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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 efficacy and safety of iptacopan in patients with C3 glomerulopathy

Maruyama Hideki

Novartis Pharma. K.K.

Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan

rinshoshiken.toroku@novartis.com

Maruyama Hideki

Novartis Pharma. K.K.

Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan

+81-120-003-293

rinshoshiken.toroku@novartis.com

Not Recruiting

Oct. 11, 2021

Interventional

randomized controlled trial

double blind

placebo control

parallel assignment

treatment purpose

1. Male and female participants age >= 12 and =< 60 years at screening.
2. Diagnosis of C3G as confirmed by renal biopsy within 12 months prior to enrollment in adults and within 3 years in adolescents.
3. 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.
4. Reduced serum C3 (defined as less than 0.85 x lower limit of the central laboratory normal range) at Screening.
5. UPCR >= 1.0 g/g sampled from the first morning void urine sample at Day -75 and Day -15.
6. Estimated GFR (using the CKD-EPI formula) or measured GFR >= 30 ml/min/1.73m2 at screening and Day -15.
7. Vaccination against Neisseria meningitidis infection prior to the start of study treatment. Vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given, if available and according to local regulations.

1. Participants who have received any cell or organ transplantation, including a kidney transplantation.
2. 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.
3. Renal biopsy showing interstitial fibrosis/tubular atrophy (IF/TA) of more than 50%.
4. Monoclonal gammopathy of undetermined significance (MGUS) confirmed by the measurement of serum free light chains or other investigation as per local standard of care.
5. Participants with an active systemic bacterial, viral or fungal infection within 14 days prior to study treatment administration
6. The presence of fever >= 38 degree (100.4 degree Fahrenheit) within 7 days prior to study treatment administration.
7. A history of recurrent invasive infections caused by encapsulated organisms, e.g., N. meningitidis and S. pneumoniae.
8. 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.
9. 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.

Both

C3G

Arm A: LNP023
Arm B: Placebo to LNP023

Change from baseline in eGFR. [ Time Frame: 6 months (double-blind) ]
- To demonstrate the superiority of iptacopan vs. placebo in improving eGFR

+81-52-744-1958

Aug. 30, 2021

Yes

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

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