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NCT06934967

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

Recruiting now Phase 3 Last updated 16 April 2026
What this trial tests

Phase 3 trial testing LNP023 in Paroxysmal Nocturnal Hemoglobinuria (PNH) in 12 participants. Currently enrolling.

Timeline
28 October 2025
Primary endpoint
19 November 2031
19 December 2031

Quick facts

Lead sponsorNovartis Pharmaceuticals
PhasePhase 3
StatusRecruiting now
Study typeINTERVENTIONAL
Allocationnon randomized
Designsequential
Maskingnone
Primary purposetreatment
Enrollment12
Start date28 October 2025
Primary completion19 November 2031
Estimated completion19 December 2031
Sites13 locations across Italy, Colombia, Netherlands, Germany, United States, Brazil

Drugs / interventions tested

Conditions studied

Sponsor

Novartis Pharmaceuticals — full company profile →

Who can join

Adults 2 to 18, any sex, with Paroxysmal Nocturnal Hemoglobinuria (PNH). Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

  1. The varieties of therapeutic experience: navigating treatment options for patients with PNH.
    Bienz M, Patriquin CJ. · · 2025 · PMID 41348037 · DOI 10.1182/hematology.2025000701

Verify or expand the search:

Other trials of LNP023

Trials testing the same drug.

Other recruiting trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)

Currently open trials in the same condition.

Other Novartis Pharmaceuticals trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT06934967.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing