NCT05876351已完成3 期
Prospective, Single-Arm, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Complement Inhibitor Treatment-Naïve Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 25
- 试验地点
- 1
- 主要终点
- Percentage of Participants With a Complete Thrombotic Microangiopathy (TMA) Response
研究概览
简要总结
This is a Phase 3b, open-label, single-arm, multicenter study to evaluate the efficacy and safety of eculizumab in participants with atypical hemolytic uremic syndrome (aHUS) in China
详细描述
This is a Phase 3b, open-label, single-arm, multicenter study to evaluate the efficacy and safety of eculizumab in participants with aHUS in China. The study will be conducted in participants of any age who weigh ≥ 5 kg and who previously have not been treated with complement inhibitors. The study consists of an up to 7-day Screening Period and a 26-week Treatment Period. An 8-week Safety Follow-up Phone Call will be required only for participants who discontinue eculizumab treatment during the study or for participants who will not receive continued access to eculizumab after completing study treatment. Approximately 25 eligible participants in China will be enrolled.
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
- 0 Years 至 99 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Any age weighing ≥ 5 kg
- •Complement treatment naïve with evidence of TMA.
- •History of aHUS prior to kidney transplant,or persistent evidence of TMA at least 4 days after modifying the immunosuppressive regimen.
- •Among participants with onset of TMA postpartum, persistent evidence of TMA for > 3 days after the day of childbirth
- •All participants must be vaccinated against N meningitidis if not already vaccinated within the time period of active coverage specified by the vaccine manufacturer.
- •Participants < 18 years of age must have been vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae according to local vaccination schedule guidelines.
- •In participants receiving treatment with medications known to cause TMA, persistent evidence of TMA at least 4 days after modifying the excluded medication
排除标准
- •Known familial or acquired ADAMTS13deficiency (activity < 5%).
- •ST-HUS as demonstrated by local guidelines.
- •Positive direct Coombs test which is indicative of a clinically significant immune-mediated hemolysis not due to aHUS.
- •HIV infection, and /or unresolved meningococcal disease
- •Ongoing sepsis, and / or presence or suspicion of active and untreated systemic infection
- •Organ transplantation history, and/or Bone marrow transplant/hematopoietic stem cell transplant within 6 months prior to the start of Screening.
- •Among participants with a kidney transplant, acute kidney dysfunction within 4 weeks of transplant consistent with the diagnosis of acute antibody-mediated rejection.
- •Among participants without a kidney transplant, history of kidney disease other than aHUS
- •Identified drug exposure-related HUS, and / or HUS related to vitamin B12 deficiency and / or known genetic defects of cobalamin C metabolism.
- •History of malignancy within 5 years of Screening.
- •Known systemic sclerosis (scleroderma), systemic lupus erythematosus, or antiphospholipid antibody positivity or syndrome.
- •Chronic dialysis.
- •Prior use of complement inhibitors.
- •Use of tranexamic acid within 7 days prior to the start of Screening.
- •Other immunosuppressive therapies.
- •Receiving chronic intravenous immunoglobulin (IVIg) within 8 weeks prior to the start of Screening.
- •Received vasopressors or inotropes within 7 days prior to Screening.
- •Previously or currently treated with a complement inhibitor.
- •Has participated in another interventional treatment study or used any experimental therapy.
- •Hypersensitivity to any excipient in eculizumab.
- •Pregnant or breastfeeding.
研究组 & 干预措施
Eculizumab
Experimental
Participants will receive Eculizumab in a single dose vial.
干预措施: Eculizumab (Drug)
结局指标
主要结局
Percentage of Participants With a Complete Thrombotic Microangiopathy (TMA) Response
时间窗: Up to Week 26
The criteria for complete TMA response were: 1. Normalization of platelet count (defined as platelet count ≥ 150000/microliter (ul). 2. Normalization of lactate dehydrogenase (LDH, defined as LDH ≤ upper limit of normal \[ULN\]). 3. ≥ 25% improvement in serum creatinine from baseline.
次要结局
- Number of Participants With an Anti-drug Antibody (ADA) Response(Up to Week 26)
- Time to Complete TMA Response(Up to Week 26)
- Proportion of Participants On or Off Dialysis at Each Timepoint(Baseline and Days 22, 43, 71, 99, 113, 127, 155 and 183)
- Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Each Scheduled Visit(Baseline, Days 22, 43, 71, 99, 113, 127, 155 and 183)
- Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline(Baseline to Days 22, 43, 71, 99, 113, 127, 155 and 183)
- Change From Baseline in Platelets(Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183)
- Change From Baseline in LDH(Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183)
- Change From Baseline in Hemoglobin(Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183)
- Mean Serum Concentration of Eculizumab(Pre-dose and post-dose at Days 1, 8, 29, 85, and 141; Pre-dose at Day 183)
- Change From Baseline in Serum Free Complement 5 (C5)(Baseline (Day 1 pre-dose) to Days 1, 8, 29, 85 and 141 (pre-dose and post-dose) and pre-dose at Day 183)
- Change From Baseline in Serum Total C5(Baseline (Day 1 pre-dose) to Days 1, 8, 29, 85 and 141 (pre-dose and post-dose) and pre-dose at Day 183)
- Number of Participants With an Adverse Event (AE)(Up to Week 34)
研究者
研究点 (1)
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