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).
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
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
Where Is This Study? (2 UK sites)
Research Site
Leeds LS9 7TF, United Kingdom
Research Site
London SE5 9RS, United Kingdom
How to Get in Touch
Alexion Pharmaceuticals, Inc. (Sponsor)
Sponsor contactCONTACT
