Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab
Study Identifier:
ACH471-101
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 (PNH)
Study Drug
- Drug: Danicopan
- Drug: Eculizumab
Date
May 2018 - Sep 2019
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18 - 65 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Diagnosed with PNH
- Have received at least one red blood cell transfusion within last 12 weeks
- Anemia with adequate reticulocytosis
- Must be on a stable regimen of eculizumab
- Platelet count ≥ 40,000/microliter without the need for platelet transfusions
- Documentation of vaccination for Neisseria meningitidis, Haemophilus influenza, and Streptococcus pneumoniae or willingness to receive vaccinations based on local guidelines
- Willingness to receive antibiotic prophylaxis
- Female participants must use highly effective birth control to prevent pregnancy during the clinical trial and for 30 days after their last dose of study drug
- Male participants must use a highly effective birth control with a female partner to prevent pregnancy during the clinical trial and for 90 days after the last dose of study drug
Exclusion Criteria
- Current evidence of bone marrow failure or aplastic anemia requiring treatment
- History of a major organ transplant or hematopoietic stem cell/marrow transplant
- Received another investigational agent within 30 days or 5 half-lives of the investigational agent prior to study entry, whichever is greater
- Documented C5 complement protein mutations
- Known or suspected complement deficiency
- Contraindication to any of the required vaccinations
- Active bacterial infection or clinically significant active viral infection, a body temperature \>38°C, or other evidence of infection
- History of meningococcal infection, or a first-degree relative or household contact with a history of meningococcal infection
- History of hypersensitivity reactions to commonly used antibacterial agents
- Note: Additional inclusion/exclusion criteria may apply, per protocol.
Sex
Female & Male
Age
18 - 65 Years
Study Details
Medical Condition
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study Drug
- Drug: Danicopan
- Drug: Eculizumab
Date
May 2018 - Sep 2019
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18 - 65 Years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Diagnosed with PNH
- Have received at least one red blood cell transfusion within last 12 weeks
- Anemia with adequate reticulocytosis
- Must be on a stable regimen of eculizumab
- Platelet count ≥ 40,000/microliter without the need for platelet transfusions
- Documentation of vaccination for Neisseria meningitidis, Haemophilus influenza, and Streptococcus pneumoniae or willingness to receive vaccinations based on local guidelines
- Willingness to receive antibiotic prophylaxis
- Female participants must use highly effective birth control to prevent pregnancy during the clinical trial and for 30 days after their last dose of study drug
- Male participants must use a highly effective birth control with a female partner to prevent pregnancy during the clinical trial and for 90 days after the last dose of study drug
Exclusion Criteria
- Current evidence of bone marrow failure or aplastic anemia requiring treatment
- History of a major organ transplant or hematopoietic stem cell/marrow transplant
- Received another investigational agent within 30 days or 5 half-lives of the investigational agent prior to study entry, whichever is greater
- Documented C5 complement protein mutations
- Known or suspected complement deficiency
- Contraindication to any of the required vaccinations
- Active bacterial infection or clinically significant active viral infection, a body temperature \>38°C, or other evidence of infection
- History of meningococcal infection, or a first-degree relative or household contact with a history of meningococcal infection
- History of hypersensitivity reactions to commonly used antibacterial agents
- Note: Additional inclusion/exclusion criteria may apply, per protocol.
Protocol Summary
To determine the effectiveness of ACH-0144471 (also known as danicopan and ALXN2040) in improving anemia when given with eculizumab for 24 weeks in participants with PNH. Danicopan dose may be increased within each participant, to a maximum of 200 milligrams (mg) three times daily (TID) based on safety and efficacy at protocol-specified time points.
Trial Locations
Location
Status
Location
Clinical Study Site
Baltimore, Maryland, United States, 21287
Status
N/A
Location
Clinical Study Site
Cleveland, Ohio, United States, 44195
Status
N/A
Location
Clinical Study Site
Florence, Italy
Status
N/A
Location
Clinical Study Site
Naples, Italy
Status
N/A
Location
Clinical Study Site
London, United Kingdom
Status
N/A