Novartis Pharmaceuticals
The study is an open-label, single arm, multicenter, Phase III study to determine proteinuria reduction, pharmacokinetics (PK), safety and tolerability (including CV surveillance) of iptacopan in primary immunoglobulin A nephropathy (IgAN) pediatric patients aged 2 to \<18 years.
The study will enroll approximately 34 pediatric patients with a diagnosis of primary IgAN by kidney biopsy performed within 3 years of Screening with \< 50% tubulointerstitial fibrosis and \<25% crescents and not previously treated with immunosuppressive or other immunomodulatory agents within 90 days (or 180 days for rituximab) prior to first study drug administration. The study comprises the following periods: * Screening Period (up to 11 weeks) * Run-in Period (2 weeks): This period will be approximately 14 days prior to the Enrollment Visit (Day 1) to obtain Baseline assessments, includ…
Inclusion Criteria: * Male and female participants 2 to \< 18 years of age as of Day 1. * eGFR ≥ 30 mL/min/1.73m2 where eGFR is calculated using the modified Schwartz formula at Screening and confirmed during the Run-in Period. * Kidney biopsy-proven primary IgAN\*, with biopsy performed within 3 years of Screening with \< 50% tubulointerstitial fibrosis and \< 25% crescents. In case a kidney biopsy within 3 years from Screening is not available, a kidney biopsy may be performed if it is part of the planned diagnostic approach and clinical management of the participant. \* Note: Primary Ig…
Cohort 1 (12 to \< 18 years of age): Iptacopan 200 mg b.i.d.(twice daily) Cohort 2 (2 to \< 12 years old): Dosing tbd