NCT06934967

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

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 2 Years – 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This is a multicenter, open-label, single arm study comprised of an up to a 8-week Screening Period, and a 26-week Treatment Period followed by a 26-week Extension Treatment Period. This study will enroll a minimum of 12 pediatric patients 2 to \< 18 years of age in a staggered manner into 3 cohorts: Cohort 1 (adolescents 12 to \< 18 years of age, approximately 6 patients), Cohort 2a (6 to \< 12 years of age, approximately 4 patients), and Cohort 2b (2 to \< 6 years of age, approximately 2 patients).

Conditions

• Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions

Type	Name	Description
DRUG	LNP023	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.

Timeline

• Start date: 2025-10-28
• Primary completion: 2031-11-19
• Completion: 2031-12-19
• First posted: 2025-04-18
• Last updated: 2026-04-16

Locations

13 sites across 6 countries: United States, Brazil, Colombia, Germany, Italy, Netherlands

Regulatory

• FDA-regulated drug study