Status:
COMPLETED
Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Paroxysmal Nocturnal Hemoglobinuria
Eligibility:
All Genders
18+ years
Phase:
PHASE3
Brief 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 cli...
Detailed Description
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). P...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
December 16 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 16 2024
Estimated Enrollment :
86 Patients enrolled
Trial Details
Trial ID
NCT04469465
Start Date
December 16 2020
End Date
January 16 2024
Last Update
May 4 2025
Active Locations (62)
Enter a location and click search to find clinical trials sorted by distance.
1
Research Site
Los Angeles, California, United States, 90089
2
Research Site
Weston, Florida, United States, 33331
3
Research Site
Chicago, Illinois, United States, 60612
4
Research Site
Kalamazoo, Michigan, United States, 49007