C3 glomerulopathy

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ORPHA:329918OMIM:609814N03.5
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2FDA treatments4Active trials29Specialists8Treatment centers2Financial resources

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

C3 glomerulopathy (C3G) is a rare kidney disease caused by problems with a part of the immune system called the complement system. The complement system normally helps the body fight infections, but in C3G, it becomes overactive and deposits a protein called C3 in the tiny filters of the kidneys (called glomeruli). This damages the kidneys over time and can lead to serious kidney problems. C3 glomerulopathy includes two main subtypes: dense deposit disease (DDD) and C3 glomerulonephritis (C3GN). Both share the same underlying problem of uncontrolled complement activation but look slightly different under the microscope. Common symptoms include blood in the urine (which may look pink or brown), foamy urine from excess protein, swelling in the legs and around the eyes, high blood pressure, and fatigue. Over time, kidney function can decline. There is currently no cure for C3G. Treatment focuses on protecting the kidneys and slowing disease progression. Doctors may prescribe blood pressure medications called ACE inhibitors or ARBs to reduce protein in the urine, immunosuppressive drugs to calm the immune system, and in some cases newer complement-targeting therapies. Some patients eventually need dialysis or a kidney transplant, though the disease can unfortunately come back in a transplanted kidney. Research into complement-targeted treatments is active and offers hope for better options in the future.

Also known as:

Key symptoms:

Blood in the urine (pink, red, or brown urine)Foamy or bubbly urine from excess proteinSwelling in the legs, ankles, or feetPuffiness around the eyes, especially in the morningHigh blood pressureFatigue and tirednessDecreased urine outputUnexplained weight gain from fluid retentionLoss of appetiteNauseaHeadaches from high blood pressureGradual decline in kidney function

Clinical phenotype terms (23)— hover any for plain English
Membranoproliferative glomerulonephritisHP:0000793Decreased circulating complement C3 concentrationHP:0005421Mesangial hypercellularityHP:0012574Glomerular extracapillary hypercellularityHP:0025364C3 nephritic factor positivityHP:0030888ParaproteinemiaHP:0031047LipodystrophyHP:0009125DrusenHP:0011510Central serous chorioretinopathyHP:0025567
Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

10 events
Feb 2026Clinical Outcomes of C3 Glomerulopathy and IC-MPGN in Russia: Hybrid Retrospective - Prospective Study

Novartis Pharmaceuticals

TrialNOT YET RECRUITING
Jan 2026CHART-C3G/CLNP023B12011

Novartis Pharmaceuticals

TrialNOT YET RECRUITING
Nov 2025Study of NM8074 in Adult C3 Glomerulopathy Patients

NovelMed Therapeutics — PHASE1, PHASE2

TrialNOT YET RECRUITING
Oct 2025Predictive Value of a Rectal Swab With Detection of Enterobacteria (ESBL-E), Carbapenemases, and High-level Cephalosporinase (HLC) on the Risk of Infections With C3G-resistant Enterobacteria

University Hospital, Strasbourg, France

TrialRECRUITING
Sep 2025Fabhalta Capsules Specified Drug-use Survey

Novartis Pharmaceuticals

TrialRECRUITING
Jun 2025A Clinical Trial to Test the Safety, Tolerability, and How the Body Processes CPV-104 in Healthy People and Patients With C3-Glomerulopathy

eleva GmbH — PHASE1

TrialRECRUITING
Apr 2025Home Reported Outcomes in C3G Study

Novartis Pharmaceuticals

TrialRECRUITING
Mar 2025

FABHALTA: FDA approved

treatment of adults with complement 3 glomerulopathy (C3G), to reduce proteinuria

FDAcompleted
Mar 2025Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of KP104 to Treat Glomerulonephritis

Kira Pharmacenticals (US), LLC. — PHASE2

TrialNOT YET RECRUITING
Jul 2021Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.

Novartis Pharmaceuticals — PHASE3

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

2 available

Empaveli

PEGCETACOPLAN· Apellis Pharmaceuticals, Inc.■ Boxed WarningOrphan DrugsNDA
Treatment of adult and pediatric patients aged 12 years and older with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN), to reduce proteinuria. FDA

Treatment of adult and pediatric patients aged 12 years and older with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN), to reduce proteinuria. FDA sNDA approval July 28, 2025. VALIANT Phase 3 trial (NCT05067127): 68% UPCR reduction vs placebo in 124 patients; 60% achieved >=50% UPCR reduction vs 5% placebo.

FABHALTA

IPTACOPAN· Novartis Pharmaceuticals Corporation■ Boxed WarningOrphan Drug

treatment of adults with complement 3 glomerulopathy (C3G), to reduce proteinuria

Clinical Trials

4 recruitingView all trials with filters →
Phase 31 trial
Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.
Phase 3
Actively Recruiting
PI: Novartis Pharmaceuticals (Novartis Pharmaceuticals) · Sites: Aurora, Colorado; Miami, Florida +85 more · Age: 1260 yrs
Phase 11 trial
A Clinical Trial to Test the Safety, Tolerability, and How the Body Processes CPV-104 in Healthy People and Patients With C3-Glomerulopathy
Phase 1
Actively Recruiting
PI: Bernd Jilma, Prof. (Medical University of Vienna) · Sites: Vienna; Brussels +14 more · Age: 1899 yrs
Other2 trials
Home Reported Outcomes in C3G Study
Actively Recruiting
PI: Novartis Pharmaceuticals (Novartis Pharmaceuticals) · Sites: East Hanover, New Jersey · Age: 18100 yrs
Fabhalta Capsules Specified Drug-use Survey
Actively Recruiting
PI: Novartis Pharmaceuticals (Novartis Pharmaceuticals) · Sites: Nagoya, Aichi-ken; Toyoake, Aichi-ken +21 more · Age: 0100 yrs

Specialists

Showing 25 of 29View all specialists →
MP
Matthew C Pickering
BAKER CITY, OR
Specialist
5 C3 glomerulopathy publications
AB
Andrew S Bomback
NEW YORK, NY
Specialist
7 C3 glomerulopathy publications
NW
Nicholas J A Webb
MISHAWAKA, IN
Specialist
6 C3 glomerulopathy publications
GR
Giuseppe Remuzzi
Specialist
6 C3 glomerulopathy publications
MP
Manuel Praga
Specialist
5 C3 glomerulopathy publications
MV
Marina Vivarelli
Specialist
5 C3 glomerulopathy publications
MM
Matthias Meier
Specialist
5 C3 glomerulopathy publications
FC
Fernando Caravaca-Fontán
Specialist
5 C3 glomerulopathy publications
CN
Carla M Nester
IOWA CITY, IA
Specialist
4 C3 glomerulopathy publications
RS
Richard J H Smith
Specialist
4 C3 glomerulopathy publications
MQ
Moglie Le Quintrec
Specialist
4 C3 glomerulopathy publications
DK
David Kavanagh
ST PETERSBURG, FL
Specialist
4 C3 glomerulopathy publications
FF
Fadi Fakhouri
Specialist
3 C3 glomerulopathy publications
DK
Diana Karpman
Specialist
PI on 1 active trial8 C3 glomerulopathy publications
BP
Bernd Jilma, Prof.
Specialist
PI on 1 active trial
AK
Alexandre Karras
Specialist
4 C3 glomerulopathy publications
SC
Sophie Chauvet
AUSTIN, TX
Specialist
4 C3 glomerulopathy publications
FM
Fernando C Fervenza, MD
ROCHESTER, MN
Specialist
PI on 1 active trial
AT
Angelo J Trapani
EAU CLAIRE, WI
Specialist
3 C3 glomerulopathy publications
DG
Daniel P Gale
OSHKOSH, WI
Specialist
2 C3 glomerulopathy publications
EW
Edwin K S Wong
Specialist
2 C3 glomerulopathy publications
NK
Nicole van de Kar
Specialist
2 C3 glomerulopathy publications
AK
Andreas Kronbichler
Specialist
2 C3 glomerulopathy publications
PZ
Peter F Zipfel
Specialist
2 C3 glomerulopathy publications

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Financial Resources

2 resources
Empaveli(PEGCETACOPLAN)Apellis Pharmaceuticals, Inc.
FABHALTA(IPTACOPAN)Novartis Pharmaceuticals Corporation

Travel Grants

No travel grants are currently matched to C3 glomerulopathy.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about C3 glomerulopathy

Disease timeline:

New recruiting trial: Predictive Value of a Rectal Swab With Detection of Enterobacteria (ESBL-E), Carbapenemases, and High-level Cephalosporinase (HLC) on the Risk of Infections With C3G-resistant Enterobacteria

A new clinical trial is recruiting patients for C3 glomerulopathy

New recruiting trial: Phase 2 Study of ADX-038 in Complement-Mediated Kidney Disease

A new clinical trial is recruiting patients for C3 glomerulopathy

New recruiting trial: A Clinical Trial to Test the Safety, Tolerability, and How the Body Processes CPV-104 in Healthy People and Patients With C3-Glomerulopathy

A new clinical trial is recruiting patients for C3 glomerulopathy

New recruiting trial: Fabhalta Capsules Specified Drug-use Survey

A new clinical trial is recruiting patients for C3 glomerulopathy

New recruiting trial: Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.

A new clinical trial is recruiting patients for C3 glomerulopathy

New recruiting trial: Home Reported Outcomes in C3G Study

A new clinical trial is recruiting patients for C3 glomerulopathy

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.What subtype of C3 glomerulopathy do I have — dense deposit disease or C3 glomerulonephritis?,What is my current kidney function level, and how quickly is it changing?,Should I have genetic testing or testing for autoantibodies like C3 nephritic factor?,What treatment options are best for my specific situation, and are there clinical trials I could join?,What is my risk of eventually needing dialysis or a kidney transplant?,If I need a transplant in the future, what is the risk of the disease coming back in the new kidney?,Are there dietary changes or lifestyle modifications that can help protect my kidneys?

Common questions about C3 glomerulopathy

What is C3 glomerulopathy?

C3 glomerulopathy (C3G) is a rare kidney disease caused by problems with a part of the immune system called the complement system. The complement system normally helps the body fight infections, but in C3G, it becomes overactive and deposits a protein called C3 in the tiny filters of the kidneys (called glomeruli). This damages the kidneys over time and can lead to serious kidney problems. C3 glomerulopathy includes two main subtypes: dense deposit disease (DDD) and C3 glomerulonephritis (C3GN). Both share the same underlying problem of uncontrolled complement activation but look slightly dif

Are there clinical trials for C3 glomerulopathy?

Yes — 4 recruiting clinical trials are currently listed for C3 glomerulopathy on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat C3 glomerulopathy?

25 specialists and care centers treating C3 glomerulopathy are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for C3 glomerulopathy?

2 patient support programs are currently tracked on UniteRare for C3 glomerulopathy. See the treatments and support programs sections for copay assistance, eligibility, and contact details.