Paroxysmal Nocturnal HemoglobinuriaPhase 1/2NCT07470762

Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria

A Phase 1/2 study testing HS-10542 in people with Paroxysmal Nocturnal Hemoglobinuria, run by Jiangsu Hansoh Pharmaceutical Co., Ltd. at 1 site in China.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts

Official record

Search all trials

Trial summary

This was a phase 1b/2,open label, multi-center study to assess efficacy and safety of HS-10542 in adulte patients with paroxysmal nocturnal hemoglobinuria (PNH) with signs of active hemolysis. Read more about this study

Who is it for?: Ages 18 to 75 years
Conditions the study is for: Paroxysmal Nocturnal Hemoglobinuria
What is being tested?: HS-10542
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 50 participants
Where: 1 siteChina
Study dates: 14 Feb 2026to 31 Jul 2029
Phase: Phase 1/2
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

Men or women aged more than or equal to (≥) 18 years, and less than (≤) 75 years.
It was confirmed to be PNH during screening, and the clone size of red blood cells or/and granulocytes or/and monocytes was detected by flow cytopy ≥10%
Stable use of C5 complement inhibitor ikuzumab/covalimab for the first 6 months of random treatment
Have at least one blood transfusion record within the last 4 months, or sustain a hemoglobin level below 100g/L the last 4 months prior to screening.
The average hemoglobin level from two tests conducted by the laboratory at the time of screening is less than 100 g/L, or hemoglobin level <100g/L before transfusion.
LDH > 1.5 x Upper Limit of Normal (ULN) at the time of screening

Show all 10Show fewer

Inoccution of Neisseris meningitis and Streptococcus pneumoniae vaccine at least 2 weeks before the first administration of HS-10542;
if HS-10542 treatment must begin less than 2 weeks after vaccination, preventive antibiotic treatment must begin at least 2 weeks after vaccination.
Male and female subjects with fertility must agree to adopt efficient contraceptive measures with their partners within 60/120 days from the signing of the informed consent form to the last administration,
Male subjects who are infertile (such as those who have undergone effective sterilization surgery) must take additional efficient contraceptive measures when it is uncertain whether they have sperm，

Who cannot join

Known or suspected hereditary or acquired complement deficiency
Currently active primary or secondary immunodeficiency
History of infection with pod bacteria (such as Neisseris meningitis, Streptococcus pneumoniae, etc.)
Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L; platelets <30x109/L; neutrophils <0.5x109/L);
Presence of a bone marrow failure disorder (e.g., aplastic anemia, myelodysplastic syndrome, myelofibrosis)
Presence of active anemia unrelated to PNH, such as renal anemia or anemia due to blood loss.

Show all 23Show fewer

There is or is suspected of systemic active bacteria, virus or fungal infection 2 weeks before the first administration of HS-10542 (according to the researcher's judgment)
During screening, there are advanced heart disease (such as NYHA level IV),
unstable thrombosis events that may exist for other causes,
Abnormal ECG: The absolute value of QTcF (QT interval corrected by Fridericia 's formula > 450 msec for males and > 470 msec for females; or other clinically significant abnormalities as judged by the investigator.
Major surgery within 3 months prior to the first dose. *Note: See Appendix for definitions of Grade 3/4 surgeries.
Known active infection requiring systemic therapy
Diagnosed malignant tumors in the past 5 years
Those who have a history of splenectomy or History of bone marrow/hematopoietic stem cells or solid organ transplantation
Severe or poorly controlled hypertension
poorly controlled diabetes
Those who are suspected of being allergic to experimental drugs or any ingredient in experimental drugs
Use any of the following drugs, unless there is a stable treatment plan before screening: a) erythropoietin (ESA), hypoxic-inducing factor proaminoyl hydroxylase inhibitor (HIF-PHI) or immunosuppressant for at least 8 weeks b) Systemic use of glucocorticoids (≤15 mg/day Prednisone or equivalent doses of glucocorticoids) at least 4 weeks c) Vitamin K antagonists (such as warfarin) have a stable international standardized ratio (INR) at least 4 weeks d) Low molecular weight heparin, oral anticoagulants such as aspirin, rvaroxaban, apifloxaban, etc. at least 4 weeks e) Iron supplements , vitamin B12, folic acid or androgen for at least 4 weeks
Except for C5 complement inhibitors (including but not limited to ecucizumab and covalizumab), the situation of participating in other clinical trials or using other study drugs or approved therapies for experimental use before screening, and the trial drug is still within 5 half-lives or 2 weeks
Participants who have previously received B-factor inhibitor treatment, with a treatment duration of no more than one week and having stopped taking the drug for more than five half-lives before screening, may not be excluded
During screening, there are serious concurrent diseases, such as severe kidney disease (such as eGFR<30 mL/min/1.73 m2, dialysis),
ALT/ALP>3×ULN,
Screening positive blood pregnancy test and breastfeeding women at the time of the visit,

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Yan Hong Tong contact

13958122357

hongyantong@aliyun.com

China1 site, 1 recruiting

The First Affiliated Hospital，Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310000
Yan Hong Tong, 13958122357, hongyantong@aliyun.com
Recruiting

Other trials in China

About this study

Official title: A Phase IB/II,Open Label Study to Assess Efficacy, and Safety, of HS-10542 in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active Hemolysis

This was a phase 1b/2,open label, multi-center study to assess efficacy and safety of HS-10542 in adulte patients with paroxysmal nocturnal hemoglobinuria (PNH) with signs of active hemolysis.

Read the detailed description

HS-10542 is a novel oral small molecular weight compound that inhibits factor B (FB) of the alternative pathway (AP). Blockade of the AP with oral HS-10542 has the potential to prevent both intra - and extravascular hemolysis. This study consists of a dose exporation(Ph1b) and a dose expansion(Ph 2):

Phase Ib:In participants with paroxysmal nocturnal hemoglobinuria (PNH), two dose levels of HS-10542 will be explored (low dose; high dose randomized 1:1), stratified by whether the patient is currently receiving C5 complement inhibitor therapy. Based on an integrated assessment of interim data on the safety, efficacy, and PK/PD profile of HS-10542 in the target population, the recommended Phase II dose (RP2D) will be determined.

Phase II:Based on the safe and effective dose identified in Phase Ib, the efficacy and safety of HS-10542 will be evaluated in participants with PNH who have an inadequate response to C5 complement inhibitor therapy.

Conditions: Paroxysmal Nocturnal Hemoglobinuria
Being tested: HS-10542
Keywords: HS-10542, paroxysmal nocturnal hemoglobinuria（PNH）, CFB
Medical subject headings: Hemoglobinuria, Paroxysmal

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

HS-10542Drug
HS-10542 low dose,QD
HS-10542Drug
HS-10542 high dose, QD
HS-10542Drug
Orally QD ，The recommended dose from Ph1b

Study groups in NCT07470762
Study group	Role	What they receive
Ph1b: HS-10542 two dose ；Ph2: RP2D	Experimental	Phase Ib：two dose levels of HS-10542 will be explored (low dose; high dose, randomized 1:1) Phase II: Based on the safe and effective dose identified in Phase Ib, the efficacy and safety of HS-10542 will be evaluated in participants with PNH who have an inadequate response to C5 complement inhibitor therapy Receives: Drug: HS-10542

What is being measured

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

Phase 1b: Incidence and severity of adverse events
Measured: 12 weeks
Phase 2: In the absence of red blood cell infusion the proportion of subjects with at least 3 times of ≥120 g/L of hemoglobin level measured 4 times between weeks 18 and 24
Measured: From the 18th to the 24th week

Secondary outcomes (17)

Phase 1b: In the absence of red blood cell infusion the proportion of subjects of ≥120 g/L of hemoglobin level between weeks 3 and 12
Measured: From the 3th to the 12th week
Phase 1b: the Proportion of Participants With Sustained Increase in Hemoglobin Levels From Baseline of ≥ 20g/L in the Absence of Red Blood Cell Transfusions
Measured: hemoglobin between Day 15 and Day 84 and absence of transfusions
Phase 1b: The proportion of subjects who did not receive red blood cell infusion from week 3 to week 12
Measured: From the 3th to the 12th week
Phase 1b: Change in LDH level from baseline
Measured: 12 weeks
Ph1b:change from baseline in hemoglobin concentration
Measured: 12 weeks
Phase 1b: Change From Baseline in Hgb.LDH.free-Hgb. haptoglobin and ferritin
Measured: by week 2 and week 4
Phase 1b: The number of red blood cell units transfused by the participants who received blood transfusions
Measured: 12 weeks
Phase 1b: The average change of the score of Chronic Disease Treatment Function Assessment (FACIT) compared to the baseline
The FACIT-F is a 13 -item, self-reported PRO measure assessing an individual's level of fatigue during their usual daily activities over the past week. This questionnaire is part of the FACIT measurement system, a compilation of questions measuring health-related QoL in patients with cancer and other chronic illnesses. The FACIT-fatigue assesses the level of fatigue using a 4-point Likert scale ranging from 0 (not at all) to 4 (very much). Scores range from 0 to 52, with higher scores indicating greater fatigue.
Measured: Baseline and week 2, week 4, week 8, week 12
Phase 1b: The incidence of breakthrough hemolysis(BTH)from Week 1 to week 12
Measured: From the 1th to the 12th week
Phase 1b: The incidence of Major Adverse Vascular Events(MAVEs) from Week 1 to week 12
MAVEs are recorded on the AE page/. MAVEs include but are not limited to the following conditions:Acute peripheral vascular occlusion; Gangrene, amputation (non-traumatic, non-diabetic participants); Transient ischemic attack; Cerebral artery/vein occlusion/cerebrovascular accident; Hepatic/portal vein thrombosis (Budd-Chiari syndrome); Mesenteric/visceral artery or vein thrombosis; Unstable angina; Myocardial infarction; Pulmonary embolism; Renal artery/vein thrombosis; Thrombophlebitis/deep vein thrombosis.
Measured: From the 1th to the 12th week
Phase 2: the Proportion of Participants With Sustained Increase in Hemoglobin Levels From Baseline of ≥ 20 g/L in the Absence of Red Blood Cell Transfusions
Measured: From the 18th to the 24th week
Phase 2: The proportion of subjects who did not receive red blood cell infusion from week 3 to week 24
Measured: From the 3th to the 24th week
Phase 2: the average change of Hemoglobin compared to the baseline
Measured: From the 18th to the 24th week
Phase 2: the average change of Ret compared to the baseline
Measured: From the 18th to the 24th week
Phase 2: the average change of LDH compared to the baseline
Measured: From the 18th to the 24th week
Phase 2: Chronic Disease Treatment Function Assessment (FACIT) from weeks 18 to 24- the average change of the score compared to the baseline
The FACIT-F is a 13 -item, self-reported PRO measure assessing an individual's level of fatigue during their usual daily activities over the past week. This questionnaire is part of the FACIT measurement system, a compilation of questions measuring health-related QoL in patients with cancer and other chronic illnesses. The FACIT-fatigue assesses the level of fatigue using a 4-point Likert scale ranging from 0 (not at all) to 4 (very much). Scores range from 0 to 52, with higher scores indicating greater fatigue.
Measured: From the 18h to the 24th week
Phase 2: Incidence and severity of adverse events
Measured: From the 1th to the 24th week

Timeline

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

14 Feb 202631 Jul 2029

First posted13 Mar 2026
Study start14 Feb 2026
Primary completion30 May 2027 (estimated)
Study completion31 Jul 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 HemoglobinuriaStudies of the same condition, open and under way.
More trials run by Jiangsu Hansoh Pharmaceutical 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 1/2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 50 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT07470762
Sponsor's protocol ID: HS-10542-201
Status last verified: Jan 2026
First submitted: 26 Jan 2026
Last updated: 13 Apr 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No

Study at a glance

From the registry record, last updated by the sponsor on 13 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase IB/II,Open Label Study to Assess Efficacy, and Safety, of HS-10542 in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active Hemolysis
ClinicalTrials.gov ID: NCT07470762
Acronym / sponsor ID: HS-10542-201
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Keywords: HS-10542, paroxysmal nocturnal hemoglobinuria（PNH）, CFB
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Recruiting
Enrolment: 50 participants (estimated)
Lead sponsor: Jiangsu Hansoh Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
Study start: 14 Feb 2026 (actual)
Primary completion: 30 May 2027 (estimated)
Study completion: 31 Jul 2029 (estimated)
First posted: 13 Mar 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.

This was a phase 1b/2,open label, multi-center study to assess efficacy and safety of HS-10542 in adulte patients with paroxysmal nocturnal hemoglobinuria (PNH) with signs of active hemolysis.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Ph1b: HS-10542 two dose ；Ph2: RP2D	Experimental	Drug: HS-10542Phase Ib：two dose levels of HS-10542 will be explored (low dose; high dose, randomized 1:1) Phase II: Based on the safe and effective dose identified in Phase Ib, the efficacy and safety of HS-10542 will be evaluated in participants with PNH who have an inadequate response to C5 complement inhibitor therapy

Who may qualify

Ages: 18 to 75 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (10 listed)

Men or women aged more than or equal to (≥) 18 years, and less than (≤) 75 years.
It was confirmed to be PNH during screening, and the clone size of red blood cells or/and granulocytes or/and monocytes was detected by flow cytopy ≥10%
Stable use of C5 complement inhibitor ikuzumab/covalimab for the first 6 months of random treatment
Have at least one blood transfusion record within the last 4 months, or sustain a hemoglobin level below 100g/L the last 4 months prior to screening.
The average hemoglobin level from two tests conducted by the laboratory at the time of screening is less than 100 g/L, or hemoglobin level <100g/L before transfusion.

Key exclusion criteria (23 listed)

Known or suspected hereditary or acquired complement deficiency
Currently active primary or secondary immunodeficiency
History of infection with pod bacteria (such as Neisseris meningitis, Streptococcus pneumoniae, etc.)
Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L; platelets <30x109/L; neutrophils <0.5x109/L);
Presence of a bone marrow failure disorder (e.g., aplastic anemia, myelodysplastic syndrome, myelofibrosis)

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
HS-10542HS-10542 low dose,QD	Drug	Ph1b: HS-10542 two dose ；Ph2: RP2D
HS-10542HS-10542 high dose, QD	Drug	Ph1b: HS-10542 two dose ；Ph2: RP2D
HS-10542Orally QD ，The recommended dose from Ph1b	Drug	Ph1b: HS-10542 two dose ；Ph2: RP2D

Primary outcome

Phase 1b: Incidence and severity of adverse events
Time frame: 12 weeks
Phase 2: In the absence of red blood cell infusion the proportion of subjects with at least 3 times of ≥120 g/L of hemoglobin level measured 4 times between weeks 18 and 24
Time frame: From the 18th to the 24th week

17 secondary outcomes

Phase 1b: In the absence of red blood cell infusion the proportion of subjects of ≥120 g/L of hemoglobin level between weeks 3 and 12
Time frame: From the 3th to the 12th week
Phase 1b: the Proportion of Participants With Sustained Increase in Hemoglobin Levels From Baseline of ≥ 20g/L in the Absence of Red Blood Cell Transfusions
Time frame: hemoglobin between Day 15 and Day 84 and absence of transfusions
Phase 1b: The proportion of subjects who did not receive red blood cell infusion from week 3 to week 12
Time frame: From the 3th to the 12th week
Phase 1b: Change in LDH level from baseline
Time frame: 12 weeks
Ph1b:change from baseline in hemoglobin concentration
Time frame: 12 weeks
Phase 1b: Change From Baseline in Hgb.LDH.free-Hgb. haptoglobin and ferritin
Time frame: by week 2 and week 4
Phase 1b: The number of red blood cell units transfused by the participants who received blood transfusions
Time frame: 12 weeks
Phase 1b: The average change of the score of Chronic Disease Treatment Function Assessment (FACIT) compared to the baseline
Time frame: Baseline and week 2, week 4, week 8, week 12
Phase 1b: The incidence of breakthrough hemolysis(BTH)from Week 1 to week 12
Time frame: From the 1th to the 12th week
Phase 1b: The incidence of Major Adverse Vascular Events(MAVEs) from Week 1 to week 12
Time frame: From the 1th to the 12th week
Phase 2: the Proportion of Participants With Sustained Increase in Hemoglobin Levels From Baseline of ≥ 20 g/L in the Absence of Red Blood Cell Transfusions
Time frame: From the 18th to the 24th week
Phase 2: The proportion of subjects who did not receive red blood cell infusion from week 3 to week 24
Time frame: From the 3th to the 24th week
Phase 2: the average change of Hemoglobin compared to the baseline
Time frame: From the 18th to the 24th week
Phase 2: the average change of Ret compared to the baseline
Time frame: From the 18th to the 24th week
Phase 2: the average change of LDH compared to the baseline
Time frame: From the 18th to the 24th week
Phase 2: Chronic Disease Treatment Function Assessment (FACIT) from weeks 18 to 24- the average change of the score compared to the baseline
Time frame: From the 18h to the 24th week
Phase 2: Incidence and severity of adverse events
Time frame: From the 1th to the 24th week

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 13 Apr 2026.

Related trials for Paroxysmal Nocturnal Hemoglobinuria

Enrolling by invitationObservational study
An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea
Hemoglobinuria, Paroxysmal
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
Paroxysmal Nocturnal Hemoglobinuria
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)

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.

Yan Hong Tong contact

13958122357

hongyantong@aliyun.com

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing HS-10542 in people with Paroxysmal Nocturnal Hemoglobinuria, run by Jiangsu Hansoh Pharmaceutical 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 10 inclusion and 23 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: looking for participants now, as of the sponsor's last update on 13 Apr 2026. 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 combined 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.

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?

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 Yan Hong Tong (13958122357, hongyantong@aliyun.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Phase 1b: Incidence and severity of adverse events (time frame: 12 weeks). 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 NCT07470762.

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 13 Apr 2026.