For Healthcare Professionals

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


About the study

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.

Who can take part

You may be eligible to participate in the study if you meet the following criteria:


Inclusion Criteria:

Diagnosis of PNH

Clinically Evident EVH defined by:

  1. Anemia (Hgb ≤9.5 gram/deciliter) with absolute reticulocyte count ≥120 x 10^9/liter
  2. Receiving an approved C5 inhibitor for at least 6 months prior to Day 1
  3. Platelet count ≥30,000/microliters (µL)
  4. Absolute neutrophil counts ≥500/μL
  5. Documentation of/or willingness to receive vaccinations for N. meningiditis and prophylactic antibiotics as required


Exclusion Criteria:

  1. History of a major organ transplant or hematopoietic stem cell transplantation (HSCT)
  2. Participants with known aplastic anemia or other bone marrow failure that requires HSCT or other therapies including anti-thymocyte globulin and/or immunosuppressants
  3. Known or suspected complement deficiency

Laboratory abnormalities at screening, including:

Alanine aminotransferase >2 x ULN (>3 x ULN in case of patients with documented liver iron overload defined by serum ferratin values

  1. 500 ng/ML)
  2. Direct bilirubin >2 x ULN (unless due to EVH or documented Gilbert's Syndrome)
  3. Current evidence of biliary cholestasis
  4. Estimated glomerular filtration rate of <30 milliliters/minute/1.73 meter squared and/or are on dialysis
  5. Evidence of human immunodeficiency virus, hepatitis B, or active hepatitis C infection at screening

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Study Locations

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

How to Apply

Contact the study center to learn if this study is a good match for you.

Study’s details


Paroxysmal Nocturnal Hemoglobinuria

Age (in years)



Phase 3

Participants needed


Est. Completion Date

Dec 31, 2023

Treatment type



Alexion Pharmaceuticals, Inc. identifier


Study number


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