Status:
COMPLETED
Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Collaborating Sponsors:
Achillion, a wholly owned subsidiary of Alexion
Conditions:
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Eligibility:
All Genders
18-65 years
Phase:
PHASE2
Brief 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 increas...
Detailed Description
The purpose of this study is to determine the effectiveness of danicopan in improving anemia, as measured by increased blood hemoglobin, when given with eculizumab (a drug commonly used for treatment ...
Eligibility Criteria
Inclusion
- Key
- 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
- Key
Exclusion
- 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.
Key Trial Info
Start Date :
May 8 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 5 2023
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT03472885
Start Date
May 8 2018
End Date
January 5 2023
Last Update
December 12 2023
Active Locations (5)
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1
Clinical Study Site
Baltimore, Maryland, United States, 21287
2
Clinical Study Site
Cleveland, Ohio, United States, 44195
3
Clinical Study Site
Florence, Italy
4
Clinical Study Site
Naples, Italy