Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgAN
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age2-18
SponsorNovartis Pharmaceuticals
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
The minimum body weight for participants in Cohort 1 is 35 kg at Screening and confirmed at Baseline (Day 1).
Disqualifiers
Any secondary IgAN observed at Screening (and confirmed at Baseline/Day 1) as defined by the Investigator; secondary IgAN can be associated with cirrhosis, celiac disease, human immunodeficiency virus (HIV) infection, herpes simplex virus infection, dermatitis herpetiformis, seronegative arthritis, small-cell carcinoma, lymphoma, disseminated tuberculosis, bronchiolitis obliterans, inflammatory bowel disease, and familial mediterranean fever.
A clinical diagnosis of immunoglobulin A vasculitis (IgAV or Henoch-Schonlein purpura) based on typical palpable purpura with or without arthralgia and abdominal pain.
Evidence of significant urinary obstruction or difficulty in voiding at Screening (and confirmed at Baseline/Day 1); any urinary tract disorder or any chronic kidney disease other than IgAN at Screening and before first study drug administration.
Current acute kidney injury (AKI) defined by Acute Kidney Injury Network (AKIN) criteria within 4 weeks of screening.
Trial design
Design model
Single group
Treatments tested in this trial
iptacopan
DrugCohort 1 (12 to \< 18 years of age): Iptacopan 200 mg b.i.d.(twice daily) Cohort 2 (2 to \< 12 years old): Dosing tbd
Treatment groups
31 Participants
are divided into 1 treatment groupGroup A: iptacopanExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Log-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 38Secondary outcomes
1
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)2
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)3
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)4
Ctrough concentrations
Pre-dose drug concentration
Time frame
Week 4, Week 12 and Week 38Sponsors and contacts
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Source ClinicalTrials.gov