Paroxysmal Nocturnal Hemoglobinuria, PNHPhase 3NCT07816237
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 Phase 3 study testing VSA012 and Eculizumab (Soliris®) in people with Paroxysmal Nocturnal Hemoglobinuria, PNH, run by Bisirna Therapeutics (Suzhou) Co., Ltd. at 14 sites in China.
Trial summary
A study of the efficacy and safety of VSA012 Injection compared to eculizumab for 24 weeks in patients with PNH. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria, PNH
- What is being tested?
- VSA012 and Eculizumab (Soliris®)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 14 sitesChina
- Study dates
- 29 Oct 2026to 31 Dec 2029
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
14 sites in 1 country. Contact the study team or a site near you to ask about taking part.
14 sites in 1 country
China14 sites
Peking Union Medical College Hospital
Beijing, Beijing Municipality
Bing Han, 010-69151188, Hanbing-li@sina.com.cn
Changsha
Changsha, Hunan
Chengdu
Chengdu, Sichuan
Guangzhou
Guangzhou, Guangdong
The First Affiliated Hospital of Zhejiang University School of Medicine
Hangzhou, Zhejiang
Hongyan Tong, 0571-87236114, hongyantong@aliyun.com
Ha'erbin
Harbin, Heilongjiang
Jilin
Jilin City, Changchun
Nanning
Nanning, Guangxi
Nantong
Nantong, Jiangsu
Quanzhou
Quanzhou, Fujian
Institute of Hematology, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality
Wuhan
Wuhan, Hubei
Xi'an
Xi’an, Shanxi
Zhengzhou
Zhengzhou, Henan
About this study
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
- Being tested
- VSA012, Eculizumab (Soliris®)
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VSA012Drug
VSA012 Injection
Eculizumab (Soliris®)Drug
Eculizumab injection
| Study group | Role | What they receive |
|---|---|---|
| Cohort1 | Experimental | Not described Receives: Drug: VSA012 |
| Cohort2 | Active comparator | Not described Receives: Drug: Eculizumab (Soliris®) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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).
Measured: between Week 20 and Week 26
The proportion of patients with hemolysis controlled (defined as LDH < 1.5 ULN) among those without RBC transfusion
Measured: between Week 20 and Week 26
Change (Expressed as Percentages) in Hb level from baseline
Measured: between Week 20 and Week 26
The proportion of patients without RBC transfusion
Measured: between Week 2 and Week 26
Change in reticulocyte count from baseline
Measured: between Week 20 and Week 26
Change in Total bilirubin count from baseline
Measured: between Week 20 and Week 26
Change in FACIT-F score from baseline
Measured: between Week 20 and Week 26
The Clinical BTH Rate
Measured: between Day 1 and Week 26
The Major Adverse Vascular Events Rate
Measured: between Day 1 and Week 26
Incidence and severity of AEs
Measured: between Day 1 and Week 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Sep 2026
- Study start29 Oct 2026 (estimated)
- Primary completion30 Sep 2027 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal Hemoglobinuria, PNHStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Bisirna Therapeutics (Suzhou) Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07816237
- Sponsor's protocol ID
- VSA012-3001
- Status last verified
- Sep 2026
- First submitted
- 4 Sep 2026
- Last updated
- 11 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07816237
- Acronym / sponsor ID
- VSA012-3001
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, PNH
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- Bisirna Therapeutics (Suzhou) Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 29 Oct 2026 (estimated)
- Primary completion
- 30 Sep 2027 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 11 Sep 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A study of the efficacy and safety of VSA012 Injection compared to eculizumab for 24 weeks in patients with PNH.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort1 | Experimental | Drug: VSA012 |
| Cohort2 | Active comparator | Drug: Eculizumab (Soliris®) |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VSA012VSA012 Injection | Drug | Cohort1 |
| Eculizumab (Soliris®)Eculizumab injection | Drug | Cohort2 |
Primary outcome
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).
Time frame: between Week 2 and Week 26
10 secondary outcomes
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).
Time frame: between Week 20 and Week 26
The proportion of patients with hemolysis controlled (defined as LDH < 1.5 ULN) among those without RBC transfusion
Time frame: between Week 20 and Week 26
Change (Expressed as Percentages) in Hb level from baseline
Time frame: between Week 20 and Week 26
The proportion of patients without RBC transfusion
Time frame: between Week 2 and Week 26
Change in reticulocyte count from baseline
Time frame: between Week 20 and Week 26
Change in Total bilirubin count from baseline
Time frame: between Week 20 and Week 26
Change in FACIT-F score from baseline
Time frame: between Week 20 and Week 26
The Clinical BTH Rate
Time frame: between Day 1 and Week 26
The Major Adverse Vascular Events Rate
Time frame: between Day 1 and Week 26
Incidence and severity of AEs
Time frame: between Day 1 and Week 26
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
14 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing VSA012 and Eculizumab (Soliris®) in people with Paroxysmal Nocturnal Hemoglobinuria, PNH, run by Bisirna Therapeutics (Suzhou) Co., Ltd. at 14 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 11 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Charlene Si (+86-021-6107 5030, charlene.si@bisirna.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). (time frame: between Week 2 and Week 26). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Sep 2026.