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

  • End date
    Dec 31, 2023
  • participants needed
  • sponsor
    Alexion Pharmaceuticals
Updated on 25 April 2022
platelet count
blood transfusion


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.


This is a multiple-region, randomized, double-blind, placebo controlled, multiple-dose, study in participants with PNH who have clinically evident EVH on a C5 inhibitor (eculizumab or ravulizumab).

Participants will be randomized to receive danicopan or placebo, in a 2:1 ratio for 12 weeks (Treatment Period 1) in addition to their C5 inhibitor (eculizumab or ravulizumab) therapy. At Week 12, participants randomized to receive placebo will be switched to danicopan in addition to their C5 inhibitor for an additional 12 weeks (Treatment Period 2) and participants randomized to danicopan will continue on danicopan for an additional 12 weeks, while remaining on their ongoing C5 inhibitor therapy.

At the end of the 2 treatment periods (Week 24), participants may enter a Long-Term Extension (LTE) Period and continue to receive danicopan in addition to their C5 inhibitor therapy. The Long-Term Extension period will consist of a first year of LTE(Year1) and a second year of optional LTE(Year2).All patients will complete 72 weeks of LTE(Year 1) assessments. After Week 72 (at the end of the first year of LTE), patients have the choice to complete participation in this study or continue to the optional second year (Year2) of LTE.

Condition Paroxysmal Nocturnal Hemoglobinuria
Treatment Placebo, Danicopan, Danicopan, C5 Inhibitor
Clinical Study IdentifierNCT04469465
SponsorAlexion Pharmaceuticals
Last Modified on25 April 2022


Yes No Not Sure

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
Receiving an approved 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 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 (>3 x ULN in case of patients with documented liver iron overload defined by serum ferratin values
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
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