Idiopathic multidrug-resistant nephrotic syndrome

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9Active trials8Treatment centers

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Overview

Idiopathic multidrug-resistant nephrotic syndrome (also called idiopathic steroid-resistant nephrotic syndrome or SRNS) is a serious kidney disease where the tiny filtering units in the kidneys, called glomeruli, stop working properly. In a healthy kidney, these filters keep important proteins — especially one called albumin — inside the blood. In this condition, the filters become leaky, allowing large amounts of protein to spill into the urine. This leads to low protein levels in the blood, which causes fluid to build up in the body, especially in the legs, belly, and around the eyes. What makes this condition particularly challenging is that it does not respond to the standard first-line treatment, steroids (like prednisone), and also fails to respond to several other immunosuppressive medications. This is why it is called 'multidrug-resistant.' The exact cause is often unknown — which is why the word 'idiopathic' is used — though in many cases, genetic changes in genes that control kidney filter function are found. Symptoms include severe swelling, foamy urine, fatigue, and high blood pressure. Over time, the kidneys can lose more and more function, and many patients eventually develop chronic kidney disease or kidney failure, requiring dialysis or a kidney transplant. Management focuses on reducing protein loss, protecting kidney function, controlling blood pressure, and preventing complications. This is a rare and complex condition that requires care from a specialist kidney doctor (nephrologist).

Key symptoms:

Severe swelling (edema), especially in the legs, ankles, feet, belly, and around the eyesFoamy or frothy urine due to high protein levelsWeight gain from fluid buildupFatigue and low energyHigh blood pressureReduced urine outputLoss of appetitePale, puffy appearanceIncreased risk of infectionsBlood clots in veins or arteriesHigh cholesterol and triglyceride levels in the bloodMuscle cramps or weakness from low blood protein

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 ↗NORD ↗

FDA & Trial Timeline

9 events
Apr 2026An Open-Label Phase 2 Study of N-Acetyl-D-Mannosamine (ManNAc) in Subjects With Primary Focal Segmental Glomerulosclerosis

National Human Genome Research Institute (NHGRI) — PHASE2

TrialRECRUITING
Feb 2026A Study to Find Out if BI 764198 Helps Adults and Adolescents With a Kidney Condition Called Focal Segmental Glomerulosclerosis (FSGS)

Boehringer Ingelheim — PHASE3

TrialRECRUITING
Dec 2025A Study of Praliciguat in Participants With Focal Segmental Glomerulosclerosis (FSGS)

Akebia Therapeutics — PHASE2

TrialRECRUITING
Dec 2024A Study to Evaluate the Efficacy and Safety of Frexalimab, Brivekimig, or Rilzabrutinib in Participants Aged 16 to 75 Years With Primary Focal Segmental Glomerulosclerosis or Minimal Change Disease

Sanofi — PHASE2

TrialRECRUITING
Dec 2023AMPK-activation by Metformin in FSGS: AMP-FSGS

Yale University — PHASE1, PHASE2

TrialRECRUITING
Dec 2022Recurrence Post-transplant Observational Study in Focal Segmental Glomerulosclerosis and Minimal Change Disease

University of Michigan

TrialRECRUITING
Jan 2020Post Approval Study for Treatment of Drug-resistant Adult and Pediatric Primary FSGS Using the LIPOSORBER® LA-15 System

Kaneka Medical America LLC — NA

TrialRECRUITING
Apr 2018Study of Sparsentan in Patients With Primary Focal Segmental Glomerulosclerosis (FSGS)

Travere Therapeutics, Inc. — PHASE3

TrialACTIVE NOT RECRUITING
May 2015Post Approval Study of Liposorber LA-15 System for the Treatment of Focal Segmental Glomerulosclerosis in Children

Kaneka Medical America LLC — NA

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Idiopathic multidrug-resistant nephrotic syndrome.

9 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

9 recruitingView all trials with filters →
Phase 32 trials
Study of Sparsentan in Patients With Primary Focal Segmental Glomerulosclerosis (FSGS)
Phase 3
Active
PI: Radko Komers, MD, PhD (Travere Therapeutics, Inc.) · Sites: Mesa, Arizona; Phoenix, Arizona +223 more · Age: 875 yrs
A Study to Find Out if BI 764198 Helps Adults and Adolescents With a Kidney Condition Called Focal Segmental Glomerulosclerosis (FSGS)
Phase 3
Actively Recruiting
· Sites: Alabaster, Alabama; Huntsville, Alabama +241 more · Age: 1299 yrs
Phase 23 trials
A Study of Praliciguat in Participants With Focal Segmental Glomerulosclerosis (FSGS)
Phase 2
Actively Recruiting
· Sites: Chula Vista, California; Coral Springs, Florida +6 more · Age: 1899 yrs
A Study to Evaluate the Efficacy and Safety of Frexalimab, Brivekimig, or Rilzabrutinib in Participants Aged 16 to 75 Years With Primary Focal Segmental Glomerulosclerosis or Minimal Change Disease
Phase 2
Actively Recruiting
· Sites: Birmingham, Alabama; Orange, California +68 more · Age: 1675 yrs
An Open-Label Phase 2 Study of N-Acetyl-D-Mannosamine (ManNAc) in Subjects With Primary Focal Segmental Glomerulosclerosis
Phase 2
Actively Recruiting
PI: William A Gahl, M.D. (National Human Genome Research Institute (NHGRI)) · Sites: Bethesda, Maryland · Age: 18115 yrs
N/A2 trials
Post Approval Study for Treatment of Drug-resistant Adult and Pediatric Primary FSGS Using the LIPOSORBER® LA-15 System
N/A
Actively Recruiting
· Sites: Loma Linda, California; Loma Linda, California +8 more · Age: 075 yrs
Post Approval Study of Liposorber LA-15 System for the Treatment of Focal Segmental Glomerulosclerosis in Children
N/A
Actively Recruiting
PI: Jeffrey I Silberzweig, MD (Weill Medical College of Cornell University) · Sites: Loma Linda, California; Los Angeles, California +10 more · Age: 021 yrs
Other1 trial
Recurrence Post-transplant Observational Study in Focal Segmental Glomerulosclerosis and Minimal Change Disease
Actively Recruiting
PI: Eloise Salmon, MD (University of Michigan) · Sites: Ann Arbor, Michigan

No specialists are currently listed for Idiopathic multidrug-resistant nephrotic syndrome.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

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

Travel Grants

No travel grants are currently matched to Idiopathic multidrug-resistant nephrotic syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Caregiver Resources

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Mental Health Support

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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.Should I have genetic testing, and which genes should be included in the panel?,What type of kidney damage was found on my biopsy, and what does that mean for my outlook?,Which medications are you recommending, and what are the risks and benefits of each?,How quickly might my kidney function decline, and what signs should I watch for at home?,What dietary changes do I need to make, and should I see a renal dietitian?,At what point should we start discussing dialysis or kidney transplantation?,Are there any clinical trials I might be eligible for?

Common questions about Idiopathic multidrug-resistant nephrotic syndrome

What is Idiopathic multidrug-resistant nephrotic syndrome?

Idiopathic multidrug-resistant nephrotic syndrome (also called idiopathic steroid-resistant nephrotic syndrome or SRNS) is a serious kidney disease where the tiny filtering units in the kidneys, called glomeruli, stop working properly. In a healthy kidney, these filters keep important proteins — especially one called albumin — inside the blood. In this condition, the filters become leaky, allowing large amounts of protein to spill into the urine. This leads to low protein levels in the blood, which causes fluid to build up in the body, especially in the legs, belly, and around the eyes. What

Are there clinical trials for Idiopathic multidrug-resistant nephrotic syndrome?

Yes — 9 recruiting clinical trials are currently listed for Idiopathic multidrug-resistant nephrotic syndrome on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.