Evidence map›Paper›PMID 36217526›Full record

ArticleKidney international reports2022

Alternative Complement Pathway Inhibition With Iptacopan for the Treatment of C3 Glomerulopathy-Study Design of the APPEAR-C3G Trial.

Andrew S Bomback, David Kavanagh, Marina Vivarelli, Matthias Meier, Yaqin Wang, Nicholas J A Webb, Angelo J Trapani, Richard J H Smith

Registry-linked trialOpen access · goldAbstract read
In one paragraph

Article in Kidney international reports, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04817618 (A Multicenter, Randomized, Double-blind, Parallel Group, Placebo-controlled Study to Evaluate the Efficacy and Safety of Iptacopan), which is not on this map. Cited by 48 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
48citing papers in PubMed, 3 pooled it
5.4field-weighted citation impact, top 3% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT04817618 phase3recruitingnot on this map

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

TypeinterventionalSponsorNovartis PharmaceuticalsRan2021 to 2027Enrolled98ConditionsC3GArmsPlacebo, iptacopan
3 · Its place in the literature

Who cites it

48 citing papers in PubMed, 3 syntheses or guidelines pooled it, 71 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Guideline
  4. Trial
  5. Article
  6. Complement activation in kidney transplantation.Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association · 2026
    Review
  7. Review
  8. Review
  9. Review
  10. Review
  11. Article
  12. A Narrative Review of C3 Glomerulopathy: From Pathogenesis to Targeted Therapy.Canadian journal of kidney health and disease · 2026
    Review
  13. Review
  14. Review
  15. Review
  16. Review
  17. Review
  18. Article
  19. Article
  20. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors at 6 institutions in 4 countries.

Andrew S BombackDivision of Nephrology, Department of Medicine, Columbia University Medical Center, New York, USA.
David KavanaghTranslational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.
Marina VivarelliDivision of Nephrology, Department of Pediatric Subspecialties, IRCCS Ospedale Pediatrico Bambino Gesu, Rome, Italy.
Matthias MeierNovartis Pharmaceutical AG, Basel, Switzerland.
Yaqin WangNovartis Pharmaceutical Corporation, East Hanover, New Jersey, USA.
Nicholas J A WebbNovartis Pharmaceutical AG, Basel, Switzerland.
Angelo J TrapaniNovartis Pharmaceutical Corporation, East Hanover, New Jersey, USA.
Richard J H SmithMolecular Otolaryngology and Renal Research Laboratories, University of Iowa, Iowa City, Iowa, USA.
Novartis (Switzerland) · CHNovartis (United States) · USColumbia University Irving Medical Center · USIstituti di Ricovero e Cura a Carattere Scientifico · ITNewcastle University · GBUniversity of Iowa · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Introduction: Complement 3 glomerulopathy (C3G) is a rare kidney disease characterized by dysregulation of the alternative pathway (AP) of the complement system. About 50% of patients with C3G progress to kidney failure within 10 years of diagnosis. Currently, there are no approved therapeutic agents for C3G. Iptacopan is an oral, first-in-class, potent, and selective inhibitor of factor B, a key component of the AP. In a Phase II study, treatment with iptacopan was associated with a reduction in proteinuria and C3 deposit scores in C3G patients with native and transplanted kidneys, respectively. Methods: APPEAR-C3G (NCT04817618) is a randomized, double-blind, and placebo-controlled Phase III study to evaluate the efficacy and safety of iptacopan in C3G patients, enrolling 68 adults with biopsy-confirmed C3G, reduced C3 (<77 mg/dl), proteinuria ≥1.0 g/g, and estimated glomerular filtration rate (eGFR) ≥30 ml/min per 1.73 m Conclusion: This study aims to demonstrate the clinical benefits of AP inhibition with iptacopan in C3G.

Indexed as

alternative pathwayC3Gclinical trialscomplement systemfactor BiptacopanLNP023

Identifiers

PMID36217526
PMCPMC9546729
OpenAlexW4289526307

What OpenQuestion holds

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LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.