ClinicalTrials.gov record NCT06449001

Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
Age12 years old to 17 years old
SexAll
LocationCanada, France, United Kingdom
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 3 open-label study tests danicopan added to ravulizumab or eculizumab in children with PNH who have clinically significant extravascular hemolysis. The main measure is change in hemoglobin at Week 12. Participants receive weight-based danicopan for a 12-week treatment period, with an available longer-term extension.

What it is studying. Whether adding danicopan to ongoing ravulizumab or eculizumab affects hemoglobin levels at Week 12 in pediatric participants with PNH and clinically significant extravascular hemolysis. The study also collects data on danicopan blood levels, transfusion avoidance, reticulocyte counts, patient-reported quality of life and fatigue, acceptability/palatability, and markers of alternative complement pathway activity.

What the researchers hope to learn. To evaluate efficacy of danicopan as an add-on treatment by measuring change in hemoglobin at Week 12. Secondary goals include measuring danicopan plasma concentrations, transfusion avoidance, changes in reticulocyte count, patient-reported quality-of-life and fatigue, acceptability/palatability, and changes in alternative pathway activity through Week 64.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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).

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Danicopan

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Confirmed diagnosis of PNH.
  • CS-EVH defined by: Anemia: Hgb ≤ 11.0 g/dL, and absolute reticulocyte count ≥ 100 × 109/L
  • Treated with ravulizumab or eculizumab for at least 12 weeks immediately preceding Day 1, the dose received should be stable during this period, and there should be no anticipated changes in dosage or interval during the first 12 weeks of this study.
  • all participants must be vaccinated against meningococcal infection from serogroups A, C, W, and Y and serogroup B within 3 years prior to, or at least 14 days prior to Day 1
  • vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae

Exclusion criteria

  • Platelet count < 30000/μL or there is a need for platelet transfusions.
  • ANC < 500/μL.
  • Clinically significant laboratory abnormalities related to liver function, including:
  • ALT > 2 × ULN or ALT > 3 × ULN for participants with documented liver iron overload defined by serum ferritin values ≥ 500 ng/mL.
  • Direct bilirubin > 2 × ULN, unless, in the Investigator's opinion, is due to hemolysis or Gilbert's syndrome based on medical history.
  • Current evidence of biliary cholestasis.
  • Known aplastic anemia or other bone marrow failure that requires HSCT or other therapies, including anti-thymocyte globulin and immunosuppressants unless the dosage of immunosuppressant has been stable for at least 12 weeks before Day 1 and is expected to remain stable through Week 12.
  • History of a major organ transplant (eg, heart, lung, kidney, liver) or HSCT.
  • Known or suspected complement deficiency.
  • Active bacterial or viral infection, a body temperature > 38°C on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to first study intervention administration.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants will receive danicopan as an add-on, using a weight-based dosing regimen for a 12-week open-label treatment period, with an option to continue in a longer open-label extension of up to about 1 year. They will have blood tests to measure hemoglobin, reticulocytes, liver tests and some drug-related blood measures (including a drug concentration measure early on), and will complete questionnaires on quality of life and fatigue; an acceptability/palatability questionnaire is done around Week 2. The study will track transfusion avoidance through Weeks 12 and 24 and some laboratory measures up to Week 64, so participants should expect clinic visits and blood sampling over that time. Vaccination against meningococcal A/C/W/Y and B, Hib, and pneumococcus is required before starting per eligibility rules.

How the study is structured. Phase 3, open-label, non-randomized interventional study with a single treatment group. Children already receiving ravulizumab or eculizumab are given weight-based danicopan for 12 weeks; an open-label extension of up to about 1 year is available. There is no placebo or masking. Participants undergo blood tests, pharmacokinetic sampling, questionnaires, and follow-up for selected biomarkers.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in Canada, France, United Kingdom. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on paroxysmal nocturnal hemoglobinuria

Clinical trials44 trials listed for paroxysmal nocturnal hemoglobinuriaEvery trial the registry lists for paroxysmal nocturnal hemoglobinuria, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in paroxysmal nocturnal hemoglobinuria researchLatest monthly update: September 2026. Includes: Treatment access. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Aug 11, 2025
Primary completion
May 28, 2027 (estimated)
Study completion
Mar 10, 2028 (estimated)
Target enrollment
6 participants (estimated)
Last updated on registry
Dec 23, 2025
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Alexion Pharmaceuticals, Inc.
Collaborators
  • AstraZeneca

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (4)

  • Research SiteRecruiting

    Saskatoon, Saskatchewan, S7N 0W8 · Canada

  • Research SiteRecruiting

    Paris, 77019 · France

  • Research SiteRecruiting

    Leeds, LS9 7TF · United Kingdom

  • Research SiteRecruiting

    London, SE5 9RS · United Kingdom

Terms used on this page · explanations by MedicalUpdates.ai

primary objective
The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
Baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
ANC
The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
× ULN
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
HSCT
A procedure that replaces blood-forming cells after high-dose treatment. 'Autologous' uses the person's own cells collected beforehand; 'allogeneic' uses cells from a donor.
immunosuppressants
A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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