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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| XH-S003 Capsule | Experimental | XH-S003 capsule orally taken according to protocol until the end of treatment |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| XH-S003 Capsule | Drug | Optimized dose |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence and severity of adverse events | through study completion,an average of 1 year |
| Measure | Description | 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Rong Fu, Doctor | Tianjin Medical University General Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| TianJin Medical University General Hospital | Tianjin | Jinan | 300000 | China |
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| 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 |
| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D009190 | Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |
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