A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (NCT05776472)
A Single Arm, Long-term, Multicentre Observational Study to Evaluate Effectiveness of Pegcetacoplan Under Real World Conditions in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
🩺 Plain-English Summary (8th-Grade Level)
This is a 36-month, long-term, multicenter, observational study designed to describe the real world effectiveness of pegcetacoplan in patients with PNH. Patients meeting the eligibility criteria will be enrolled in the study and followed prospectively for approximately 36 months. Patient data will be collected from start of pegcetacoplan treatment to end of follow-up. Retrospective data on pegcetacoplan will be captured from the time of pegcetacoplan treatment initiation. Pegcetacoplan treatment data will be collected for a minimum of approximately 36 months and up to a maximum of approximately 72 months, including retrospective period depending on when the patient started pegcetacoplan treatment. After pegcetacoplan treatment discontinuation, patients will remain in the study for 8 weeks to capture any AEs. The scope of the study is to collect both retrospective and prospective data. Baseline is defined as start of pegcetacoplan treatment. The main part of the study will be prospective,collecting data on effectiveness, safety (all AEs), patient- and clinician-reported outcomes and health care resource use.
Who can join: Review the eligibility criteria below.
Location: Liverpool Hospital — Liverpool, New South Wales
Age range: 18 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (87 Sites)
Showing the first 25 of 87 sites.
- Liverpool Hospital — Liverpool, New South Wales
- Calvary Mater Newcastle — Waratah, New South Wales
- Sunshine Coast University Hospital — Birtinya, Queensland
- Canberra Hospital — Garran,
- Institut Jules Bordet — Anderlecht,
- AZ Sint-Jan — Bruges,
- CHU Brugmann-Site Horta — Brussels,
- Centre Hospitalier Universitaire de Liege — Liège,
- AZ Herentals — Turnhout,
- London Health Sciences Centre (LHSC) - Victoria Hospital — London, Ontario
- University Health Network — Toronto, Ontario
- Hopital Maisonneuve-Rosemont d/b/a CIUSSS de l'Est-de-l'Île-de-Montréal — Montreal, Quebec
- University hospital centre Zagreb — Zagreb,
- Fakultni nemocnice Brno — Brno,
- Ustav hematologie a krevni transfuze — Prague,
- Helsingin yliopistollinen keskussairaala — Helsinki,
- CHU Nice - Hôpital de l'Archet 1 — Nice, Alpes Maritimes
- CHU de Bordeaux - Hôpital Haut-Lévêque — Pessac, Gironde
- Hopital Foch — Suresnes, Hauts de Seine
- CHU de Grenoble - Hôpital Albert Michallon — Grenoble, Isere
- CHU de Nantes - Hotel Dieu — Nantes, Loire Atlantique
- CHU de Nancy - Hôpital de Brabois Adultes — Vandœuvre-lès-Nancy, Meurthe et Moselle
- Hopital Claude Huriez - CHRU Lille — Lille, Nord
- Hôpital Saint-Louis — La Defense, Paris
- Centre Hospitalier Lyon Sud — Pierre-Bénite, Rhone
📞 Contact the Study Coordinator
Phone: +46 (0)8 697 20 00
Email: medical.info@sobi.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,
- Long-term Safety of Danicopan: IPIG Registry-based Cohort Study — Boston, Massachusetts
- A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH — Los Angeles, California
- Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria — Duarte, California
- A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment — Whittier, California
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.