Overview
Idiopathic steroid-sensitive nephrotic syndrome (ISSNS), sometimes called minimal change nephrotic syndrome or childhood nephrotic syndrome, is a kidney disorder where the tiny filtering units in the kidneys (called glomeruli) become leaky. Normally, these filters keep important proteins — especially one called albumin — inside the blood. In ISSNS, the filters stop working properly and large amounts of protein spill into the urine. This causes a chain reaction: protein levels in the blood drop, fluid leaks out of blood vessels into body tissues, and swelling (called edema) develops, often first noticed around the eyes, ankles, and belly. The word 'idiopathic' means doctors do not know the exact cause. The word 'steroid-sensitive' means the condition responds well to steroid medicines (like prednisone or prednisolone), which is actually a hopeful sign. Most children diagnosed with this condition go into remission — meaning their symptoms disappear — after a course of steroids. However, many children experience relapses, where symptoms come back, sometimes repeatedly over months or years. The main symptoms include puffy eyes (especially in the morning), swollen legs and feet, a swollen belly, foamy or frothy urine, weight gain from fluid, and fatigue. Treatment with corticosteroids is the cornerstone of care, and most patients respond well. Some children eventually outgrow the condition, while others may need long-term management. With proper medical care, serious long-term kidney damage is uncommon in the steroid-sensitive form.
Key symptoms:
Puffy or swollen eyelids, especially noticeable in the morningSwelling of the legs, ankles, and feetSwollen or bloated bellyFoamy or frothy urine (caused by protein in the urine)Unexplained weight gain from fluid buildupFatigue and low energyReduced amount of urineLoss of appetitePale appearanceIncreased risk of infections due to low protein levelsHigh cholesterol levels (found on blood tests)High blood pressure in some cases
Clinical phenotype terms (2)— hover any for plain English
Multifactorial
Caused by a mix of several genes and environmental factors
Childhood
Begins in childhood, roughly ages 1 to 12
FDA & Trial Timeline
3 eventsBio-Thera Solutions — PHASE2, PHASE3
Air Force Military Medical University, China — PHASE3
Prof. Dr. Paul Brinkkoetter
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
No FDA-approved treatments are currently listed for Idiopathic steroid-sensitive nephrotic syndrome.
2 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.
View clinical trials →Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Travel Grants
No travel grants are currently matched to Idiopathic steroid-sensitive nephrotic syndrome.
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Caregiver Resources
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Family & Caregiver Grants
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Social Security Disability
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Questions for your doctor
Bring these to your next appointment
- Q1.How will we know if my child is in remission or having a relapse, and what should I do at home to monitor this?,What are the signs that we should go to the emergency room versus call the office?,How long will my child need to take steroids, and what side effects should I watch for?,If my child relapses frequently, what other medicines are available and what are their risks?,Does my child need a kidney biopsy, and if so, why?,Are there any dietary changes we should make, and do we need to see a dietitian?,What vaccinations does my child need, and are there any we should avoid while on steroids?
Common questions about Idiopathic steroid-sensitive nephrotic syndrome
What is Idiopathic steroid-sensitive nephrotic syndrome?
Idiopathic steroid-sensitive nephrotic syndrome (ISSNS), sometimes called minimal change nephrotic syndrome or childhood nephrotic syndrome, is a kidney disorder where the tiny filtering units in the kidneys (called glomeruli) become leaky. Normally, these filters keep important proteins — especially one called albumin — inside the blood. In ISSNS, the filters stop working properly and large amounts of protein spill into the urine. This causes a chain reaction: protein levels in the blood drop, fluid leaks out of blood vessels into body tissues, and swelling (called edema) develops, often firs
How is Idiopathic steroid-sensitive nephrotic syndrome inherited?
Idiopathic steroid-sensitive nephrotic syndrome follows a multifactorial inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Idiopathic steroid-sensitive nephrotic syndrome typically begin?
Typical onset of Idiopathic steroid-sensitive nephrotic syndrome is childhood. Age of onset can vary across affected individuals.
Are there clinical trials for Idiopathic steroid-sensitive nephrotic syndrome?
Yes — 2 recruiting clinical trials are currently listed for Idiopathic steroid-sensitive nephrotic syndrome on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Idiopathic steroid-sensitive nephrotic syndrome?
17 specialists and care centers treating Idiopathic steroid-sensitive nephrotic syndrome are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.