NCT07154745招募中3 期
A Single Arm Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Control of Intravascular Hemolysis on Currently Available C5 Inhibitor Therapy
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 35
- 试验地点
- 20
- 主要终点
- Percent change in Lactate Dehydrogenase (LDH) during TP
研究概览
简要总结
This study is researching a treatment combination with two experimental drugs called pozelimab and cemdisiran referred to as "study drugs". Researchers are looking for a better way to treat Paroxysmal Nocturnal Hemoglobinuria (PNH).
The aim of the study is to see how well the pozelimab and cemdisiran combination works to lower hemolysis in participants whose PNH has been not well controlled even after taking other complement component 5 (C5) inhibitors, eculizumab/eculizumab biosimilar, ravulizumab or crovalimab.
The study is looking at several other research questions, including:
- What side effects may happen from taking the study drugs?
- How much of the study drugs are in the blood at different times?
- Whether the body makes antibodies against the study drug (which could make the study drugs not work as well or could lead to side effects)
详细描述
The treatment period has two parts, a Treatment Period (TP, 28 weeks) and an Extension treatment Period (EP, 52 weeks).
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
- •Diagnosis of PNH confirmed by a history of high-sensitivity flow cytometry from prior testing
- •Currently treated with marketed eculizumab, ravulizumab, or crovalimab at the labeled dose for at least 6 months
- •LDH persistently > 1.5 × Upper Limit of Normal (ULN) in the previous 6 months that the Principal Investigator (PI) attributes is due to intravascular hemolysis
- •At least 2 screening LDH values from different visits as described in the protocol
- •Willing and able to comply with clinic/remote visits and study-related procedures, including completion of the full series of meningococcal vaccinations required per protocol and agreement to continue to remain up to date with these vaccinations during the study
排除标准
- •Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplants
- •Body weight <40 kilograms at screening visit
- •Patients with a known or suspected C5 mutation that is refractory to their current C5i treatment as described in the protocol
- •Any active or ongoing infection within 2 weeks of screening or during the screening period or any recent infection as described in the protocol
- •Known hereditary complement deficiency
- •Note: Other protocol-defined Inclusion/ Exclusion Criteria apply
研究组 & 干预措施
Pozelimab + Cemdisiran Combo
Experimental
干预措施: Cemdisiran (Drug)
Pozelimab + Cemdisiran Combo
Experimental
干预措施: Pozelimab (Drug)
结局指标
主要结局
Percent change in Lactate Dehydrogenase (LDH) during TP
时间窗: From baseline to week 28
次要结局
- Change in hemoglobin from baseline(Through week 52)
- Normalization of LDH(Through week 52)
- Transfusion avoidance(Through week 52)
- Hemoglobin stabilization(Through week 52)
- Change in fatigue(Through week 52)
- Severity of all AEs(Through week 52)
- Occurrence of all Treatment-Emergent Adverse Events (TEAEs)(Through week 52)
- Severity of all TEAEs(Through week 52)
- Change from baseline in Total Complement Hemolytic Activity Assay (CH50)(Through week 52)
- Concentrations of total pozelimab(Through week 52)
- Concentrations of cemdisiran(Through week 52)
- Concentrations of total C5(Through week 52)
- Incidence of ADA to cemdisiran(Through week 52)
- Percent change in LDH during EP(From baseline to week 24 and week 52)
- Incidence of Anti-Drug Antibody (ADA) to pozelimab(Through week 52)
- Magnitude of ADA to pozelimab(Through week 52)
- Incidence of ADA to cemdisiran(Through week 52)
- Magnitude of ADA to cemdisiran(Through week 52)
- Percent change in LDH during EP(From baseline to week 24 and week 52)
- Concentrations of total pozelimab(Through week 52)
- Concentrations of cemdisiran(Through week 52)
- Concentrations of total C5(Through week 52)
- Normalization of LDH(Through week 52)
- Adequate control of hemolysis (LDH ≤1.5 × ULN)(Through week 52)
- Transfusion avoidance(Through week 52)
- Hemoglobin stabilization(Through week 52)
- Change in hemoglobin from baseline(Through week 52)
- Change in fatigue(Through week 52)
- Occurrence of all Adverse Events (AEs)(Through week 52)
- Severity of all AEs(Through week 52)
- Occurrence of all Treatment-Emergent Adverse Events (TEAEs)(Through week 52)
- Severity of all TEAEs(Through week 52)
- Change from baseline in Total Complement Hemolytic Activity Assay (CH50)(Through week 52)
研究者
研究点 (20)
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