Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgAN

Conditions

• Primary Immunoglobulin A Nephropathy (IgAN)

Eligibility Criteria

Sex

ALL

Age

2 Years - 18 Years

Healthy Volunteers

Not accepted

Interventions

• iptacopan — DRUG
  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

Study Details

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.

Key Dates

Start date

Nov 27, 2025

Status verified

May 2026

Primary completion

Jun 28, 2030

Completion

Jun 30, 2030

Study Design

Enrollment

31 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: iptacopan
  Participants in Cohort 1 (12 to \< 18 years old) will receive iptacopan at the dose of 200 mg twice per day. Participants in Cohort 2 (2 to \< 12 years old) will receive iptacopan at a dose tbd.

Primary Outcome Measure

Log-transformed ratio to Baseline in UPCR (based on FMV) [ Time Frame: Baseline, Week 38 ]

Central Contacts

• Novartis Pharmaceuticals
  1-888-669-6682
  [email protected]
• Novartis Pharmaceuticals
  +41613241111

Locations (3)

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