ClinicalTrials.gov record NCT07657052
A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria
A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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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 multicenter, open-label study follows about 26 people with PNH who previously received and completed XH-S003 study treatment. Participants take optimized-dose XH-S003 capsules orally until the study ends. The study measures long-term safety (adverse events) and blood-related outcomes over an average of one year.
What it is studying. The study is testing the long-term safety of continued oral XH-S003 and its effects on hemoglobin levels, need for red blood cell transfusions, occurrence of breakthrough hemolysis, and major vascular events in people with PNH.
What the researchers hope to learn. To evaluate long-term safety by recording the incidence and severity of adverse events; to assess efficacy measures including proportion of participants with hemoglobin ≥120 g/L without transfusion, change in hemoglobin without transfusions, proportion remaining transfusion-free, proportion with breakthrough hemolysis, and proportion with major adverse vascular events (MAVEs).
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- XH-S003 Capsule
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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);
Exclusion criteria
- . 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
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
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 XH-S003 capsules by mouth according to the study protocol and may continue treatment until the study ends. They will have regular safety monitoring and testing through study completion (an average of 1 year) to record adverse events and measures such as hemoglobin level, transfusion use, breakthrough hemolysis, and major adverse vascular events. Vaccination against Neisseria meningitidis and Streptococcus pneumoniae must be up to date and boosters may be required. The registry lists drug treatment and safety/efficacy monitoring but does not give a detailed visit schedule or list other specific procedures.
How the study is structured. Open-label, single-group drug study. About 26 participants who previously completed XH-S003 treatment will take XH-S003 capsules by mouth at an optimized dose and continue treatment until study end. There is no placebo or masking. Safety and blood-related outcomes are monitored through study completion (about one year on average).
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry says participants are enrolled by invitation. Who decides who is invited, and how?
- 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
- Aug 15, 2025
- Primary completion
- Sep 30, 2029 (estimated)
- Study completion
- Sep 30, 2029 (estimated)
- Target enrollment
- 26 participants (estimated)
- Last updated on registry
- Sep 25, 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
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Participating sites (1)
- TianJin Medical University General Hospital
Tianjin, Jinan, 300000 · China
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.
- prior to screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- estimated glomerular filtration rate
- 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.
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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