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

Completed · Phase 3 · Has a placebo group

Conditions studied: Paroxysmal Nocturnal Hemoglobinuria

In brief

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.

Key facts

Study ID

NCT04469465

Run by

Alexion Pharmaceuticals, Inc.

People needed

86

Starts

2020-12-16

Expected to finish

2024-01-16

Last updated by the study team

2025-05-04

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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
• 500 ng/ML)
• Direct bilirubin >2 x ULN (unless due to EVH or documented Gilbert's Syndrome)
• Current evidence of biliary cholestasis
• Estimated glomerular filtration rate of <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

Where it is running

• Research Site — Weston, Florida, United States
• Research Site — Chicago, Illinois, United States
• Research Site — Kalamazoo, Michigan, United States
• Research Site — New York, New York, United States
• Research Site — Dallas, Texas, United States
• Research Site — Milwaukee, Wisconsin, United States
• Research Site — Belém, Brazil
• Research Site — Curitiba, Brazil
• Research Site — Goiânia, Brazil
• Research Site — Porto Alegre, Brazil
• Research Site — Rio de de Janeiro, Brazil
• Research Site — Toronto, Ontario, Canada
• Research Site — Brno, Czechia
• Research Site — Lille, France
• Research Site — Paris, France
• Research Site — Pessac, France
• Research Site — Pierre-Bénite, France
• Research Site — Ulm, Germany
• Research Site — Athens, Greece
• Research Site — Thessaloniki, Greece
• Research Site — Haifa, Israel
• Research Site — Jerusalem, Israel
• Research Site — Avellino, Italy
• Research Site — Bassano del Grappa, Italy
• Research Site — Los Angeles, California, United States

Full record on ClinicalTrials.gov

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