Recruiting
Phase 3

Iptacopan

Sponsor:

Novartis Pharmaceuticals

Code:

NCT04817618

Conditions

C3G

Eligibility Criteria

Sex: All

Age: 12 - 60

Healthy Volunteers: Not accepted

Interventions

Placebo

iptacopan

Study Details

Brief summary:

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.

Conditions

C3G

Study ID

NCT04817618

Start date

Jul 28, 2021

Status verified date

Jul, 2026

Completion date

Jan 29, 2027

Anticipated

Primary completion date

Jan 29, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 12 - 60

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

98 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: iptacopan 200mg

iptacopan 200 mg b.i.d.

placebo comparator: Placebo to iptacopan 200mg

Placebo to iptacopan 200mg b.i.d.

Interventions

Placebo

Placebo to iptacopan 200mg b.i.d. (Adults 200mg b.i.d; Adolescents 2x 100mg b.i.d)

iptacopan

iptacopan 200 mg b.i.d. (Adults 200mg b.i.d; Adolescents 2x 100mg b.i.d)

Primary outcome measure

  • Adult cohort: Log-transformed ratio to baseline in UPCR (sampled from a 24-hour urine collection) [ Time Frame: 6 months (double-blind) ]
  • Adolescent cohort: Log-transformed ratio to baseline in UPCR (sampled from a 24-hour urine collection) [ Time Frame: 6 months (double-blind) ]
  • Change from baseline in log-transformed UPCR at the 12-month visit (both study treatment arms). [ Time Frame: 12 months (double-blind and open-label) ]
  • Change in log-transformed UPCR from the 6-month visit to the 12-month visit in the placebo arm [ Time Frame: From month 6 to month 12 (open-label) ]

Central Contacts and Locations

Central contacts

Novartis Pharmaceuticals

1-888-669-6682novartis.email@novartis.com

Novartis Pharmaceuticals

+41613241111

Locations

Childrens Hospital Colorado

Recruiting

Aurora, Colorado, United States, 80045

Contacts

Kati Dugan

+1 720 777 1234Kati.Dugan@childrenscolorado.org

Principal Investigator:

Bradley Dixon

Nicklaus Childrens Hospital

Recruiting

Miami, Florida, United States, 33155

Contacts

Jorge Haddad

+1 305 661 1515Jorge.Haddad@Nicklaushealth.org

Principal Investigator:

Ana Paredes

University of Iowa Health Care

Recruiting

Iowa City, Iowa, United States, 52242-1091

Contacts

Nicole Gerot

+1 319 335 7555nicole-gerot@uiowa.edu

Principal Investigator:

Carla Nester

University of Iowa Health Care

Recruiting

Iowa City, Iowa, United States, 52242

Contacts

Nikki Gerot

+1 (319) 335-0348

Principal Investigator:

Carla Nester

University of Wisconsin

Recruiting

Madison, Wisconsin, United States, 53792

Contacts

Alana Quackenbush

+1 608 265 6020akquackenbus@wisc.edu

Principal Investigator:

Sharon Bartosh

More Information

Sponsor

Novartis Pharmaceuticals

Last update posted

Jul 13, 2026

Last verified

Jul, 2026

Keywords

  • LNP023
  • iptacopan
  • C3G
  • UPCR
  • eGFR
  • proteinuria
  • Quality of life

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Novartis Pharmaceuticals on 2026-07-13.