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Paroxysmal Nocturnal Hemoglobinuria (PNH)Phase 1VSA012-1002NCT06848296

Phase Ib Clinical Study to Evaluate the Safety and Tolerability of VSA012 Injection in Paroxysmal Nocturnal Hemoglobinuria

A Phase 1 study testing VSA012 in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Bisirna Therapeutics Pte. Ltd. at 2 sites in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 18 to 75 years
What is being tested?
VSA012
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
50 participants
Where
2 sitesChina
Study dates
15 Apr 2025to 31 Aug 2027
Phase
Phase 1
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

  • Participants voluntarily participate in this clinical study, and voluntarily sign the ICF;
  • BMI ≥ 18.0 kg/m2; male or female; 18 to 75 years of age;
  • Confirmed diagnosis of PNH by clinical manifestation and flow cytometry; granulocyte clone size ≥ 10%;
  • Presence of one or more of PNH-related signs or symptoms within 3 months prior to screening;
  • Hb < 100 g/L;
  • LDH value > 1.5 × ULN;
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  • One of the following criteria for prior drug therapy for PNH must be met:
  • Having never received any complement inhibitor therapy;
  • Having received C5, C3, or CFB complement inhibitors and having discontinued the complement inhibitor for more than 5 half-lives or 3 months prior to screening;
  • Participants are willing to receive meningococcal vaccine and pneumococcal vaccine at least 14 days prior to dosing.
  • Participants voluntarily participate in this clinical study, and voluntarily sign the ICF;
  • BMI ≥ 18.0 kg/m2; male or female; 18 to 75 years of age;
  • Confirmed diagnosis of PNH by clinical manifestation and flow cytometry; granulocyte clone size ≥ 10%;
  • Participants who have been on a stable dose and interval of a C5 complement inhibitor (approved locally) for at least 3 months prior to the first dose of VSA012;
  • Within the 3 months prior to screening, have a documented Hb level of < 105 g/L while on C5 complement inhibitor therapy;
  • Hb < 105 g/L;
  • Participants are willing to receive meningococcal vaccine and pneumococcal vaccine at least 14 days prior to dosing.

Who cannot join

  • History of hypersensitivity to VSA012 or its excipients;
  • Use of any complement inhibitors within 3 months prior to screening
  • Use of any targeted small interfering RNA (siRNA) within 18 months prior to screening, or any antisense oligonucleotide molecule within 6 months prior to screening;
  • Supportive care for PNH does not meet the stable-dose requirements:
  • Participants have infections;
  • Laboratory tests meet the following criteria:
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  • Reticulocyte count < 100 × 109/L;
  • Platelet count < 30 × 109/L;
  • Neutrophil count < 0.5 × 109/L;
  • Creatinine clearance < 30 mL/min (calculated by the Cockcroft-Gault formula);
  • History of hypersensitivity to VSA012 or its excipients;
  • Use of any targeted small interfering RNA (siRNA) within 18 months prior to screening, or any antisense oligonucleotide molecule within 6 months prior to screening;
  • Supportive care for PNH does not meet the stable-dose requirements:
  • Participants have infections;
  • History of splenectomy;
  • Previous suspected/confirmed hereditary complement deficiency;
  • History of recurrent invasive infections with capsular bacteria, e.g., meningococcus or pneumococcus;
  • Laboratory tests meet the following criteria:
  • Reticulocyte count < 100 × 109/L;
  • Platelet count < 30 × 109/L;
  • Neutrophil count < 0.5 × 109/L;
  • Creatinine clearance < 30 mL/min (calculated by the Cockcroft-Gault formula);

Trial locations and contacts

2 sites in 1 country.

China2 sites

  • Peking Union Medical College Hospital

    Beijing, Beijing Municipality

  • The First Affiliated Hospital of Zhejiang University School of Medicine

    Hangzhou, Zhejiang

Other trials in China

About this study

Official title: A Phase Ib Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VSA012 Injection in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Naïve or Have Not Received Complement Inhibitor Recently and Have Persistent Anemia Despite Previous Stable Use of C5 Complement Inhibitor

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.

Being tested
VSA012
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

Study groups in NCT06848296
Study groupRoleWhat they receive
VSA012 dose AExperimentalNot described
Receives: Drug: VSA012
VSA012 dose BExperimentalNot described
Receives: Drug: VSA012
VSA012 dose CExperimentalNot described
Receives: Drug: VSA012
VSA012 dose DExperimentalNot described
Receives: Drug: VSA012
VSA012 dose EExperimentalNot described
Receives: Drug: VSA012

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants with Treatment-Emergent Adverse Events (AEs) and/or Serious Adverse Events (SAEs)

    Measured: up to Day 540

  2. preliminary efficacy

    Percentage change from baseline in lactate dehydrogenase (LDH) by visit Change from baseline in hemoglobin (Hb) level by visit

    Measured: up to Day 540

Secondary outcomes (11)

  1. Pharmacokinetics (PK) of VSA012 (First dose): Maximum Observed Plasma Concentration (Cmax)

    Measured: Up to 48 hours post-dose

  2. PK of VSA012(First dose): Time to Maximum Observed Plasma Concentration (Tmax)

    Measured: Up to 48 hours post-dose

  3. PK of VSA012(First dose)：Area under the concentration-time curve during the dosing interval (AUC 0-tau)

    Measured: Up to 48 hours post-dose

  4. PK of VSA012 (Multiple dose)：Trough concentration (C min)

    Measured: Up to 48 hours post-dose

  5. PK of VSA012 (Multiple dose)：Accumulation ratio of C max

    Measured: Up to 48 hours post-dose

  6. PK of VSA012 (Multiple dose)：AUC 0-tau

    Measured: Up to 48 hours post-dose

  7. PK of VSA012 (Multiple dose)：T max

    Measured: Up to 48 hours post-dose

  8. PK of VSA012 (Multiple dose)：C max (RacC max)

    Measured: Up to 48 hours post-dose

  9. PK of VSA012 (Multiple dose)：Accumulation ratio of AUC 0-tau (RacAUC 0-tau)

    Measured: Up to 48 hours post-dose

  10. Pharmacodynamic (PD) profile of VSA012:Change from baseline in complement factor B (CFB) and complement bypass pathway (CAP) activities

    Measured: up to Day 540

  11. 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

    Measured: up to Day 540

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Feb 2025
  2. Study start15 Apr 2025
  3. Primary completion28 Feb 2027 (estimated)
  4. Study completion31 Aug 2027 (estimated)

Resources

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 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
50 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06848296
Sponsor's protocol ID
VSA012-1002
Acronym
VSA012-1002
Status last verified
Apr 2026
First submitted
17 Feb 2025
Last updated
4 May 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing VSA012 in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Bisirna Therapeutics Pte. Ltd. at 2 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 17 inclusion and 22 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: under way, not looking for participants, as of the sponsor's last update on 4 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants with Treatment-Emergent Adverse Events (AEs) and/or Serious Adverse Events (SAEs) (time frame: up to Day 540). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06848296.

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 4 May 2026.

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