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Evaluating iptacopan for treating C3 glomerulopathy

A Multicenter, Randomized, Double-blind, Parallel Group, Placebo-controlled Study to Evaluate the Efficacy and Safety of Iptacopan (LNP023) in Complement 3 Glomerulopathy.

PHASE3 · Novartis · NCT04817618

This study is testing if a new medication called iptacopan can help people with C3 glomerulopathy improve their kidney function and reduce protein in their urine.

Quick facts

PhasePHASE3
Study typeInterventional
Enrollment98 (estimated)
Ages12 Years to 60 Years
SexAll
SponsorNovartis (industry)
Drugs / interventionscyclophosphamide
Locations87 sites (Aurora, Colorado and 86 other locations)
Trial IDNCT04817618 on ClinicalTrials.gov

What this trial studies

This study is a multicenter, randomized, double-blind, parallel group, placebo-controlled trial designed to assess the efficacy and safety of iptacopan (LNP023) in patients diagnosed with C3 glomerulopathy (C3G). Participants will receive either iptacopan or a placebo alongside standard care, with the primary focus on measuring reductions in urine protein-to-creatinine ratio (UPCR) and improvements in estimated glomerular filtration rate (eGFR). Additionally, kidney biopsies will be conducted to evaluate histopathological changes that correlate with functional improvements. The study aims to provide robust evidence for the potential registration of iptacopan as a treatment for C3G.

Who should consider this trial

Good fit: Ideal candidates include males and females aged 12 to 60 with a confirmed diagnosis of C3G and specific renal function criteria.

Not a fit: Patients who do not have C3 glomerulopathy or those outside the specified age range may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve kidney function and reduce proteinuria in patients with C3 glomerulopathy.

How similar studies have performed: Other studies have shown promise with similar approaches targeting complement pathways, suggesting potential for success in this trial.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Male and female participants age ≥ 12 and ≤ 60 years at screening.
* Diagnosis of C3G as confirmed by renal biopsy within 12 months prior to enrollment in adults and within 3 years in adolescents.
* Prior to randomization, all participants must have been on a maximally recommended or tolerated dose of an angiotensin converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB) for at least 90 days. The doses of other antiproteinuric medications including mycophenolic acid, corticosteroids and mineralocorticoid receptor antagonists should be stable for at least 90 days prior to randomization.
* Reduced serum C3 (defined as less than 0.85 x lower limit of the central laboratory normal range) at Screening.
* UPCR ≥ 1.0 g/g sampled from the first morning void urine sample at Day -75 and Day -15.
* Estimated GFR (using the CKD-EPI formula for ages ≥ 18 years and modified Schwartz formula for ages 12 to 17 years) or measured GFR ≥ 30 ml/min/1.73m2 at screening and Day -15.
* Mandatory vaccination against Neisseria meningitidis and Streptococcus pneumoniae prior to the start of study treatment.
* If not previously vaccinated or if a booster is required, vaccination against Haemophilus influenzae infections should be given, if available and according to local regulations, at least 2 weeks prior to the first study treatment administration. If study treatment has to start earlier than 2 weeks post vaccination, prophylactic antibiotic treatment should be initiated.

Exclusion Criteria:

* Participants who have received any cell or organ transplantation, including a kidney transplantation.
* Rapidly progressive crescentic glomerulonephritis defined as a 50% decline in the eGFR within 3 months with renal biopsy findings of glomerular crescent formation seen in at least 50% of glomeruli.
* Renal biopsy showing interstitial fibrosis/tubular atrophy (IF/TA) of more than 50%
* Monoclonal gammopathy of undetermined significance (MGUS) confirmed by the measurement of serum free light chains or other investigation as per local standard of care.
* Participants with an active systemic bacterial, viral or fungal infection within 14 days prior to study treatment administration
* The presence of fever ≥ 38°C (100.4°F) within 7 days prior to study treatment administration.
* A history of recurrent invasive infections caused by encapsulated organisms, e.g., N. meningitidis and S. pneumoniae.
* The use of inhibitors of complement factors (e.g., Factor B, Factor D, C3 inhibitors, anti C5 antibodies, C5a receptor antagonists) within 6 months prior to the Screening visit.
* The use of immunosuppressants (except mycophenolic acids), cyclophosphamide or systemic corticosteroids at a dose \>7.5 mg/day (or equivalent for a similar medication) within 90 days of study drug administration.
* Acute post-infectious glomerulonephritis at screening based upon the opinion of the investigator.

Where this trial is running

Aurora, Colorado and 86 other locations

+37 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: C3G, LNP023, iptacopan, UPCR, eGFR, proteinuria, Quality of life

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.