Early Access Program (EAP) for Danicopan as Add-on Treatment to SOLIRIS® or ULTOMIRIS® in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Clinically Significant Extravascular Hemolysis (csEVH)

Alexion Pharmaceuticals, Inc.15 个研究点分布在 1 个国家开始时间: 2023年8月9日最近更新: 2026年6月4日

适应症

试验速览

阶段: 不适用
状态: Available
发起方: Alexion Pharmaceuticals, Inc.
试验地点: 15

研究概览入排标准研究者研究点记录与标识符相似试验

研究概览

简要总结

This is an EAP, designed to provide early access to danicopan for participants with PNH experiencing clinically significant EVH who have failed, not tolerated, or are unable to receive other approved treatments; in the Treating Physician's opinion, the participant is not eligible for or is not able to participate in an ongoing clinical trial of danicopan or a comparable treatment; and according to the Treating Physician, the benefit of danicopan treatment outweighs the potential risks.

研究设计

研究类型: Expanded Access

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

•Male or female, aged 18 years and older
•Diagnosed with PNH with clinically significant EVH as defined by anemia (Hgb ≤ 9.5 g/dL) with absolute reticulocyte count ≥ 120 × 109/L.
•Currently receiving treatment with SOLIRIS® or ULTOMIRIS® for at least 6 months prior to initiation of danicopan treatment.
•Platelet count ≥ 30,000/µL without the need for platelet transfusions.
•Absolute neutrophil counts ≥ 500/µL.
•Vaccinated against N meningitidis.

排除标准

•Participants who are nursing or pregnant (or women who are planning to become pregnant during treatment with danicopan).
•Laboratory abnormalities at screening, as follows:
•Alanine aminotransferase (ALT) > 2 × upper limit of normal (ULN)
•Direct bilirubin > 2 × ULN, with the exception of:
•participants with increased bilirubin due to EVH in the opinion of the Treating Physician or
•participants with documented Gilbert's syndrome
•Active bacterial or viral infection, a body temperature > 38°C on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to initiating danicopan treatment.

研究者

发起方

Alexion Pharmaceuticals, Inc.

申办方类型

Industry

责任方

Sponsor

研究点 (15)

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