Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
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
Novartis Pharmaceuticals
12 participants
Oct 28, 2025
INTERVENTIONAL
Conditions
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.
Eligibility
Plain Language Summary
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Interventions
Cohort 1-administered orally a dosing scheme of 200 mg twice-daily (two 100 mg capsules). Cohort 2- administered orally a dosing scheme based on weight at the Day 1, Week 12, 26 and 38.
Locations(13)
View Full Details on ClinicalTrials.gov
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NCT06934967