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A Multicenter, Randomized, Double-blind, Parallel Group, Placebo-controlled Study to Evaluate the Efficacy and Safety of Iptacopan (LNP023) in Complement 3 Glomerulopathy.
Study of Investigational Medication to Investigate the Potential to Reduce Protein in Urine for Complement 3 Glomerulopathy
M
Myda Khalid
Primary Investigator
Enrolling By Invitation
12 years - 60 years
All
Phase
N/A
1 participants needed
1 Location
Brief description of study
The purpose of this study is to evaluate the efficacy and safety of iptacopan compared to placebo and standard of care in patients with native C3G. CLNP023B12301 is a Phase 3 pivotal trial for registration of iptacopan in C3G. The study aims to determine the reduction in UPCR and improvement in eGFR in participants treated with iptacopan compared to placebo, as well as the proportion of participants who achieve a composite renal endpoint consisting of eGFR and UPCR elements. These effects of iptacopan in conjunction with increases in serum C3 levels will provide support for an iptacopan profile that includes stabilization of eGFR, clinically meaningful reductions in proteinuria and inhibition of the complement AP.
THIS STUDY IS ENROLLING BY INVITATION ONLY
Detailed description of study
The study is designed as a multicenter, randomized, double-blind, parallel group, placebo-controlled study to evaluate the efficacy and safety of iptacopan (LNP023) in complement 3 glomerulopathy.
- Kidney biopsies will be performed in adult participants to evaluate histopathological improvements in immunofluorescence and light microscopy that support these functional benefits of iptacopan.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Complement 3 Glomerulopathy, C3G, Kidney disease, Riley
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Age: 12 years - 60 years
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Gender: All
Inclusion Criteria:
- 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.
This study investigates the effects of an investigational medication compared to a placebo in people with Complement 3 Glomerulopathy (C3G), a kidney disease. C3G is a condition where a protein called complement 3 is deposited in the kidneys, causing damage. The purpose is to see if the investigational medication can help reduce protein in urine and improve kidney function.
Participants will undergo kidney biopsies, which involve taking a small piece of kidney tissue to look for changes under a microscope. The study will compare these changes between those receiving the investigational medication and those receiving a placebo, an inactive substance that looks like the investigational medicine but does not contain any medicine.
- Who can participate: Adults and adolescents with a confirmed diagnosis of C3G from a kidney biopsy can participate. They must have been on specific medications for at least 90 days and meet criteria for kidney function and protein levels in urine.
- Study details: Participants will be randomly assigned to receive either the investigational medication or a placebo. They will continue their usual care and undergo procedures like kidney biopsies.
Updated on
08 Jan 2026.
Study ID: PNEPH-NOVARTIS-LNP023, 20176, 25047
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