Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)

• Status: Recruiting
• Trial end date: 2023-12-01
• Target enrollment: 0
• Participant gender: All

Summary

The main objective of this study is to evaluate the efficacy of danicopan as add-on therapy to a complement component 5 (C5) inhibitor (eculizumab or ravulizumab) in participants with PNH who have clinically evident EVH.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alexion Pharmaceuticals

Criteria

Inclusion Criteria:

• 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 for N. meningiditis and prophylactic antibiotics as required

Exclusion Criteria:

• 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:

• 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

• Evidence of human immunodeficiency virus, hepatitis B, or active hepatitis C infection at screening