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, including two UPCR FMV measurements (required prior to Day 1). * Treatment Period ( 52 weeks): The study will enroll approximately 34 pediatric patients in a staggered manner. The approach will be to first enroll Cohort 1 (12 to \< 18 years of age) to collect safety data (including CV surveillance data), tolerability data, PK data and biomarker data for up to 52 weeks (EOS). Cohort 1 participants will be enrolled in a single group of 22 participants. Cohort 2 (2 to \< 12 years of age) participants will be enrolled in 2 groups with a total 12 participants. * Follow-up Period (1 week): Participants who completed the Week 52 (EOS) visit and meet all the eligibility criteria may be enrolled in a rollover extension program (REP) in which they will receive open-label iptacopan. Participants who do not enroll in the REP or do not complete Day 1 of the REP within 7 days of the EOS, should be contacted 7 days after the EOS visit for AE and SAE monitoring and any AEs reported by the participant should be recorded in the study case report form (CRF).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
34
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
Childrens Hospital Colorado
Aurora, Colorado, United States
RECRUITINGChildrens Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
NOT_YET_RECRUITINGLog-transformed ratio to Baseline in UPCR (based on FMV)
UPCR is measured based on the geometric mean of 2 FMVs obtained preceding each scheduled visit.
Time frame: Baseline, Week 38
Pharmacokinetic Parameter Cmax in Plasma
Cmax is the maximum (peak) observed plasma drug concentration after dose administration (mass x volume-1)
Time frame: Week 12 (Pre-dose (0), 2, 4, 6, and 8 hours post-dose)
Pharmacokinetic Parameter AUClast in Plasma
AUClast is the AUC from time zero to the last measurable concentration sampling time (tlast) (mass x time x volume-1)
Time frame: Week 12 (Pre-dose (0), 2, 4, 6, and 8 hours post-dose)
Pharmacokinetic Parameter AUCtau in Plasma
AUCtau is the AUC calculated to the end of a dosing interval (tau) at steady-state (amount x time x volume-1)
Time frame: Week 12 (Pre-dose (0), 2, 4, 6, and 8 hours post-dose)
Ctrough concentrations
Pre-dose drug concentration
Time frame: Week 4, Week 12 and Week 38
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Prim Childrens Hosp Inv Pharm
Salt Lake City, Utah, United States
Novartis Investigative Site
Nedlands, Western Australia, Australia
RECRUITINGNovartis Investigative Site
Beijing, China
RECRUITINGNovartis Investigative Site
Beersheba, Israel
RECRUITINGNovartis Investigative Site
Haifa, Israel
RECRUITINGNovartis Investigative Site
Jerusalem, Israel
RECRUITINGNovartis Investigative Site
Petah Tikva, Israel
RECRUITINGNovartis Investigative Site
Fuchū, Tokyo, Japan
RECRUITING