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

NCT07470762 | Recruiting Phase 1

• 1 other identifier: HS-10542-201
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

HS-10542; paroxysmal nocturnal hemoglobinuria（PNH）; CFB

Outcome Measures

Primary Outcomes (2)

• Phase 1b: Incidence and severity of adverse events

  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

  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

  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

  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

  From the 3th to the 12th week

• Phase 1b: Change in LDH level from baseline

  12 weeks

• Ph1b:change from baseline in hemoglobin concentration

  12 weeks

Study Arms (1)

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

Drug: HS-10542

Interventions

HS-10542 DRUG

HS-10542 low dose,QD

Ph1b: HS-10542 two dose ；Ph2: RP2D

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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，

You may not qualify if:

• 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

Sponsors & Collaborators

• Jiangsu Hansoh Pharmaceutical Co., Ltd.: lead

Study Sites (1)

The First Affiliated Hospital，Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310000, China

RECRUITING

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Central Study Contacts

Yan Hong Tong

CONTACT

13958122357; hongyantong@aliyun.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 26, 2026
• First Posted: March 13, 2026
• Study Start: February 14, 2026
• Primary Completion (Estimated): May 30, 2027
• Study Completion (Estimated): July 31, 2029
• Last Updated: April 13, 2026

Record last verified: 2026-01

Locations

CN (1)