A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (XH-S003-II-102)

A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

This is a multicenter, open-label study to evaluate the long-term safety and efficacy of XH-S003 capsules in PNH patients. About 26 PNH patients who had received and completed XH-S003 study treatment will be enrolled to take XH-S003 capsules orally according to protocol. Eligible subjects can maintain XH-S003 treatment until the end of the study.

Study Overview

• Status: Enrolling by invitation
• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Intervention / Treatment: Drug: XH-S003 Capsule

• Study Type: Interventional
• Enrollment (Estimated): 26
• Phase: Not Applicable

Contacts and Locations

Study Locations

• China
  • Jinan
    • Tianjin, Jinan, China, 300000
      • Tianjin Medical University General Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. PNH patients who have previously received and completed the XH-S003 study treatment, and according to the investigator's evaluation, the benefits of treatment outweigh risks and may benefit from continued treatment with XH-S003;
2. Subjects who have been vaccinated against Neisseria meningitidis and Streptococcus pneumoniae as required by previous studies (if the vaccine protection period does not cover the duration of this study, timely booster vaccinations should be administered according to vaccination guidelines and local vaccination requirements);

Exclusion Criteria:

1. . History of malignancy in any organ or system within the past 5 years prior to screening (excluding localized basal cell carcinoma of the skin or in situ cervical cancer);
2. History of bone marrow/hematopoietic stem cell or solid organ transplantation;
3. History of splenectomy;
4. History of serious comorbidities , including but not limited to: advanced stage heart disease (e.g., New York Heart Association [NYHA] Class IV), severe lung disease (e.g., severe pulmonary hypertension [WHO Class IV]), severe liver function injury, severe renal disease (estimated glomerular filtration rate [eGFR] < 30 mL/min/1.73 m2 or chronic kidney disease [CKD] Stage 4 or dialysis patients), unstable thrombus, other hematologic system diseases (e.g., chronic anemia unrelated to PNH), and deemed unsuitable for study participation by the investigator;
5. Pregnant or lactating women, or those with a positive pregnancy test;
6. Known or suspected immunodeficiency diseases or hereditary complement deficiency at screening;
7. A history of Neisseria meningitidis infection

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: XH-S003 Capsule; XH-S003 capsule orally taken according to protocol until the end of treatment	Drug: XH-S003 Capsule; Optimized dose

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence and severity of adverse events	through study completion，an average of 1 year

Secondary Outcome Measures

Outcome Measure	Time Frame
Proportion of subjects with Hb ≥120g/L compared with baseline (without RBC transfusion)	through study completion，an average of 1 year
Change From Baseline in Hemoglobin in the absence of red blood cell transfusions	through study completion，an average of 1 year
Proportion of participants who remain free from transfusions	through study completion，an average of 1 year
Proportion of participants with breakthrough hemolysis (BTH)	through study completion，an average of 1 year
Proportion of participants with Major Adverse Vascular Events (MAVEs)	through study completion，an average of 1 year

Collaborators and Investigators

• Sponsor: S-INFINITY Pharmaceuticals Co., Ltd
• Investigators: Principal Investigator: Rong Fu, Doctor, Tianjin Medical University General Hospital

Study record dates

Study Major Dates

• Study Start (Actual): August 15, 2025
• Primary Completion (Estimated): September 30, 2029
• Study Completion (Estimated): September 30, 2029

Study Registration Dates

• First Submitted: May 29, 2026
• First Submitted That Met QC Criteria: June 17, 2026
• First Posted (Actual): June 18, 2026

Study Record Updates

• Last Update Posted (Actual): June 18, 2026
• Last Update Submitted That Met QC Criteria: June 17, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Anemia, Hemolytic; Anemia; Myelodysplastic Syndromes; Hemic and Lymphatic Diseases; Hemoglobinuria, Paroxysmal
• Other Study ID Numbers: XH-S003-II-102

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No