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 Inc.0 个研究点目标入组 84 人开始时间: 2020年1月23日最近更新: 2024年2月19日

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•Diagnosis of PNH
•Clinically evident extravascular hemolysis (EVH) defined by:
•- Anemia (Hgb =9.5 gram/deciliter) with absolute reticulocyte count =120 x 10^9/liter.
•Receiving a C5 inhibitor for at least 6 months prior to Day 1
•Platelet count =30,000/microliters (µL)
•Absolute neutrophil counts =500/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 59
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 27

•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, unless the dosage regimen of immunosuppressant has been stable for at least 12 weeks before Day 1 and patient is expected to remain on stable doses through Week 24.
•Known or suspected complement deficiency.
•Laboratory abnormalities at screening, including:
•- Alanine aminotransferase >2 x ULN (> 3 x ULN in the case of patients with documented liver iron overload defined by serum ferritin values = 500 ng/mL). The inclusion of patients with documented iron overload and ALT>2 X ULN will be done in a case by case basis, with prior
•discussion with the Medical Monitor.
•- Direct bilirubin >2 x ULN - patients who, in the opinion of investigator, have direct bilirubin > 2 x ULN due to EVH and/or patients with documented Gilbert's Syndrome. If Gilbert's syndrome is
•suspected, the patient will be tested for this condition at screening.
•Current evidence of biliary cholestasis.
•Estimated glomerular filtration rate <30 milliliters/minute/1.73 meter squared and/or are on dialysis.
•Evidence of human immunodeficiency virus, hepatitis B, or active hepatitis C infection at screening.

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