临床试验/2023-504867-18-00

2023-504867-18-00招募中3 期

A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated with Danicopan in an Alexion-sponsored Clinical Study.

Alexion Pharmaceuticals Inc., Alexion Pharmaceuticals Inc.18 个研究点分布在 6 个国家目标入组 25 人开始时间: 2024年7月4日最近更新: 2025年3月21日

试验速览

阶段: 3 期
状态: 招募中
发起方
入组人数: 25
试验地点: 18
主要终点: Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs

研究概览入排标准结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

To characterize the long-term safety of treatment with danicopan as an add-on therapy to a complement component 5 inhibitor (C5i)

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: 是

入选标准

•All participants who completed their participation in an Alexion sponsored clinical study with danicopan as an add on to a C5i treatment
•Documentation of vaccination for Neisseria meningitidis: All participants must be revaccinated as per national vaccination guidelines or local practice for vaccination use with complement inhibitors.

排除标准

•Any medical condition (for example, cardiac, pulmonary, renal, oncologic, or psychiatric) that, in the opinion of the Investigator, might interfere with participation in the study, pose any added risk to the participant, or confound the assessment of the participant.
•Female participants who are pregnant, breastfeeding, or intending to conceive during the course of the study.

结局指标

主要结局

Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs

次要结局

Change in hemoglobin (Hgb) values over time
Proportion of patients with Hgb increase of ≥ 2g/dL in the absence of transfusion over time
Change in absolute reticulocyte count over time
Change in lactate dehydrogenase (LDH) over time
Proportion of patients with LDH ≤ 1.5 × upper limit of normal (ULN) over time
Proportion of patients with transfusion avoidance (TA), defined as patients who remain transfusion free and do not require transfusion as per protocol specified guidelines
Change in FACIT Fatigue scores over time
Change in EORTC-QLQ-C30 scores over time
Change in safety laboratory parameters over time
TEAEs leading to discontinuation

研究者

发起方

Alexion Pharmaceuticals Inc., Alexion Pharmaceuticals Inc.

申办方类型

Pharmaceutical company, Pharmaceutical company

责任方

Principal Investigator

主要研究者

European Clinical Trial Information

Scientific

Alexion Pharmaceuticals Inc.

研究点 (18)

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CTR2024345878