The Primary Completion Date and Study Completion Date have been updated to reflect completion of the adolescent cohort, which has been added to the protocol.
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.
Eligibility
Sex
ALL
Min age
12 Years
Max age
60 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
Adult cohort: Log-transformed ratio to baseline in UPCR (sampled from a 24-hour urine collection) — 6 months (double-blind) To demonstrate the superiority of iptacopan compared to placebo in reducing proteinuria at 6 months of treatment.
Adolescent cohort: Log-transformed ratio to baseline in UPCR (sampled from a 24-hour urine collection) — 6 months (double-blind) To evaluate the effect of iptacopan on proteinuria at 6 months.
Change from baseline in log-transformed UPCR at the 12-month visit (both study treatment arms). — 12 months (double-blind and open-label) To evaluate the effect of iptacopan on proteinuria at 12 months.
Change in log-transformed UPCR from the 6-month visit to the 12-month visit in the placebo arm — From month 6 to month 12 (open-label) To evaluate the effect of iptacopan on proteinuria at 12 months.
Trial sites (87)
Facility
City
Region
Status
Childrens Hospital Colorado
Aurora
Colorado
Recruiting
Nicklaus Childrens Hospital
Miami
Florida
Recruiting
Georgia Nephrology Research Inst
Lawrenceville
Georgia
Completed
IN University School of Med
Indianapolis
Indiana
Withdrawn
University of Iowa Health Care
Iowa City
Iowa
Recruiting
University of Iowa Health Care
Iowa City
Iowa
Recruiting
Johns Hopkins Hospital
Baltimore
Maryland
Withdrawn
Brigham and Womens Hosp Harvard Med School
Boston
Massachusetts
Withdrawn
Hackensack Uni Medical Center
Hackensack
New Jersey
Withdrawn
Albany Medical Center
Albany
New York
Withdrawn
Col Uni Med Center New York Presby
New York
New York
Completed
Baylor Scott and White Research
Temple
Texas
Withdrawn
University of Wisconsin
Madison
Wisconsin
Recruiting
Novartis Investigative Site
Córdoba
Córdoba Province
Withdrawn
Novartis Investigative Site
Buenos Aires
Argentina
Completed
Novartis Investigative Site
CABA
Argentina
Withdrawn
Novartis Investigative Site
Edegem
Belgium
Withdrawn
Novartis Investigative Site
Leuven
Belgium
Withdrawn
Novartis Investigative Site
Belo Horizonte
Minas Gerais
Active Not Recruiting
Novartis Investigative Site
Recife
Pernambuco
Recruiting
Novartis Investigative Site
Passo Fundo
Rio Grande do Sul
Recruiting
Novartis Investigative Site
Joinville
Santa Catarina
Withdrawn
Novartis Investigative Site
Santo André
São Paulo
Withdrawn
Novartis Investigative Site
São Paulo
São Paulo
Recruiting
Novartis Investigative Site
São Paulo
São Paulo
Withdrawn
Novartis Investigative Site
Salvador
Brazil
Recruiting
Novartis Investigative Site
London
Ontario
Completed
Novartis Investigative Site
Toronto
Ontario
Completed
Novartis Investigative Site
Montreal
Quebec
Withdrawn
Novartis Investigative Site
Guangzhou
Guangdong
Active Not Recruiting
Novartis Investigative Site
Wuhan
Hubei
Withdrawn
Novartis Investigative Site
Beijing
China
Active Not Recruiting
Novartis Investigative Site
Beijing
China
Recruiting
Novartis Investigative Site
Shanghai
China
Active Not Recruiting
Novartis Investigative Site
Prague
Czechia
Completed
Novartis Investigative Site
Lille
France
Completed
Novartis Investigative Site
Marseille
France
Withdrawn
Novartis Investigative Site
Montpellier
France
Withdrawn
Novartis Investigative Site
Paris
France
Completed
Novartis Investigative Site
Paris
France
Withdrawn
+ 47 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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