MedTrace Logo

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

NCT06934967 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-04-16

No results posted yet for this study

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.

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.

Sponsors & Collaborators

Principal Investigators

  • Novartis Pharmaceuticals · Novartis Pharmaceuticals

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
2 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-10-28
Primary Completion
2031-11-19
Completion
2031-12-19
FDA Drug
Yes

Countries

  • United States
  • Brazil
  • Colombia
  • Germany
  • Italy
  • Netherlands

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06934967 on ClinicalTrials.gov