Clinical Trial

Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients

Recruiting Phase 3
View on ClinicalTrials.gov →
Summary
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.
Protocol Amendment History 2 changes
notable Primary completion moved earlier: 2031-11-19 -> 2031-10-14 2026-08-08
minor Completion moved earlier: 2031-12-19 -> 2031-11-13 2026-08-08
Trial Details
NCT Number NCT06934967
Lead Sponsor Novartis Pharmaceuticals
Conditions Paroxysmal Nocturnal Hemoglobinuria (PNH)
Enrollment 12 participants
Start Date 2025-10-28
Primary Completion 2031-10-14 (estimated)
Study Completion 2031-11-13 (estimated)
Updated on ClinicalTrials.gov 2026-08-07