Danicopan for Paroxysmal Nocturnal Hemoglobinuria
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a treatment called danicopan to see if it helps children with paroxysmal nocturnal hemoglobinuria (PNH), a condition where red blood cells break down too easily, causing anemia. The goal is to determine if adding danicopan to their current medication, either ravulizumab or eculizumab, can improve hemoglobin levels, which are crucial for carrying oxygen in the blood. Children who have been receiving either of these medications for at least 12 weeks and still experience low hemoglobin despite treatment might be suitable candidates. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.
Do I have to stop taking my current medications for the trial?
The trial requires that you continue taking ravulizumab or eculizumab, as you must have been on a stable dose for at least 12 weeks before starting the trial and continue with no changes during the first 12 weeks of the study.
Is there any evidence suggesting that danicopan is likely to be safe for humans?
Research has shown that danicopan, when combined with treatments like ravulizumab or eculizumab for paroxysmal nocturnal hemoglobinuria (PNH), is generally safe. In earlier studies, patients using danicopan did not report any new or unusual side effects. The treatment has been used for a long time without unexpected issues. Some individuals experienced mild side effects, but these were not serious.
Since this study is in an advanced phase, earlier safety tests have been successful. Danicopan is already approved for other uses, suggesting its safety. Overall, evidence indicates that danicopan is well-tolerated by patients over long periods.12345Why do researchers think this study treatment might be promising for PNH?
Danicopan is unique because it targets the alternative pathway of the complement system, which is a different approach compared to most existing treatments for Paroxysmal Nocturnal Hemoglobinuria (PNH) that primarily focus on the terminal complement pathway. This new mechanism of action has the potential to provide better control over the disease by addressing a different part of the immune system's malfunction. Researchers are excited about Danicopan because it could offer a more comprehensive treatment option and possibly improve outcomes for patients who may not fully respond to current therapies like eculizumab and ravulizumab.
What evidence suggests that danicopan might be an effective treatment for paroxysmal nocturnal hemoglobinuria?
Research shows that danicopan can help manage paroxysmal nocturnal hemoglobinuria (PNH) when combined with treatments like ravulizumab or eculizumab. Studies have found that danicopan improves anemia by raising hemoglobin levels. In past patients, this combination successfully controlled the disease and managed symptoms over time. A review of research supports danicopan as a promising option for PNH patients who still experience anemia despite other treatments. These findings suggest that danicopan could significantly aid those dealing with this condition. Participants in this trial will receive danicopan during a 12-week weight-based open-label treatment period, with the option to continue in an open-label long-term extension period.24678
Are You a Good Fit for This Trial?
This trial is for pediatric patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are already being treated with ravulizumab or eculizumab. They must have stable doses of these drugs, be vaccinated against certain infections, and show signs of anemia and high reticulocyte counts indicating significant red blood cell breakdown.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive danicopan as an add-on treatment to ravulizumab or eculizumab for 12 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
Participants may continue to receive danicopan for up to 1 year
What Are the Treatments Tested in This Trial?
Interventions
- Danicopan
Find a Clinic Near You
Who Is Running the Clinical Trial?
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
AstraZeneca
Industry Sponsor
Sir Pascal Soriot
AstraZeneca
Chief Executive Officer since 2012
Veterinary Medicine from École nationale vétérinaire d'Alfort, MBA from HEC Paris
Dr. Cristian Massacesi
AstraZeneca
Chief Medical Officer since 2021
MD from Marche Polytechnic University, Oncology training at Royal Marsden Hospital, Kaplan Comprehensive Cancer Center, and European Institute of Oncology
Pascal Soriot
AstraZeneca
Chief Executive Officer since 2012
Veterinary Medicine from École nationale vétérinaire d'Alfort, MBA from HEC Paris
Cristian Massacesi
AstraZeneca
Chief Medical Officer since 2021
MD from Marche Polytechnic University, Medical Oncology training at Royal Marsden Hospital, Kaplan Comprehensive Cancer Center, and European Institute of Oncology