🏛️ Verified NIH Record • Updated 2026-06-17

A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH (NCT05389449)

A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated With Danicopan in an Alexion-sponsored Clinical Study

🔵 Active, Not Recruiting Phase3 💊 Investigational Drug / Biologic Sponsor: Alexion Pharmaceuticals, Inc.

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

This is a single-arm long-term extension study that will enroll participants with PNH who have completed participation in Alexion-sponsored clinical studies with danicopan as an add on therapy to a C5i.

Who can join: Review the eligibility criteria below.

Location: Research Site — Los Angeles, California

🧪 Interventions in This Study

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

Instant Checklist

Full eligibility criteria are available on the official NIH protocol. View the criteria ↗

📍 All Participating Trial Locations (45 Sites)

Showing the first 25 of 45 sites.

  • Research Site — Los Angeles, California
  • Research Site — Chicago, Illinois
  • Research Site — Baltimore, Maryland
  • Research Site — Kansas City, Missouri
  • Research Site — Long Island City, New York
  • Research Site — Cleveland, Ohio
  • Research Site — Dallas, Texas
  • Research Site — Porto Alegre,
  • Research Site — Rio de Janeiro,
  • Research Site — Toronto, Ontario
  • Research Site — Brno,
  • Research Site — Lille,
  • Research Site — Paris,
  • Research Site — Pessac,
  • Research Site — Pierre-Bénite,
  • Research Site — Athens,
  • Research Site — Thessaloniki,
  • Research Site — Haifa,
  • Research Site — Jerusalem,
  • Research Site — Avellino,
  • Research Site — Bassano del Grappa,
  • Research Site — Florence,
  • Research Site — Milan,
  • Research Site — Reggio Calabria,
  • Research Site — Roma,

💰 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.

💡 How to Participate

  1. Check the checklist above to see if you may qualify.
  2. Review the official NIH record below to find the coordinating site.
  3. Ask about the visit schedule, what is covered at no cost, and any travel support.
  4. Confirm with your own doctor before making a decision.
🏛️ View Official NIH Record ↗