NCT07657052Enrolling By Invitation不适用
A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.1 个研究点 分布在 1 个国家目标入组 26 人开始时间: 2025年8月15日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 26
- 试验地点
- 1
- 主要终点
- Incidence and severity of adverse events
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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);
排除标准
- •. 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
研究组 & 干预措施
XH-S003 Capsule
Experimental
XH-S003 capsule orally taken according to protocol until the end of treatment
干预措施: XH-S003 Capsule (Drug)
结局指标
主要结局
Incidence and severity of adverse events
时间窗: through study completion，an average of 1 year
次要结局
- 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 subjects with Hb ≥120g/L compared with baseline (without RBC transfusion)(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)
- Change From Baseline in Hemoglobin in the absence of red blood cell transfusions(through study completion，an average of 1 year)
研究者
研究点 (1)
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