Danicopan Add-On Therapy for Paroxysmal Nocturnal Hemoglobinuria
Trial Summary
Do I need to stop my current medications for this trial?
The trial information does not specify if you need to stop taking your current medications. It seems likely that you can continue your existing treatment with C5i, as danicopan is an add-on therapy.
What data supports the effectiveness of the drug Danicopan for treating paroxysmal nocturnal hemoglobinuria?
In a study, patients with paroxysmal nocturnal hemoglobinuria who were already on eculizumab and received Danicopan showed a significant increase in hemoglobin levels and a reduction in the need for blood transfusions. This suggests that Danicopan can help improve blood health and reduce symptoms in these patients.12345
How does the drug Danicopan differ from other treatments for paroxysmal nocturnal hemoglobinuria?
Danicopan is unique because it is an oral drug that specifically inhibits complement factor D, a part of the immune system that contributes to the destruction of red blood cells in paroxysmal nocturnal hemoglobinuria (PNH). This approach helps control both intravascular and extravascular hemolysis, offering a novel mechanism compared to existing treatments.678910
What is the purpose of this trial?
This is a single-arm long-term extension study that will enroll participants with PNH who have completed participation in Alexion-sponsored clinical studies with danicopan as an add on therapy to a C5i.
Eligibility Criteria
This trial is for people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have finished a previous Alexion study using Danicopan as an extra treatment. They must be able to consent, follow the study rules, and be vaccinated against Neisseria meningitidis. Those with conditions that could affect the study or pose risks can't join, nor can women who are pregnant, breastfeeding, or planning pregnancy.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive danicopan as an add-on to a background C5i therapy, continuing from the parent study
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
Participants continue to receive danicopan as an add-on therapy to C5i for long-term safety and efficacy assessment
Treatment Details
Interventions
- Danicopan
Find a Clinic Near You
Who Is Running the Clinical Trial?
Alexion
Lead Sponsor
Marc Dunoyer
Alexion
Chief Executive Officer since 2021
PhD in Molecular Biology, University of Brussels
Christophe Hotermans
Alexion
Chief Medical Officer since 2021
MD, University of Leuven
Alexion Pharmaceuticals, Inc.
Lead Sponsor
Dr. Alberto R. Martinez
Alexion Pharmaceuticals, Inc.
Chief Medical Officer since 2010
MD from University of Sao Paulo
Marc Dunoyer
Alexion Pharmaceuticals, Inc.
Chief Executive Officer since 2021
PhD in Molecular Biology
Alexion Pharmaceuticals
Lead Sponsor
Marc Dunoyer
Alexion Pharmaceuticals
Chief Executive Officer since 2021
B.A. in Psychology from the University of New Hampshire
Christophe Hotermans
Alexion Pharmaceuticals
Chief Medical Officer since 2023
MD