U.S., April 19 -- ClinicalTrials.gov registry received information related to the study (NCT06934967) titled 'Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients' on March 31.
Brief 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.
Study Start Date: July 28, 2025
Study Type: INTERVENTIONAL
Condition:
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Intervention:
DRUG: LNP023
Cohort 1-administered orally a dosing scheme of...