Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric… (NCT06994845) | Clinical Trial Compass
RecruitingPhase 3
Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgAN
United States, Australia, China31 participantsStarted 2025-11-27
Plain-language summary
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.
Who can participate
Age range
2 Years – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 IgAN is defined as any IgAN that is proven by biopsy showing IgA deposits prevalent over the other classes of immunoglobulins and deemed not to be associated with causes of secondary IgAN as per clinical judgment of the Investigator.
* The minimum body weight for participants in Cohort 1 is 35 kg at Screening and confirmed at Baseline (Day 1).
* Proteinuria due to primary diagnosis of IgAN as assessed by UPCR ≥ 1 g/g (113 mg/mmoL) sampled from FMV at Screening on Day -90 and Day -60 as well as during the Run-in Period despite treatment with maximum tolerated dose of ACE inhibitor/ARB for at least 120 days prior to Day 1. Note: UPCR will be assessed based on one FMV sample at Day -90 and based on the geometric mean of 2 FMV samples for the Day -60 visit and during the Run-in Period.
* Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is required prior to the …
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Log-transformed ratio to Baseline in UPCR (based on FMV)