NCT06978699招募中2 期
A Multicenter, Randomized, Single-blind Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
S-INFINITY Pharmaceuticals Co., Ltd1 个研究点 分布在 1 个国家目标入组 24 人开始时间: 2025年4月30日最近更新:
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 24
- 试验地点
- 1
- 主要终点
- Changes of hemoglobin compared with baseline
研究概览
简要总结
This is a multicenter, randomized, single-blind Phase II trial to evaluate the efficacy and safety of XH-S003 capsules in PNH patients. About 24 PNH patients will be enrolled and randomized to three dose levels and take XH-S003 capsules orally
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Participant)
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
- •Male or female with aged ≥18 years old;
- •Weight ≥40 kg and BMI≥18 kg/m2 ;
- •Diagnosed with PNH: with red blood cell or granulocyte clone levels >10% detected by flow cytopy within 6 months prior to screening or during screening;
- •Patients who have not previously received any complement inhibitor therayp;
- •LDH > 1.5×ULN detected two times during the screening period (interval of 2 to 8 weeks);
- •Hb meets one of the following conditions: (1) Hb <100 g/L at the first screening visit, and subjects receive RBC transfusion because of PNH-related anemia during the screening period; (2) The average Hb of two tests during the screening period <100 g/L (interval of 2~8 weeks);
- •Vaccination against Neisseria meningitidis and Streptococcus pneumoniae before the first administration. If the subject has not been vaccinated previously or requires booster vaccination (according to local vaccination policies), vaccination must be administered at least 2 weeks before the first administration. If the first administration must begin less than 2 weeks after vaccination, preventive antibiotic treatment must begin at least 2 weeks after vaccination;
排除标准
- •Subjects with laboratory evidence of bone marrow failure during the screening period (reticulocyte count <100×109/L, platelet count <30×109/L, or neutrophil count <0.5×109/L);
- •Subjects receiving other therapies prior to screening who have not achieved the following treatment durations:
- •Erythropoietin or immunosuppressants for at least 8 weeks; • Systemic corticosteroids for at least 4 weeks; • Iron supplements, vitamin B12, or folic acid for at least 4 weeks; • Anticoagulants: Vitamin K antagonists for at least 4 weeks with stable international normalized ratio (INR) (as determined by the investigator), low molecular weight heparin for at least 4 weeks; • Hypoxic-inducing factor prolyl hydroxylase inhibitors (HIF-PHI) for at least 8 weeks; • Androgens for at least 4 weeks;
- •A history of bone marrow/hematopoietic stem cell or solid organ transplantation;
- •Alanine aminotransferase (ALT), γ-glutamyl transpeptidase (GGT), or alkaline phosphatase (ALP) >3×ULN at screening; - Positive HIV antibody, active syphilis infection, positive HBsAg, active HCV infection, or active tuberculosis infection at screening;
- •Known or suspected immunodeficiency diseases or hereditary complement deficiency at screening;
- •A history of Neisseria meningitidis infection;
- •Subjects with chronic active or recurrent infections within 1 year prior to screening;
- •Subjects with systemic active bacterial, viral (including COVID-19), or fungal infections within 2 weeks prior to the first administration; subjects with body temperature >38°C within 7 days prior to the first administration;
研究组 & 干预措施
Dose 1
Experimental
Once daily
干预措施: XH-S003 Capsule (Drug)
Dose 2
Experimental
Once daily
干预措施: XH-S003 Capsule (Drug)
Dose 3
Experimental
Once daily
干预措施: XH-S003 Capsule (Drug)
结局指标
主要结局
Changes of hemoglobin compared with baseline
时间窗: 8 weeks
Changes of hemoglobin compared with baseline
次要结局
- Proportion of subjects with an increase in Hb ≥20g/L compared with baseline (without RBC transfusion);(8 weeks)
- Changes in Indirect Bilirubin compared to baseline;(8 weeks)
- Changes in reticulocyte counts compared to baseline;(8 weeks)
- Proportion of subjects without RBC transfusion(8 weeks)
- Changes in LDH compared with baseline(8 weeks)
研究者
研究点 (1)
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