2024-519709-37-00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
Regeneron Pharmaceuticals Inc.7 个研究点 分布在 3 个国家目标入组 8 人开始时间: 2025年10月21日最近更新:
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 8
- 试验地点
- 7
- 主要终点
- Percent change in Lactate Dehydrogenase (LDH) during treatment period (TP) from baseline to week 28.
研究概览
简要总结
The primary objective of the study is to evaluate the effect of pozelimab + cemdisiran combination treatment on intravascular hemolysis (IVH) in patients with inadequate control of IVH on a currently available C5i treatment.
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 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.
- •Note: Other protocol-defined Inclusion Criteria apply.
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 Exclusion Criteria apply.
结局指标
主要结局
Percent change in Lactate Dehydrogenase (LDH) during treatment period (TP) from baseline to week 28.
Percent change in Lactate Dehydrogenase (LDH) during treatment period (TP) from baseline to week 28.
次要结局
- Adequate control of hemolysis through week 52.
- Normalization of LDH through week 52.
- Transfusion avoidance through week 52.
- Hemoglobin stabilization through week 52.
- Change in hemoglobin through week 52.
- Change in fatigue through week 52, as measured by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale.
- 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.
- Concentrations of total pozelimab through week 52.
- Concentrations of cemdisiran through week 52.
- Concentrations of total C5 through week 52.
- Incidence of anti-drug antibody (ADA) to pozelimab through week 52.
- Titer of ADA to pozelimab though week 52.
- Incidence of ADA to cemdisiran through week 52.
- Titer of ADA to cemdisiran through week 52.
- Percent change in LDH during extension treatment period (EP) from baseline to week 24 and week 52.
研究者
Medical Affairs
Scientific
Regeneron Pharmaceuticals Inc.
研究点 (7)
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