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Study identifier: NCT06934967 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

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

Condition: Paroxysmal Nocturnal Hemoglobinuria (PNH)  ·  Sponsor: Novartis Pharmaceuticals

PhasePhase 3
Planned participants12
Who can joinAll sexes, 2 Years to 18 Years
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Novartis Pharmaceuticals  ·  1-888-669-6682  ·  novartis.email@novartis.com

Novartis Pharmaceuticals  ·  +41613241111

Always discuss trial participation with your own doctor first.

Locations (13)

Cancer Institute of New JerseyNew Brunswick, New Jersey, United StatesRecruiting
Childrens Hospital of PhiladelphiaPhiladelphia, Pennsylvania, United StatesRecruiting
St Jude Childrens Research HospitalMemphis, Tennessee, United StatesRecruiting
Novartis Investigative SiteBrasília, Federal District, BrazilRecruiting
Novartis Investigative SiteNatal, Rio Grande do Norte, BrazilRecruiting
Novartis Investigative SitePorto Alegre, Rio Grande do Sul, BrazilRecruiting
Novartis Investigative SiteSanto André, São Paulo, BrazilRecruiting
Novartis Investigative SiteSão Paulo, São Paulo, BrazilRecruiting
Novartis Investigative SiteSão Paulo, São Paulo, BrazilRecruiting
Novartis Investigative SiteCali, Valle del Cauca Department, ColombiaRecruiting
Novartis Investigative SiteBerlin, GermanyRecruiting
Novartis Investigative SiteGenova, GE, ItalyRecruiting

+ 1 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT06934967