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Looking for participantsPhase3

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

Sponsor: Alexion Pharmaceuticals, Inc.

NCT ID: NCT06449001

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
Danicopan (drug)
How long the study runs
Study runs about 31 months (dates as stated)
About the drug or intervention
Danicopan — drug: Participants will receive danicopan on a weight-based dosing regimen.
Patient visit burden
Not specified by the sponsor

In plain English

This study looks at danicopan as an add-on treatment for children with paroxysmal nocturnal haemoglobinuria (PNH), a rare blood condition. It is for children already treated with ravulizumab or eculizumab who still have clinically significant extravascular haemolysis, meaning their red blood cells are broken down outside the blood vessels. The study is sponsored by Alexion Pharmaceuticals, Inc.

Who can take part

  • A confirmed diagnosis of PNH
  • Anaemia with a haemoglobin level of 11.0 g/dL or less, and a certain level of young red blood cells (reticulocytes) in the blood
  • Already treated with ravulizumab or eculizumab for at least 12 weeks, on a stable dose with no expected dose changes in the first 12 weeks of the study
  • Vaccinated against meningococcal infection (types A, C, W, Y and B), either within the past 3 years or at least 14 days before the study starts
  • Vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae

Who may not be able to

  • A platelet count below 30,000 per microlitre, or needing platelet transfusions
  • A certain type of white blood cell count (ANC) below 500 per microlitre
  • Significant liver function problems on blood tests, unless explained by haemolysis or Gilbert's syndrome
  • Current evidence of a bile flow problem (cholestasis)
  • Known aplastic anaemia or other bone marrow failure needing a stem cell transplant or certain other treatments, unless on a stable dose of immunosuppressants for at least 12 weeks
  • Past major organ transplant (such as heart, lung, kidney or liver) or stem cell transplant
  • Known or suspected complement deficiency (a part of the immune system)
  • Active infection, fever above 38°C on 2 days in a row, or a feverish illness within 14 days before the first study treatment

What taking part involves

  • • Taking danicopan as an add-on to existing treatment with ravulizumab or eculizumab

Time commitment: Not stated — ask the trial team about how many visits there are, how long the study lasts, and what taking part involves.

Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.

Type of study
Testing a treatment
Ages
12 Years to 17 Years
Who
All
Number of participants
6
Started
2025-08-11
Last checked
2025-12

Plain English Summary

What is this study?

  • • Testing a new treatment for paroxysmal nocturnal hemoglobinuria
  • • Phase3 - 6 participants
  • • 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)

Who can take part?

  • • Ages 12 Years to 17 Years
  • • Diagnosed with paroxysmal nocturnal hemoglobinuria

Where?

  • • Leeds - Research Site
  • • London - Research Site

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

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).

Paroxysmal Nocturnal HemoglobinuriaPNHExtravascular Hemolysis

How this trial compares with your answers

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What we know so far

Condition· Matched your search
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Still need:

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Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 12 Years - 17 Years
  • Who can join: All genders

What the study is looking for

  • ✓Confirmed diagnosis of PNH.
  • ✓CS-EVH defined by: Anemia: Hgb ≤ 11.0 g/dL, and absolute reticulocyte count ≥ 100 × 109/L
  • ✓all participants must be vaccinated against meningococcal infection from serogroups A, C, W, and Y and serogroup B...
  • ✓vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae

Who cannot take part

  • ✗blood clotting cell count \< 30000/μL or there is a need for platelet transfusions.
  • ✗ANC \< 500/μL.
  • ✗Clinically significant laboratory abnormalities related to liver function, including:
  • ✗liver enzymes \> 2 × ULN or liver enzymes \> 3 × ULN for participants with documented liver iron overload defined by serum ferritin...
  • ✗Direct liver blood test \> 2 × ULN, unless, in the Investigator's opinion, is due to hemolysis or Gilbert's syndrome based...
See the full 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.

Where Is This Study? (2 UK sites)

Research Site

Leeds LS9 7TF, United Kingdom

Recruiting

Research Site

London SE5 9RS, United Kingdom

Recruiting

How to Get in Touch

Alexion Pharmaceuticals, Inc. (Sponsor)

Sponsor contact

CONTACT

1-855-752-2356 clinicaltrials@alexion.com
Data sourced from ClinicalTrials.gov · Last verified: 2025-12