Evaluating Danicopan with Ravulizumab or Eculizumab for Pediatric PNH Patients

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study aims to assess the efficacy of danicopan as an additional treatment for pediatric patients diagnosed with paroxysmal nocturnal hemoglobinuria (PNH) who are already receiving ravulizumab or eculizumab. The primary measure of success will be the change in hemoglobin levels from baseline after 12 weeks of treatment. Participants must have clinically significant extravascular hemolysis and have been on stable doses of their current treatment for at least 12 weeks prior to the study. The study will also ensure that all participants are vaccinated against specific infections.

Why it matters

Who can join: the full rules

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 it's running

• Research Site
  Saskatoon, Saskatchewan, Canada Recruiting
• Research Site
  Paris, France Recruiting
• Research Site
  Leeds, United Kingdom Recruiting
• Research Site
  London, United Kingdom Recruiting

Study contacts

• Study coordinator: Alexion Pharmaceuticals, Inc. (Sponsor)
• Email: clinicaltrials@alexion.com
• Phone: 1-855-752-2356

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• Danicopan added to ravulizumab or eculizumab for PNH in Korea

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.