Evaluating Danicopan with Ravulizumab or Eculizumab for Pediatric PNH Patients
A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis
This study is testing if adding danicopan to the current treatments of ravulizumab or eculizumab can help kids with PNH have better hemoglobin levels.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 6 (estimated) |
| Ages | 12 Years to 17 Years |
| Sex | All |
| Sponsor | Alexion Pharmaceuticals, Inc. Industry-sponsored |
| Drugs / interventions | ravulizumab, eculizumab |
| Locations | 4 sites (Saskatoon, Saskatchewan and 3 other locations) |
| Trial ID | NCT06449001 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are pediatric patients with a confirmed diagnosis of PNH and clinically significant extravascular hemolysis who have been treated with ravulizumab or eculizumab.
Not a fit: Patients with severe thrombocytopenia or significant liver function abnormalities may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve hemoglobin levels and overall health outcomes for pediatric patients with PNH.
How similar studies have performed: Other studies have shown promise in using add-on therapies for PNH, but the specific combination of danicopan with ravulizumab or eculizumab in pediatric patients is relatively novel.
Eligibility criteria
Show full inclusion / exclusion 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 this trial is running
Saskatoon, Saskatchewan and 3 other locations
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.