Actively Recruiting
Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis
Led by Alexion Pharmaceuticals, Inc. · Updated on 2025-12-23
6
Participants Needed
4
Research Sites
134 weeks
Total Duration
On this page
Sponsors
A
Alexion Pharmaceuticals, Inc.
Lead Sponsor
A
AstraZeneca
Collaborating Sponsor
AI-Summary
What this Trial Is About
The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH).
CONDITIONS
Official 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 ≤ 11.0 g/dL and absolute reticulocyte count ≥ 100 × 10^9/L.
- Treatment with ravulizumab or eculizumab for at least 12 weeks immediately before Day 1, with stable dosing and no planned changes during the first 12 weeks.
- Vaccinated against meningococcal infection from serogroups A, C, W, Y, and B within 3 years before 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 30000/μL or need for platelet transfusions.
- Absolute neutrophil count (ANC) less than 500/μL.
- Significant liver function abnormalities including ALT > 2 × upper limit of normal (ULN), or ALT > 3 × ULN with documented liver iron overload.
- Direct bilirubin > 2 × ULN unless due to hemolysis or Gilbert's syndrome.
- Current evidence of biliary cholestasis.
- Known aplastic anemia or other bone marrow failure requiring hematopoietic stem cell transplant (HSCT) or other therapies, unless immunosuppressant dosage is stable for at least 12 weeks before Day 1 and expected to remain stable.
- History of major organ transplant or HSCT.
- Known or suspected complement deficiency.
- Active bacterial or viral infection, fever over 38°C on two consecutive days, other infection evidence, or febrile illness within 14 days prior to first study intervention.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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)
CONTACT
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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