A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) - .

ALEXION PHARMACEUTICALS INCORPORATED0 个研究点目标入组 84 人开始时间: 2021年5月24日最近更新: 2021年6月7日

适应症

试验速览

暂无简介。

•Diagnosis of PNH
•Clinically Evident EVH defined by:
•- Anemia (Hgb =9.5 gram/deciliter) with absolute reticulocyte count =120 x 10^9/liter.
•- At least 1 packed red blood cell or whole blood transfusion within 6 months prior to the start of the study.
•Receiving a C5 inhibitor for at least 6 months prior to Day 1
•Platelet count =30,000/microliters (µL)
•Absolute neutrophil counts =750/µL.
•Documentation of/or willingness to receive vaccinations and prophylactic antibiotics as required.
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 82
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 2

•History of a major organ transplant or hematopoietic stem cell transplantation (HSCT).
•Participants with known aplastic anemia or other bone marrow failure that requires HSCT or other therapies including anti-thymocyte globulin and/or immunosuppressants.
•Known or suspected complement deficiency.
•Laboratory abnormalities at screening, including:
•- Alkaline phosphatase >2 x upper limit of normal (ULN).
•- Alanine aminotransferase >2 x ULN.
•- Direct bilirubin >2 x ULN (unless due to EVH or documented Gilbert’s Syndrome.
•Current evidence of biliary cholestasis.
•Estimated glomerular filtration rate <30 milliliters/minute/1.73 meter squared and/or are on dialysis.

发起方

ALEXION PHARMACEUTICALS INCORPORATED