A Phase II Clinical Study Evaluating SLN12140 in Complement Inhibitor-Naïve Adult Subjects With Paroxysmal Nocturnal Hemoglobinuria
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 10
- 主要终点
- During the 12-week treatment period, the proportion of participants whose Lactate Dehydrogenase (LDH) decreased by 60% or more from baseline or whose LDH was below the upper limit
研究概览
简要总结
The goal of this clinical trial is to learn if drug SLN12140 works to treat Complement Inhibitor-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria in adults. It will also learn about the safety, pharmacokinetic characteristics, and dosing of drug SLN12140.
The study is divided into four phases: screening period, core treatment period, extended dosing period, and follow-up period, and includes two cohorts (Cohorts 1-2), with each cohort enrolling at least 5 treatment-naïve adult PNH subjects for complement inhibitor therapy.
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 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult complement inhibitor naïve PNH patients (age>=18), which is confirmed by flow cytometry evaluation
- •Must be vaccinated against meningococcal vaccine and pneumococcal vaccine
排除标准
- •Significant bone marrow failure
- •Meningitidis infection or unresolved meningococcal disease
- •Other significant systemic diseases that might have impact on efficacy and safety assessment
研究组 & 干预措施
SLN 12140 will be administered subcutaneously .
5 participants will receive SLN12140 100mg QW for 4 weeks, then 300mg QW for 8 weeks, then 200mg QW for 52 weeks.
5 participants will receive SLN12140 200mg QW for 4 weeks, then 600mg Q4W for 60 weeks
干预措施: SLN12140 (Drug)
结局指标
主要结局
During the 12-week treatment period, the proportion of participants whose Lactate Dehydrogenase (LDH) decreased by 60% or more from baseline or whose LDH was below the upper limit
时间窗: 12weeks after baseline
To assess efficacy of SLN12140 in participants with PNH
次要结局
- Percentage change of LDH from baseline(Baseline through Week 64)
- Proportion of participants achieving hemolysis control (LDH ≤ 1.5×ULN)(Baseline through Week 64)
- Change in hemoglobin (Hb) levels from baseline(Baseline through week 64)
- The proportion of participants whose hemoglobin (Hb) increased by ≥2 g/dL from baseline and who avoided blood transfusion(Baseline through Week 64)
- Proportion of participants who avoided blood transfusion(Baseline through Week 64)
- Incidence(%) of Breakthrough Hemolysis (BTH)(Baseline through Week 64)
- Changes from baseline in intravascular and extravascular hemolysis indicators (including but not limited to reticulocytes, bilirubin, red blood cell count, platelet count, ferritin, etc.)(baseline through week 64)
- Changes in thrombus formation risk markers from baseline (including but not limited to fibrinogen, prothrombin time, activated partial thromboplastin time, thrombin time, fibrin D-dimer, etc.);(Baseline through week 64)
- Change in functional assessment of Functional Assessment of Chronic Illness Therapy (FACIT)(Baseline through Week 64)
- Number(%) of participants with Adverse Events (AEs) , Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)(Baseline through Week 64)
- Pharmacokinetics (PK)parameters of SLN12140: Area Under The Plasma Concentration-time Curve(Baseline through week 64 (predose and postdose))
- Immunegenicity in Paraxysmal Nocturnal Hemoglobinuria(Baseline through Week 64)
- PK: Maximum Plasma Concentration (Cmax)(Baseline through week 64( predose and postdose))
- PK: Time To Maximum Concentration (Tmax)(Baseline through week 64( predose and postdose))
- Complement Alternative Pathway (AP) Functional Activity(Baseline through week 64( predose and post dose))
- Complement FP(Baseline through week 64(predose and postdose))