NCT06934967View on ClinicalTrials.gov

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

PHASE3Not yet recruitingINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

July 28, 2025

Primary Completion Date

October 1, 2031

Study Completion Date

October 31, 2031

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions

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.