned to one of two drug groups and followed with laboratory and clinical assessments through Week 26. The main outcome is the proportion achieving sustained hemoglobin ≥120 g/L without red blood cell transfusion between Week 2 and Week 26.

What it is studying. The study tests VSA012 injection compared with eculizumab to evaluate efficacy and safety in people with PNH who have not previously received complement inhibitors.

What the researchers hope to learn. Scientifically, the study aims to compare VSA012 and eculizumab on blood hemoglobin levels and transfusion avoidance, evaluate control of hemolysis (LDH), measure changes in reticulocyte and bilirubin levels, assess patient fatigue (FACIT-F), and record rates of breakthrough hemolysis, major adverse vascular events, and adverse events.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • VSA012
  • Eculizumab (Soliris®)

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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 description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants are randomly assigned to receive injections of either VSA012 or eculizumab and are followed through about Week 26. Vaccination against Neisseria meningitidis is required before starting study treatment; pneumococcal and Haemophilus influenzae vaccination should be given if not previously received. During the study participants will have blood tests to measure hemoglobin, LDH, reticulocyte count and bilirubin, complete a fatigue questionnaire (FACIT‑F), have clinic visits to monitor side effects, and have any red blood cell transfusions recorded. The registry does not describe invasive procedures such as biopsies or spinal taps.

How the study is structured. Participants are randomly assigned to one of two parallel groups: VSA012 or eculizumab (Soliris®). The trial is open-label (no masking) and conducted as a Phase III treatment study. Participants receive the assigned injections and are followed with blood tests and clinical assessments up to Week 26 to measure hemoglobin, markers of hemolysis, patient-reported fatigue (FACIT-F), transfusion requirements, vascular events, and adverse events.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on paroxysmal nocturnal hemoglobinuria

Clinical trials44 trials listed for paroxysmal nocturnal hemoglobinuriaEvery trial the registry lists for paroxysmal nocturnal hemoglobinuria, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in paroxysmal nocturnal hemoglobinuria researchLatest monthly update: September 2026. Includes: Treatment access. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Oct 29, 2026 (estimated)
Primary completion
Sep 30, 2027 (estimated)
Study completion
Dec 31, 2029 (estimated)
Target enrollment
70 participants (estimated)
Last updated on registry
Sep 11, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Bisirna Therapeutics (Suzhou) Co., Ltd.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (14)

  • Peking Union Medical College Hospital

    Beijing, Beijing Municipality · China

  • Jilin

    Jilin City, Changchun · China

  • Quanzhou

    Quanzhou, Fujian · China

  • Guangzhou

    Guangzhou, Guangdong · China

  • Nanning

    Nanning, Guangxi · China

  • Ha'erbin

    Harbin, Heilongjiang · China

  • Zhengzhou

    Zhengzhou, Henan · China

  • Wuhan

    Wuhan, Hubei · China

  • Changsha

    Changsha, Hunan · China

  • Nantong

    Nantong, Jiangsu · China

Terms used on this page · explanations by MedicalUpdates.ai

at screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
Upper Limit of Normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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