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Disease

Nephrotic Syndrome

Late-stage therapeutic developmentEmerging research
MeSHNephrotic Syndrome
1
Publications
20
Clinical trials
12
Associated genes
4
Related proteins
2020
Latest publication
Latest activity
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Recent clinical, regulatory, research and industry developments relating to this disease.

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Low
Key developments
  • 1 clinical trial expected to report results, the earliest in Q2 2028.
  • 5 industry developments reported.
Upcoming Milestones
Clinical Milestones3View
Results expected 1
Recently completed 2
Industry & Market5View
Activity timeline8

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Spironolactoneapproved

Original application approval — In the management of refractory oedema associated with congestive cardiac failure; hepati… (2023)

Lomitapideapproved

Original application approval — Lojuxta is indicated as an adjunct to a low?fat diet and other lipid?lowering medicinal p… (2013)

Clinical trials

14 sponsors · 0 new · 0 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme
20
All trials
6
Active
17
Late-stage
6
Completed
Late-stage studies

The Multi-center, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Rituximab (Genetical Recombination) for the Treatment for Idiopathic Membranous Nephropathy With Nephrotic Syndrome （PRIME Study）

Phase 3 · Active, not recruiting · Shoichi Maruyama MD PhD

Efficacy and Safety of Obinutuzumab Versus Rituximab in Childhood Steroid Dependant and Frequent Relapsing Nephrotic Syndrome : a Double-blind Multicenter Randomized Controlled Study

Phase 2/3 · Recruiting · Assistance Publique - Hôpitaux de Paris

Study of the Telitacicept in Pediatric Patients With Frequently Relapsing or Steroid Dependent Nephrotic Syndrome

Phase 3 · Recruiting · The Children's Hospital of Zhejiang University School of Medicine

FORMA - a Multicenter Randomized-controlled Trial to Evaluate the Efficacy and Safety of Ketoanalogues of Essential Amino Acids in Prophylaxis of Protein-energy Wasting in Nephrotic Syndrome

Phase 3 · Recruiting · Military Institute od Medicine National Research Institute

A Phase III, International, Multicenter, Randomised Open Label Study to Evaluate the Efficacy and Safety of Obinutuzumab Versus MMF in Patients With Childhood Onset Idiopathic Nephrotic Syndrome

Phase 3 · Active, not recruiting · Hoffmann-La Roche

Randomized Clinical Trial to Compare Efficacy and Safety of Repeated Courses of Rituximab to That of Maintenance Mycophenolate Mofetil Following Single Course of Rituximab in Maintaining Remission Over 24 Months Among Children With Steroid Dependent Nephrotic Syndrome

Phase 3 · Completed · Nilratan Sircar Medical College
Recruiting

Efficacy and Safety of Obinutuzumab Versus Rituximab in Childhood Steroid Dependant and Frequent Relapsing Nephrotic Syndrome : a Double-blind Multicenter Randomized Controlled Study

Phase 2/3 · Recruiting · Assistance Publique - Hôpitaux de Paris

Study of the Telitacicept in Pediatric Patients With Frequently Relapsing or Steroid Dependent Nephrotic Syndrome

Phase 3 · Recruiting · The Children's Hospital of Zhejiang University School of Medicine

FORMA - a Multicenter Randomized-controlled Trial to Evaluate the Efficacy and Safety of Ketoanalogues of Essential Amino Acids in Prophylaxis of Protein-energy Wasting in Nephrotic Syndrome

Phase 3 · Recruiting · Military Institute od Medicine National Research Institute

Study of Blinatumomab for the Treatment of Calcineurin Inhibitor-Resistant or Intolerant Steriod-Resistant Nephrotic Syndrome in Pediatric Patients

Phase 1 · Recruiting · The Children's Hospital of Zhejiang University School of Medicine
Recently completed

Randomized Clinical Trial to Compare Efficacy and Safety of Repeated Courses of Rituximab to That of Maintenance Mycophenolate Mofetil Following Single Course of Rituximab in Maintaining Remission Over 24 Months Among Children With Steroid Dependent Nephrotic Syndrome

Phase 3 · Completed · Nilratan Sircar Medical College

Clinical Value of Adding Dapagliflozin in Patients With Nephrotic Syndrome

Phase 4 · Completed · Ain Shams University

Study of Tacrolimus vs Mycophenolate Mofetil in Pediatric Patients With Frequently Relapsing or Steroid Dependent Nephrotic Syndrome: a Randomized, Multicenter, Open-label, Parallel-arm Study

Phase 4 · Completed · The Children's Hospital of Zhejiang University School of Medicine

A Multicenter, Randomised, Double-blind Placebo-controlled Trial Assessing the Efficiency of Levamisole for Maintaining Remission After the First Flare of Steroid Sensitive Nephrotic Syndrome in Children.

Phase 3 · Completed · Assistance Publique - Hôpitaux de Paris

Open-Label, Randomized, Comparative, Multi-Center Clinical Trial on the Therapeutic Effect of Tacrolimus (Prograf Cap.®) in Combination With Low-Dose Corticosteroid Compared With High-Dose Corticosteroid Alone in Patients With Minimal-Change Nephrotic Syndrome (MCNS)

Phase 4 · Completed · Astellas Pharma Korea, Inc.

Rituximab in Patients With Low Dose Steroid-dependent Idiopathic Nephrotic Syndrome

Phase 3 · Completed · Istituto Giannina Gaslini

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

Product approvals and authorisations
2023emaOriginal application approval — Spironolactone· In the management of refractory oedema associated with congestive cardiac failure; hepatic cirrhosis with ascites and oedema, malignant ascites, nephrotic syndrome, diagnosis and treatment of primary aldosteronism, essential hypertension. Neonates, children and adolescents should only be treated under guidance of a paediatric specialist (see sections 5.1 and 5.2). source ↗
2013emaOriginal application approval — Lomitapide· Lojuxta is indicated as an adjunct to a low?fat diet and other lipid?lowering medicinal products with or without low-density-lipoprotein (LDL) apheresis in adult patients with homozygous familial hypercholesterolaemia (HoFH). Genetic confirmation of HoFH should be obtained whenever possible. Other forms of primary hyperlipoproteinaemia and secondary causes of hypercholesterolaemia (e.g. nephrotic syndrome, hypothyroidism) must be excluded. source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Research activity

1 paper

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Leading journals1
  • Pediatric nephrology (Berlin, Germany)1
Leading researchers8
  • Anochie I1
  • Becker JU1
  • Boyer O1
  • Cano F1
  • Feltran L1
  • Gipson D1
  • Haffner D1
  • Hodson E1
Affiliations (unnormalised)6
  • Alberta Children's Hospital1
  • All India Institute of Medical Sciences1
  • Bambino Gesù Pediatric Hospital and Research Center1
  • Bristol Royal Hospital for Children1
  • Center for Pediatrics and Adolescent Medicine1
  • Center for Rare Diseases1

Associated genes

12 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

NPHS20.80COQ8B0.75NPHS10.70NUP1330.64NUP850.64PLCE10.63SLC12A10.63SLC12A30.63AVIL0.63NR3C20.58EMP20.58AXDND10.52

Disease biology

4 matches

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Solute carrier family 12 member 1via SLC12A10.63Solute carrier family 12 member 3via SLC12A30.63Mineralocorticoid receptorvia NR3C20.58Short transient receptor potential channel 6via TRPC60.46

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

A condition characterized by severe PROTEINURIA, greater than 3.5 g/day in an average adult. The substantial loss of protein in the urine results in complications such as HYPOPROTEINEMIA; generalized EDEMA; HYPERTENSION; and HYPERLIPIDEMIAS. Diseases associated with nephrotic syndrome generally cause chronic kidney dysfunction.

Identifiers
MeSHNephrotic Syndrome
References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.