Actively Recruiting
A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis
Led by Alexion Pharmaceuticals, Inc. · Updated on 2025-12-23
6
Participants Needed
4
Research Sites
41 weeks
Total Duration
On this page
Sponsors
A
Alexion Pharmaceuticals, Inc.
Lead Sponsor
A
AstraZeneca
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating danicopan as an add-on treatment to ravulizumab or eculizumab in pediatric participants aged 12 to 17 years who have paroxysmal nocturnal hemoglobinuria (PNH) with clinically significant extravascular hemolysis (CS-EVH). The main goal is to assess the change in hemoglobin levels from the start of the study to Week 12, focusing on how adding danicopan affects this blood condition. Participants receive danicopan based on their weight during a 12-week open-label treatment period, followed by up to a one-year open-label long-term extension. The study does not include randomization or blinding, and all participants continue their stable dose of ravulizumab or eculizumab throughout the trial. During the study, researchers monitor hemoglobin changes, reticulocyte counts, plasma danicopan levels, quality of life, fatigue, and other blood markers at various points up to Week 64. Safety and tolerability are followed closely through laboratory tests and questionnaires. The total participation includes the initial 12-week treatment and the optional extended follow-up period.
CONDITIONS
Brief Title
Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed diagnosis of paroxysmal nocturnal hemoglobinuria (PNH).
- Clinically significant extravascular hemolysis defined by hemoglobin 64; 11.0 g/dL and absolute reticulocyte count 65; 100 �d7 10^9/L.
- Treated with ravulizumab or eculizumab for at least 12 weeks with a stable dose before Day 1 and no planned dose changes during the first 12 weeks.
- Vaccinated against meningococcal infection serogroups A, C, W, Y, and B within 3 years or at least 14 days before Day 1.
- Vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae.
You will not qualify if you...
- Platelet count less than 30,000/bcL or need for platelet transfusions.
- Absolute neutrophil count (ANC) less than 500/bcL.
- Clinically significant liver function abnormalities including ALT > 2 times upper limit of normal (ULN) or > 3 times ULN with liver iron overload, and direct bilirubin > 2 times ULN unless due to hemolysis or Gilbert's syndrome.
- Current evidence of biliary cholestasis.
- Known aplastic anemia or bone marrow failure requiring transplant or other therapies unless immunosuppressant dose is stable for 12 weeks prior and expected stable through Week 12.
- History of major organ transplant or hematopoietic stem cell transplant (HSCT).
- Known or suspected complement deficiency.
- Active bacterial or viral infection, fever over 38�b0C on 2 consecutive days, or febrile illness within 14 days before starting the study intervention.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 12 weeks
Participants receive a 12-week weight-based open-label treatment with danicopan as add-on to their stable ravulizumab or eculizumab regimen.
Regular visits during the 12-week treatment period
Duration - Up to 1 year
Participants who complete the initial treatment may continue danicopan treatment in an open-label long-term extension period for up to 1 year.
Visits scheduled during the extension period
Trial Site Locations
Total: 4 locations
1
Research Site
Saskatoon, Saskatchewan, Canada, S7N 0W8
Actively Recruiting
2
Research Site
Paris, France, 77019
Actively Recruiting
3
Research Site
Leeds, United Kingdom, LS9 7TF
Actively Recruiting
4
Research Site
London, United Kingdom, SE5 9RS
Actively Recruiting
Research Team
A
Alexion Pharmaceuticals, Inc. (Sponsor)
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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