Recruiting
Phase 3

Danicopan with Ravulizumab & Eculizumab

Sponsor:

Alexion Pharmaceuticals, Inc.

Code:

NCT06449001

Conditions

Paroxysmal Nocturnal Hemoglobinuria

PNH

Extravascular Hemolysis

Eligibility Criteria

Sex: All

Age: 12 - 17

Healthy Volunteers: Not accepted

Interventions

Danicopan

Study Details

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 participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH).

Conditions

Paroxysmal Nocturnal Hemoglobinuria

PNH

Extravascular Hemolysis

Study ID

NCT06449001

Start date

Aug 11, 2025

Status verified date

Dec, 2025

Completion date

Mar 10, 2028

Anticipated

Primary completion date

May 28, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 12 - 17

Healthy Volunteers: Not accepted

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 Design

Enrollment

6 participants

Anticipated

Allocation

Non randomized

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Danicopan

Participants will receive a 12-week weight-based open-label treatment period and up to 1 year open-label long term extension period.

Interventions

Danicopan

Participants will receive danicopan on a weight-based dosing regimen.

Primary outcome measure

  • Change From Baseline in Hemoglobin (Hgb) Concentration at Week 12 [ Time Frame: Baseline, Week 12 ]

Central Contacts and Locations

Central contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

1-855-752-2356clinicaltrials@alexion.com

Locations

Research Site

Recruiting

Saskatoon, Saskatchewan, Canada, S7N 0W8

More Information

Sponsor

Alexion Pharmaceuticals, Inc.

Last update posted

Dec 23, 2025

Last verified

Dec, 2025

Keywords

  • Paroxysmal Nocturnal Hemoglobinuria
  • PNH
  • Extravascular Hemolysis
  • Ravulizumab
  • Eculizumab
  • Danicopan

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Alexion Pharmaceuticals, Inc. on 2025-12-23.