Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients (NCT06934967)
An Open-label, Single-arm, Multicenter, Phase 3 Study to Assess Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric PNH Patients 2 to <18 Years of Age
🩺 Plain-English Summary (8th-Grade Level)
The purpose of this open-label, single arm, multicenter, phase 3 study is to assess the pharmacokinetics of iptacopan in pediatric patients and to assess whether iptacopan is safe and well tolerated when used for the treatment of pediatric paroxysmal nocturnal hemoglobinuria (PNH) patients 2 to \< 18 years of age.
Who can join: Review the eligibility criteria below.
Location: Childrens Healthcare of Atlanta — Atlanta, Georgia
Age range: 2 Years to 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 (15 Sites)
- Childrens Healthcare of Atlanta — Atlanta, Georgia
- Cancer Institute of New Jersey — New Brunswick, New Jersey
- Childrens Hospital of Philadelphia — Philadelphia, Pennsylvania
- St Jude Childrens Research Hospital — Memphis, Tennessee
- Novartis Investigative Site — Brasília, Federal District
- Novartis Investigative Site — Natal, Rio Grande do Norte
- Novartis Investigative Site — Porto Alegre, Rio Grande do Sul
- Novartis Investigative Site — Santo André, São Paulo
- Novartis Investigative Site — São Paulo, São Paulo
- Novartis Investigative Site — São Paulo, São Paulo
- Novartis Investigative Site — Cali, Valle del Cauca Department
- Novartis Investigative Site — Cologne, North Rhine-Westphalia
- Novartis Investigative Site — Berlin,
- Novartis Investigative Site — Genova, GE
- Novartis Investigative Site — Utrecht,
📞 Contact the Study Coordinator
Phone: (888) 669-6682
Email: novartis.email@novartis.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)
- A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) — 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.