Paroxysmal Nocturnal Hemoglobinuria (PNH)Phase 2NCT06561841
A Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）
A Phase 2 study testing HSK39297 in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Haisco Pharmaceutical Group Co., Ltd. at 1 site in China.
Trial summary
This is a multicenter, randomized, open-label phase 2 study. Adult Patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included. Subjects were treated with HSK39297 for 24 weeks. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- What is being tested?
- HSK39297
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 47 participants
- Where
- 1 siteChina
- Study dates
- 17 Jul 2024to 2 Apr 2025
- Phase
- Phase 2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female participants ≥ 18 years of age;
- Diagnosis of PNH based on flow cytometry with clone size > 10% by granulocytes;
- Have not received complement inhibitor treatment;
- Blood lactate dehydrogenase(LDH) values > 1.5 ×upper limit of the normal range (ULN) ;
- Hemoglobin level < 100 g/L during the screening period.
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
1 site in 1 country.
China1 site
The First Affiliated Hospital of Nanjing Medical University
Nanjing, Jiangsu, 210029
About this study
Official title: A Multicenter, Randomized, Open-label Phase 2 Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）
This is a multicenter, randomized, open-label phase 2 study. Adult Patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included. Subjects were treated with HSK39297 for 24 weeks.
- Being tested
- HSK39297
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HSK39297Drug
HSK39297 tablets for 24 weeks
| Study group | Role | What they receive |
|---|---|---|
| Treatment group A | Experimental | Not described Receives: Drug: HSK39297 |
| Treatment group B | Experimental | Not described Receives: Drug: HSK39297 |
| Treatment group C | Experimental | Not described Receives: Drug: HSK39297 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of participants with increase in hemoglobin levels from baseline of ≥20 g/L in the absence of red blood cell transfusions
Measured: Baseline, 24 weeks
Secondary outcomes (12)
Proportion of participants with at least 60% reduction in LDH compared to baseline or LDH below the upper limit of normal
Measured: Baseline, 24 weeks
Change from baseline in hemoglobin
Measured: Baseline, 24 weeks
Change from baseline in reticulocyte count
Measured: Baseline, 24 weeks
Change from baseline in LDH
Measured: Baseline, 24 weeks
Change from baseline in Indirect bilirubin
Measured: Baseline, 24 weeks
Change from baseline in free hemoglobin
Measured: Baseline, 24 weeks
Proportion of participants without requiring red blood cells (RBC) transfusions
Measured: From week 4 to week 24
Change in the average number of RBC transfused per week
Measured: From week 4 to week 24
Change from baseline in PNH RBC clone size
Measured: Baseline, 24 weeks
Change from baseline in C3 fragment deposition on PNH RBC
Measured: Baseline, 24 weeks
Change from baseline in FACIT-Fatigue score
Measured: Baseline, 24 weeks
Incidence and severity of adverse events
Measured: 28 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2024
- Study start17 Jul 2024
- Primary completion2 Apr 2025
- Study completion2 Apr 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)Studies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 47 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
- NCT06561841
- Sponsor's protocol ID
- HSK39297-201
- Status last verified
- Aug 2024
- First submitted
- 16 Aug 2024
- Last updated
- 29 Jun 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Open-label Phase 2 Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）
- ClinicalTrials.gov ID
- NCT06561841
- Acronym / sponsor ID
- HSK39297-201
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 47 participants (actual)
- Lead sponsor
- Haisco Pharmaceutical Group Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 17 Jul 2024 (actual)
- Primary completion
- 2 Apr 2025 (actual)
- Study completion
- 2 Apr 2025 (actual)
- First posted
- 20 Aug 2024 (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.
This is a multicenter, randomized, open-label phase 2 study. Adult Patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included. Subjects were treated with HSK39297 for 24 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Treatment group A | Experimental | Drug: HSK39297 |
| Treatment group B | Experimental | Drug: HSK39297 |
| Treatment group C | Experimental | Drug: HSK39297 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Male and female participants ≥ 18 years of age;
- Diagnosis of PNH based on flow cytometry with clone size > 10% by granulocytes;
- Have not received complement inhibitor treatment;
- Blood lactate dehydrogenase(LDH) values > 1.5 ×upper limit of the normal range (ULN) ;
- Hemoglobin level < 100 g/L during the screening period.
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 |
|---|---|---|
| HSK39297HSK39297 tablets for 24 weeks | Drug | Treatment group A, Treatment group B, Treatment group C |
Primary outcome
Proportion of participants with increase in hemoglobin levels from baseline of ≥20 g/L in the absence of red blood cell transfusions
Time frame: Baseline, 24 weeks
12 secondary outcomes
Proportion of participants with at least 60% reduction in LDH compared to baseline or LDH below the upper limit of normal
Time frame: Baseline, 24 weeks
Change from baseline in hemoglobin
Time frame: Baseline, 24 weeks
Change from baseline in reticulocyte count
Time frame: Baseline, 24 weeks
Change from baseline in LDH
Time frame: Baseline, 24 weeks
Change from baseline in Indirect bilirubin
Time frame: Baseline, 24 weeks
Change from baseline in free hemoglobin
Time frame: Baseline, 24 weeks
Proportion of participants without requiring red blood cells (RBC) transfusions
Time frame: From week 4 to week 24
Change in the average number of RBC transfused per week
Time frame: From week 4 to week 24
Change from baseline in PNH RBC clone size
Time frame: Baseline, 24 weeks
Change from baseline in C3 fragment deposition on PNH RBC
Time frame: Baseline, 24 weeks
Change from baseline in FACIT-Fatigue score
Time frame: Baseline, 24 weeks
Incidence and severity of adverse events
Time frame: 28 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 29 Jun 2025.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing HSK39297 in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Haisco Pharmaceutical Group Co., Ltd. at 1 site 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 29 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 with increase in hemoglobin levels from baseline of ≥20 g/L in the absence of red blood cell transfusions (time frame: Baseline, 24 weeks). 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 29 Jun 2025.