PNHPhase 3NCT06799546
A Phase III Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy
A Phase 3 study testing HSK39297 tablets and Eculizumab Injection in people with PNH, run by Haisco Pharmaceutical Group Co., Ltd. at 2 sites in China.
Trial summary
The purpose of this study is to evaluate the efficacy and safety of HSK39297 tablets compared to eculizumab in Patients with PNH who Are Naive to Complement Inhibitor Therapy. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- PNH
- What is being tested?
- HSK39297 tablets and Eculizumab Injection
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 73 participants
- Where
- 2 sitesChina
- Study dates
- 13 Feb 2025to 5 Nov 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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
- Age ≥ 18 and ≤ 75 years, Male and female patients;
- Diagnosis of PNH based on flow cytometry with clone size > 10% by granulocytes;
- Have not received complement inhibitor treatment;
- Blood LDH values > 1.5 ×upper limit of the normal range (ULN) ;
- Hemoglobin level < 10 g/dL at screening.
Who cannot join
- Hereditary or acquired complement deficiency;
- Active primary or secondary immunodeficiency;
- History of splenectomy, bone marrow/ hematopoietic stem cell or solid organ transplants;
- History of recurrent invasive infections caused by encapsulated organisms( e.g. meningococcus or pneumococcus) or Mycobacterium tuberculosis;
- Patients with laboratory evidence of bone marrow failure (reticulocytes < 100x10^9/L, or platelets < 30x10^9/L or neutrophils < 0.5x10^9/L) ;
- Active systemic infection within 2 weeks prior to study drug administration;
- History of serious comorbidities that have been determined to be unsuitable for participation in the study.
- Pregnant or Lactating women.
Trial locations and contacts
2 sites in 1 country.
China2 sites
The First Affiliated Hospital, College of Medicine, Zhejiang University
Hangzhou, Zhejiang, 310003
The First Affiliated Hospital of Nanjing Medical University
Nanjing, Jiangsu, 210029
About this study
Official title: A Phase III, Multicenter, Randomized, Open Label, Active-Controlled Study to Evaluate the Efficacy and Safety of HSK39297 Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Naive to Complement Inhibitor Therapy
The purpose of this study is to evaluate the efficacy and safety of HSK39297 tablets compared to eculizumab in Patients with PNH who Are Naive to Complement Inhibitor Therapy.
- Conditions
- PNH
- Being tested
- HSK39297 tablets, Eculizumab Injection
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HSK39297 tabletsDrug
200mg QD for 24 weeks
Eculizumab InjectionDrug
Eculizumab Injection for 24 weeks
| Study group | Role | What they receive |
|---|---|---|
| HSK39297 tablets | Experimental | Not described Receives: Drug: HSK39297 tablets |
| Eculizumab Injection | Active comparator | Not described Receives: Drug: Eculizumab Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of participants achieving hemoglobin levels ≥ 12 g/dL at least on three out of four measurements in the absence of red blood cell transfusions
Measured: Between Week 18 and Week 24
Secondary outcomes (8)
Proportion of participants with increase in hemoglobin levels from baseline of ≥20 g/L at least on three out of four measurements in the absence of red blood cell transfusions
Measured: Between Week 18 and Week 24
Proportion of participants who did not receive blood transfusion.
Measured: Between Week 2 and Week 24
Change from baseline in hemoglobin
Measured: Baseline, week 18 to 24
Change From Baseline in Reticulocyte Count
Measured: Baseline, week 18 to 24
Percent change from baseline in LDH
Measured: Baseline, week 18 to 24
Change from baseline in FACIT-Fatigue score
Measured: Baseline, week 18 to 24
Rate of breakthrough hemolysis (BTH)
Measured: 24 weeks
Proportion of participants with Major Adverse Vascular Events (MAVEs)
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jan 2025
- Study start13 Feb 2025
- Primary completion5 Nov 2025
- Study completion5 Nov 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PNHStudies of the same condition, open and under way.
- More trials run by Haisco Pharmaceutical Group Co., Ltd.The sponsor's other registered studies.
- 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
- 73 participants (actual)
Sponsor and investigators
- Lead sponsor
- Haisco Pharmaceutical Group Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06799546
- Sponsor's protocol ID
- HSK39297-301
- Status last verified
- Dec 2025
- First submitted
- 23 Jan 2025
- Last updated
- 22 Dec 2025
- 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 22 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III, Multicenter, Randomized, Open Label, Active-Controlled Study to Evaluate the Efficacy and Safety of HSK39297 Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Naive to Complement Inhibitor Therapy
- ClinicalTrials.gov ID
- NCT06799546
- Acronym / sponsor ID
- HSK39297-301
- Conditions
- PNH
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 73 participants (actual)
- Lead sponsor
- Haisco Pharmaceutical Group Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 13 Feb 2025 (actual)
- Primary completion
- 5 Nov 2025 (actual)
- Study completion
- 5 Nov 2025 (actual)
- First posted
- 29 Jan 2025 (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.
The purpose of this study is to evaluate the efficacy and safety of HSK39297 tablets compared to eculizumab in Patients with PNH who Are Naive to Complement Inhibitor Therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| HSK39297 tablets | Experimental | Drug: HSK39297 tablets |
| Eculizumab Injection | Active comparator | Drug: Eculizumab Injection |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age ≥ 18 and ≤ 75 years, Male and female patients;
- Diagnosis of PNH based on flow cytometry with clone size > 10% by granulocytes;
- Have not received complement inhibitor treatment;
- Blood LDH values > 1.5 ×upper limit of the normal range (ULN) ;
- Hemoglobin level < 10 g/dL at screening.
Key exclusion criteria (8 listed)
- Hereditary or acquired complement deficiency;
- Active primary or secondary immunodeficiency;
- History of splenectomy, bone marrow/ hematopoietic stem cell or solid organ transplants;
- History of recurrent invasive infections caused by encapsulated organisms( e.g. meningococcus or pneumococcus) or Mycobacterium tuberculosis;
- Patients with laboratory evidence of bone marrow failure (reticulocytes < 100x10^9/L, or platelets < 30x10^9/L or neutrophils < 0.5x10^9/L) ;
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 |
|---|---|---|
| HSK39297 tablets200mg QD for 24 weeks | Drug | HSK39297 tablets |
| Eculizumab InjectionEculizumab Injection for 24 weeks | Drug | Eculizumab Injection |
Primary outcome
Proportion of participants achieving hemoglobin levels ≥ 12 g/dL at least on three out of four measurements in the absence of red blood cell transfusions
Time frame: Between Week 18 and Week 24
8 secondary outcomes
Proportion of participants with increase in hemoglobin levels from baseline of ≥20 g/L at least on three out of four measurements in the absence of red blood cell transfusions
Time frame: Between Week 18 and Week 24
Proportion of participants who did not receive blood transfusion.
Time frame: Between Week 2 and Week 24
Change from baseline in hemoglobin
Time frame: Baseline, week 18 to 24
Change From Baseline in Reticulocyte Count
Time frame: Baseline, week 18 to 24
Percent change from baseline in LDH
Time frame: Baseline, week 18 to 24
Change from baseline in FACIT-Fatigue score
Time frame: Baseline, week 18 to 24
Rate of breakthrough hemolysis (BTH)
Time frame: 24 weeks
Proportion of participants with Major Adverse Vascular Events (MAVEs)
Time frame: 24 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Dec 2025.
- Individual participant dataSharing planned: Undecided
Related trials for PNH
Active, not recruitingPhase 3
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Paroxysmal Nocturnal Hemoglobinuria
Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
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RecruitingObservational study
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 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 HSK39297 tablets and Eculizumab Injection in people with PNH, run by Haisco Pharmaceutical Group Co., 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 5 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: finished, as of the sponsor's last update on 22 Dec 2025. 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 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Proportion of participants achieving hemoglobin levels ≥ 12 g/dL at least on three out of four measurements in the absence of red blood cell transfusions (time frame: Between Week 18 and Week 24). 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 22 Dec 2025.