ClinicalTrials.gov record NCT07470762
Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria
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
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This is an open-label Phase 1b/2 study of oral HS-10542 in adults with PNH and signs of active hemolysis. Phase 1b explores two dose levels (low and high, randomized 1:1) to assess safety, pharmacology, and to select a recommended Phase 2 dose. Phase 2 evaluates that selected dose in participants with PNH who have an inadequate response to C5 complement inhibitor therapy.
What it is studying. The study is testing whether oral HS-10542 (which inhibits factor B of the complement alternative pathway) is safe, what dose should be used in further studies, and how the drug affects blood markers and clinical measures of hemolysis in adults with PNH who have active hemolysis.
What the researchers hope to learn. Determine the safety and tolerability of HS-10542; identify the recommended Phase 2 dose. Evaluate effects on hemoglobin levels, lactate dehydrogenase (LDH), transfusion requirements, incidence of breakthrough hemolysis and major vascular events, and patient-reported fatigue scores.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- HS-10542
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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
- 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，
Exclusion criteria
- 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.
- 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,
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants take the study drug HS-10542 by mouth once daily (Phase Ib: randomized low or high dose; Phase II: the recommended dose from Phase Ib). They will attend regular clinic visits for safety checks and blood tests (including hemoglobin, LDH, reticulocytes) and will have records kept of any red‑blood‑cell transfusions. Participants will complete questionnaires such as the FACIT‑fatigue and be monitored for adverse events; Phase Ib outcomes are followed through about 12 weeks and Phase II through about 24 weeks. Vaccination against meningococcal and pneumococcal infection is required before dosing (or preventive antibiotics per the study rules).
How the study is structured. Open-label, interventional Phase 1b/2 study. Phase 1b randomizes participants 1:1 to low or high daily oral doses of HS-10542 (stratified by current C5 inhibitor use) to evaluate safety and pharmacology and choose a recommended Phase 2 dose. Phase 2 gives the selected daily oral dose to a group of participants with PNH who have an inadequate response to C5 inhibitors to evaluate safety and clinical effects. There is no masking; the trial uses parallel treatment groups.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
- The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on paroxysmal nocturnal hemoglobinuria
Important dates
- Study start
- Feb 14, 2026
- Primary completion
- May 30, 2027 (estimated)
- Study completion
- Jul 31, 2029 (estimated)
- Target enrollment
- 50 participants (estimated)
- Last updated on registry
- Apr 13, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Jiangsu Hansoh Pharmaceutical Co., Ltd.
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Yan Hong Tong 📞 13958122357 ✉ hongyantong@aliyun.com
Participating sites (1)
- The First Affiliated Hospital，Zhejiang University School of MedicineRecruiting
Hangzhou, Zhejiang, 310000 · China
- Yan Hong Tong · 13958122357 · hongyantong@aliyun.com
Terms used on this page · explanations by MedicalUpdates.ai
- open label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- during screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- Upper Limit of Normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- informed consent form
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- ECG
- A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
- immunosuppressant
- A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
- glucocorticoids
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- 5 half-lives
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- eGFR
- A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- randomized
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- 1:1
- How many participants go to each group. In a 2:1 ratio, two are assigned to the first group for every one assigned to the second.
- stratified
- Randomisation done separately within subgroups, such as age bands or disease severity, so that each group contains a similar mix.
- PK
- How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
- Phase II
- A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
- RP2D
- The dose chosen at the end of a phase 1 study for testing in later studies, based on safety and other measurements.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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