Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)
Study Identifier:
ALXN2040-PNH-301
ClinicalTrials.gov Identifier:
EudraCT Identifier:
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete
Available Documents
Study Details
Medical Condition
- Paroxysmal Nocturnal Hemoglobinuria
Study Drug
- Drug: Danicopan
- Drug: Placebo
- Drug: C5 Inhibitor
Date
Dec 2020 - Jun 2022
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18+ years
Requirements Information
Inclusion and Exclusion 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
- 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
- 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
Sex
Female & Male
Age
18+ years
Study Details
Medical Condition
- Paroxysmal Nocturnal Hemoglobinuria
Study Drug
- Drug: Danicopan
- Drug: Placebo
- Drug: C5 Inhibitor
Date
Dec 2020 - Jun 2022
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18+ years years
Requirements Information
Inclusion and Exclusion 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
- 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
- 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
Protocol 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.
Trial Locations
Location
Status
Location
Research Site
Los Angeles, California, United States, 90089
Status
N/A
Location
Research Site
Weston, Florida, United States, 33331
Status
N/A
Location
Research Site
Chicago, Illinois, United States, 60612
Status
N/A
Location
Research Site
Kalamazoo, Michigan, United States, 49007
Status
N/A
Location
Research Site
New York, New York, United States, 10065
Status
N/A
Location
Research Site
Dallas, Texas, United States, 75390
Status
N/A
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