A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (NCT04901936)
An Open Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria
🩺 Plain-English Summary (8th-Grade Level)
The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds (adolescents) who have paroxysmal nocturnal hemoglobinuria (PNH).
Who can join: Review the eligibility criteria below.
Location: Children's Hospital of Atlanta — Atlanta, Georgia
Age range: 12 Years to 17 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 (12 Sites)
- Children's Hospital of Atlanta — Atlanta, Georgia
- Motol University Hospital — Prague,
- Robert-Debré Hospital Paris — Paris,
- Hospital Ampang — Ampang,
- Radboud University Hospital Nijmegen — Nijmegen,
- University Medical Center Utrecht — Utrecht,
- University Children's Hospital — Belgrade,
- University Hospital Vall d'Hebron — Barcelona,
- University Hospital 12 de Octubre — Madrid,
- Phramongkutklao Hospital and College of Medicine — Bangkok,
- Maharaj Nakorn Chiang Mai hospital — Chiang Mai,
- St. Mary's Hospital — London,
📞 Contact the Study Coordinator
Phone: 1-833-284-6361 (833-CT Info-1)
Email: clinicaltrials@apellis.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 (PNH)
- Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients — Atlanta, Georgia
- A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor — Toronto, Ontario
Explore by condition: All Paroxysmal Nocturnal Hemoglobinuria (PNH) 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.