Who is eligible for clinical trial NCT06449001?

Participants must meet the protocol's inclusion criteria for Paroxysmal Nocturnal Hemoglobinuria. The stated age range is 12 Years to 17 Years. Confirm all requirements with the study coordinator.

Does clinical trial NCT06449001 provide travel or cost reimbursement?

Check with the site coordinator at the participating facility for travel and lodging stipend availability, and for which routine-care costs are covered.

🏛️ Verified NIH Record • Updated 2025-12-19

Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis (NCT06449001)

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

🟢 Recruiting Phase3 💊 Investigational Drug / Biologic Sponsor: Alexion Pharmaceuticals, Inc.

🩺 Plain-English Summary (8th-Grade Level)

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 join: To join this study, participants generally need to meet the listed inclusion criteria (5 items) and avoid the listed exclusions (11 items).

Location: Research Site — Saskatoon, Saskatchewan

Age range: 12 Years to 17 Years

🧪 Interventions in This Study

Danicopan (DRUG)

📋 "Do I Qualify?" — 1-Minute Self Screener

Instant Checklist

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

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

Usually NOT eligible if any of the following apply:

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.

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (4 Sites)

Research Site — Saskatoon, Saskatchewan
Research Site — Paris,
Research Site — Leeds,
Research Site — London,

📞 Contact the Study Coordinator

Phone: (855) 752-2356

Email: clinicaltrials@alexion.com

Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.

💰 Cost, Insurance & Patient Rights

100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

More studies in Paroxysmal Nocturnal Hemoglobinuria

Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data — Boston, Massachusetts
A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors — Ciudad Autonoma Buenos Aires,
The International PNH Interest Group PNH Registry — Altamonte Springs, Florida
A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently — Ribeirão Preto, São Paulo
Long-term Safety of Danicopan: IPIG Registry-based Cohort Study — Boston, Massachusetts

Explore by condition: All Paroxysmal Nocturnal Hemoglobinuria clinical trials

💡 How to Participate

Check the checklist above to see if you may qualify.
Contact the study coordinator using the phone/email above.
Ask about the visit schedule, what is covered at no cost, and any travel support.
Confirm with your own doctor before making a decision.

🏛️ View Official NIH Record ↗