Status:

RECRUITING

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

Lead Sponsor:

Alexion Pharmaceuticals, Inc.

Collaborating Sponsors:

AstraZeneca

Conditions:

Paroxysmal Nocturnal Hemoglobinuria

PNH

Eligibility:

All Genders

12-17 years

Phase:

PHASE3

Brief Summary

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 p...

Eligibility Criteria

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.

Key Trial Info

Start Date :

August 11 2025

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

March 10 2028

Estimated Enrollment :

6 Patients enrolled

Trial Details

Trial ID

NCT06449001

Start Date

August 11 2025

End Date

March 10 2028

Last Update

December 23 2025

Active Locations (4)

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Page 1 of 1 (4 locations)

1

Research Site

Saskatoon, Saskatchewan, Canada, S7N 0W8

2

Research Site

Paris, France, 77019

3

Research Site

Leeds, United Kingdom, LS9 7TF

4

Research Site

London, United Kingdom, SE5 9RS