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

NCT07657052 | Enrolling By Invitation

• 1 other identifier: XH-S003-II-102
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of adverse events

  through study completion，an average of 1 year

Secondary Outcomes (5)

• 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

Study Arms (1)

XH-S003 Capsule

EXPERIMENTAL

XH-S003 capsule orally taken according to protocol until the end of treatment

Drug: XH-S003 Capsule

Interventions

XH-S003 Capsule DRUG

Optimized dose

XH-S003 Capsule

Eligibility Criteria

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

You may qualify if:

• 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;
• 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);

You may not qualify if:

• 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);
• History of bone marrow/hematopoietic stem cell or solid organ transplantation;
• History of splenectomy;
• 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;
• Pregnant or lactating women, or those with a positive pregnancy test;
• Known or suspected immunodeficiency diseases or hereditary complement deficiency at screening;
• A history of Neisseria meningitidis infection

Sponsors & Collaborators

• S-INFINITY Pharmaceuticals Co., Ltd: lead

Study Sites (1)

TianJin Medical University General Hospital

Tianjin, Jinan, 300000, China

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

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

Study Officials

• Rong Fu, Doctor

  Tianjin Medical University General Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 29, 2026
• First Posted: June 18, 2026
• Study Start: August 15, 2025
• Primary Completion (Estimated): September 30, 2029
• Study Completion (Estimated): September 30, 2029
• Last Updated: June 18, 2026

Record last verified: 2026-06

Locations

CN (1)