Deze pagina is automatisch vertaald en de nauwkeurigheid van de vertaling kan niet worden gegarandeerd. Raadpleeg de Engelse versie voor een brontekst.

Onderzoeksnetwerk nefrotisch syndroom (NEPTUNE)

5 november 2026 bijgewerkt door: Matthias Kretzler, University of Michigan

Onderzoeksnetwerk voor nefrotisch syndroom onder het klinisch onderzoeksnetwerk voor zeldzame ziekten

Minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), and Membranous nephropathy (MN), generate an enormous individual and societal financial burden, accounting for approximately 12% of prevalent end stage renal disease (ESRD) cases (2005) at an annual cost in the US of more than $3 billion. However, the clinical classification of these diseases is widely believed to be inadequate by the scientific community. Given the poor understanding of MCD/FSGS and MN biology, it is not surprising that the available therapies are imperfect. The therapies lack a clear biological basis, and as many families have experienced, they are often not beneficial, and in fact may be significantly toxic. Given these observations, it is essential that research be conducted that address these serious obstacles to effectively caring for patients.

In response to a request for applications by the National Institutes of Health, Office of Rare Diseases (NIH, ORD) for the creation of Rare Disease Clinical Research Consortia, a number of affiliated universities joined together with The NephCure Foundation the NIDDK, the ORDR, and the University of Michigan in collaboration towards the establishment of a Nephrotic Syndrome (NS) Rare Diseases Clinical Research Consortium.

Through this consortium the investigators hope to understand the fundamental biology of these rare diseases and aim to bank long-term observational data and corresponding biological specimens for researchers to access and further enrich.

Studie Overzicht

Toestand

Werving

Conditie

Interventie / Behandeling

Gedetailleerde beschrijving

Idiopathic Nephrotic Syndrome (NS) is a rare disease syndrome responsible for approximately 12% of all causes of end-stage kidney disease (ESRD) and up to 20% of ESRD in children. Treatment strategies for Focal and Segmental Glomerulosclerosis (FSGS), Minimal Change Disease (MCD) and Membranous Nephropathy (MN), the major causes of NS, include high dose prolonged steroid therapy, cyclophosphamide, cyclosporine A, tacrolimus, mycophenolate mofetil and other immunosuppressive agents, which all carry significant side effects. Failure to obtain remission using the current treatment approaches frequently results in progression to ESRD with its associated costs, morbidities, and mortality. In the North American Pediatric Renal Trials and Collaborative Studies (NAPRTCS) registry, half of the pediatric patients with Steroid Resistant Nephrotic Syndrome required renal replacement therapy within two years of being enrolled in the disease registry. FSGS also has a high recurrence rate following kidney transplantation (30-40%) and is the most common recurrent disease leading to allograft loss.

The prevailing classification of Nephrotic Syndrome categorizes patients into FSGS, MCD, and MN, if in the absence of other underlying causes, glomerular histology shows a specific histological pattern. This classification does not adequately predict the heterogeneous natural history of patients with FSGS, MCD, and MN. Major advances in understanding the pathogenesis of FSGS and MCD have come over the last ten years from the identification of several mutated genes responsible for causing Steroid Resistant Nephrotic Syndrome (SRNS) presenting with FSGS or MCD histopathology in humans and model organisms. These functionally distinct genetic disorders can present with indistinguishable FSGS lesions on histology confirming the presence of heterogeneous pathogenic mechanisms under the current histological diagnoses.

The limited understanding of FSGS, MCD, and MN biology in humans has necessitated a descriptive classification system in which heterogeneous disorders are grouped together. This invariably consigns these heterogeneous patients to the same therapeutic approaches, which use blunt immunosuppressive drugs that lack a clear biological basis, are often not beneficial, and are complicated by significant toxicity. The foregoing shortcomings make a strong case that concerted and innovative investigational strategies combining basic science, translational, and clinical methods should be employed to study FSGS, MCD, and MN. It is for these reasons that the Nephrotic Syndrome Study Network is established to conduct clinical and translational research in patients with FSGS/MCD and MN.

Studietype

Observationeel

Inschrijving (Geschat)

1200

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studiecontact

Studie Contact Back-up

Studie Locaties

  • Canada
    • Ontario
      • Richmond Hill, Ontario, Canada, L4C 4Z3
        • Voltooid
        • York Central Hospital
      • Scarborough Village, Ontario, Canada, M1H 3G4
        • Voltooid
        • Scarborough Hospital
      • Toronto, Ontario, Canada, M4N 3M5
      • Toronto, Ontario, Canada, L5M 2N1
        • Werving
        • Credit Valley Hospital
        • Onderonderzoeker:
          • Phillip Boll, MD
        • Contact:
      • Toronto, Ontario, Canada, M5G2C4
        • Werving
        • University Health Network
        • Contact:
        • Contact:
        • Hoofdonderzoeker:
          • Daniel Cattran, MD
        • Onderonderzoeker:
          • Heather Reich, MD
        • Onderonderzoeker:
          • Michelle Hladunewich, MD
  • Verenigde Staten
    • California
      • Los Angeles, California, Verenigde Staten, 90227
        • Werving
        • University of Southern California-Children's Hospital
        • Hoofdonderzoeker:
          • Kevin Lemley, MD, PhD
        • Contact:
      • Palo Alto, California, Verenigde Staten, 94304
        • Werving
        • Stanford University School of Medicine
        • Contact:
        • Hoofdonderzoeker:
          • Richard Lafayette, MD
      • San Francisco, California, Verenigde Staten, 94158
        • Nog niet aan het werven
        • University of California San Francisco Benioff Children's Hospitals
        • Hoofdonderzoeker:
          • Paul Brakeman, MD, PhD
        • Contact:
      • Torrance, California, Verenigde Staten, 90502
        • Werving
        • Lundquist Biomedical Research Institute at Harbor UCLA Medical Center
        • Contact:
        • Hoofdonderzoeker:
          • Sharon Adler, MD
    • Colorado
      • Aurora, Colorado, Verenigde Staten, 80045
      • Aurora, Colorado, Verenigde Staten, 80045
        • Nog niet aan het werven
        • University of Colorado Anschutz School of Medicine
        • Hoofdonderzoeker:
          • Amber Podoll, MD
        • Contact:
    • Florida
      • Miami, Florida, Verenigde Staten, 33136
        • Werving
        • University of Miami Miller School of Medicine
        • Contact:
        • Hoofdonderzoeker:
          • Alessia Fornoni, MD. PhD
    • Georgia
      • Atlanta, Georgia, Verenigde Staten, 30322
        • Werving
        • Emory University and Children's Healthcare of Atlanta
        • Hoofdonderzoeker:
          • Laurence Greenbaum, MD, PhD
        • Contact:
    • Illinois
      • Chicago, Illinois, Verenigde Staten, 60680
        • Werving
        • John Stroger Cook County Hospital
        • Contact:
        • Hoofdonderzoeker:
          • Ambarish Athavale, MD
    • Kansas
      • Kansas City, Kansas, Verenigde Staten, 66160
        • Werving
        • University of Kansas Medical Center
        • Contact:
        • Hoofdonderzoeker:
          • Ellen McCarthy, MD
    • Maryland
      • Baltimore, Maryland, Verenigde Staten, 21287
        • Werving
        • Johns Hopkins Medical Institute
        • Contact:
        • Hoofdonderzoeker:
          • Alicia Neu, MD
        • Onderonderzoeker:
          • Michael Choi, MD
      • Bethesda, Maryland, Verenigde Staten, 20892
        • Voltooid
        • Kidney Disease Section, NIDDK, NIH
    • Michigan
      • Ann Arbor, Michigan, Verenigde Staten, 48109
        • Werving
        • University of Michigan Medical Center
        • Contact:
        • Hoofdonderzoeker:
          • Matthias Kretzler, MD
        • Contact:
          • Amanda Williams, BS - adult
          • Telefoonnummer: 734-615-5017
          • E-mail: amwi@umich.edu
      • Ann Arbor, Michigan, Verenigde Staten, 48109
        • Werving
        • CS Mott Children's Hospital, University of Michigan
        • Onderonderzoeker:
          • Debbie Gipson, MD, MS
        • Contact:
    • Minnesota
      • Rochester, Minnesota, Verenigde Staten, 55905
        • Voltooid
        • Mayo Clinic
    • Missouri
      • Kansas City, Missouri, Verenigde Staten, 64108
        • Werving
        • Children's Mercy Hospital
        • Hoofdonderzoeker:
          • Tarak Srivastava, MD
        • Contact:
      • St Louis, Missouri, Verenigde Staten, 63110
        • Nog niet aan het werven
        • Washington University - St Louis
        • Contact:
        • Hoofdonderzoeker:
          • Vikas Dharnidharka, MD, PhD
    • New York
      • New Hyde Park, New York, Verenigde Staten, 11040
        • Werving
        • Cohen Children's Hospital
        • Hoofdonderzoeker:
          • Christine Sethna, MD
        • Contact:
      • New York, New York, Verenigde Staten, 10032
        • Werving
        • Columbia University Medical Center
        • Hoofdonderzoeker:
          • Andrew Bomback, MD, MPH
        • Contact:
      • New York, New York, Verenigde Staten, 10010
        • Werving
        • New York University Medical Center
        • Contact:
        • Hoofdonderzoeker:
          • Olga Zhadnova, MD
        • Hoofdonderzoeker:
          • Howard Trachtman, MD
        • Contact:
      • New York, New York, Verenigde Staten, 10016
        • Werving
        • Bellevue Hospital
        • Contact:
        • Hoofdonderzoeker:
          • Laura Barisoni, MD
        • Onderonderzoeker:
          • Olga Zhadnova, MD
      • New York, New York, Verenigde Staten, 10016
        • Werving
        • New York University Veterans Administration
        • Contact:
        • Hoofdonderzoeker:
          • Laura Barisoni, MD
        • Onderonderzoeker:
          • Olga Zhadnova, MD
      • The Bronx, New York, Verenigde Staten, 11040
        • Werving
        • Montefiore Medical Center
        • Contact:
        • Onderonderzoeker:
          • Robert Woroniecki, MD
        • Hoofdonderzoeker:
          • Frederick Kaskel, MD
    • North Carolina
      • Chapel Hill, North Carolina, Verenigde Staten, 27599
        • Werving
        • University of North Carolina at Chapel Hill
        • Contact:
        • Hoofdonderzoeker:
          • Patrick H. Nachman, MD
        • Onderonderzoeker:
          • Susan L. Hogan, PhD, MPH
        • Onderonderzoeker:
          • Keisha Gibson, MD
        • Contact:
      • Charlotte, North Carolina, Verenigde Staten, 28203
        • Werving
        • Atrium Health Levine Children's Hospital
        • Hoofdonderzoeker:
          • Susan Massengill, MD
        • Contact:
      • Winston-Salem, North Carolina, Verenigde Staten, 27157
        • Ingetrokken
        • Wake Forest School of Medicine
    • Ohio
      • Cleveland, Ohio, Verenigde Staten, 44195
        • Werving
        • Cleveland Clinic
        • Onderonderzoeker:
          • Katherine Dell, MD
        • Contact:
        • Contact:
          • Villarreal
      • Cleveland, Ohio, Verenigde Staten, 44106
        • Voltooid
        • University Hospital Rainbow Babies & Children's Hospital
      • Cleveland, Ohio, Verenigde Staten, 44109
        • Voltooid
        • MetroHealth Hospital at Case Western Medical Center
      • Columbus, Ohio, Verenigde Staten, 43210
        • Werving
        • The Ohio State University Wexner Medical Center
        • Contact:
        • Hoofdonderzoeker:
          • Salem Almaani, MD
    • Pennsylvania
      • Philadelphia, Pennsylvania, Verenigde Staten, 19104
        • Werving
        • University of Pennsylvania
        • Contact:
        • Hoofdonderzoeker:
          • Lawrence Holzman, MD
        • Contact:
      • Philadelphia, Pennsylvania, Verenigde Staten, 19104
        • Werving
        • Children's Hospital of Philadelphia
        • Onderonderzoeker:
          • Kevin Meyers, MD
        • Contact:
      • Philadelphia, Pennsylvania, Verenigde Staten, 19140
        • Werving
        • Temple University
        • Contact:
        • Hoofdonderzoeker:
          • Crystal Gadegbeku, MD
        • Hoofdonderzoeker:
          • Iris Lee, MD
    • South Carolina
      • Charleston, South Carolina, Verenigde Staten, 29425
        • Werving
        • Medical University of South Carolina
        • Hoofdonderzoeker:
          • David Selewski, MD, MS
    • Texas
      • Dallas, Texas, Verenigde Staten, 75390
      • Houston, Texas, Verenigde Staten, 77030
        • Werving
        • Texas Children's Hospital - Baylor College of Medicine
        • Contact:
        • Hoofdonderzoeker:
          • Shweta S. Shah, MD
    • Washington
      • Seattle, Washington, Verenigde Staten, 98195
      • Seattle, Washington, Verenigde Staten, 98145
      • Spokane, Washington, Verenigde Staten, 99204

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

Niet ouder dan 80 jaar (Kind, Volwassen, Oudere volwassene)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Bemonsteringsmethode

Niet-waarschijnlijkheidssteekproef

Studie Bevolking

Patients with signs and symptoms of kidney disease consistent with FSGS, MCD, MN or proteinuric renal disease or pediatric participants not previously biopsied, who present for patient care at participating clinical centers will be eligible for Cohort A (biopsy cohort) study population targeted for enrollment into the NEPTUNE study.

To establish a cohort of pediatric participants with incident nephrotic syndrome, Cohort B, a non-biopsy cohort has been initiated for Protocol V4.0. This population, <19 years of age, presenting for nephrotic syndrome and less than 30 days of immunosuppression therapy exposure, will also be targeted for enrollment into the cNEPTUNE study.

Potential participants willing to receive their initial care and subsequent follow-up study visits at one of these sites are welcome to participate.

Beschrijving

Cohort A (biopsy cohort) Inclusion Criteria:

Patients presenting with an incipient clinical diagnosis for FSGS/MCD or MN or pediatric participants not previously biopsied, with a clinical diagnosis for FSGS/MCD or MN meeting the following inclusion criteria:

  • Documented urinary protein excretion ≥1500 mg/24 hours or spot protein: creatinine ratio equivalent at the time of diagnosis or within 3 months of the screening/eligibility visit.
  • Scheduled renal biopsy

Cohort B (non-biopsy, cNEPTUNE) Inclusion Criteria:

  • Age <19 years of age

  • Initial presentation with <30 days immunosuppression therapy

  • Proteinuria/nephrotic

    • UA>2+ and edema OR
    • UA>2+ and serum albumin <3 OR
    • UPC > 2g/g and serum albumin <3

Exclusion Criteria (Cohort A&B):

  • Prior solid organ transplant
  • A clinical diagnosis of glomerulopathy without diagnostic renal biopsy
  • Clinical, serological or histological evidence of systemic lupus erythematosus (SLE) as defined by the ARA criteria. Patients with membranous in combination with SLE will be excluded because this entity is well defined within the International Society of Nephrology/Renal Pathology Society categories of lupus nephritis, and frequently overlaps with other classification categories of SLE nephritis (68)
  • Clinical or histological evidence of other renal diseases (Alport, Nail Patella, Diabetic Nephropathy, IgA-nephritis, monoclonal gammopathy (multiple myelomas), genito-urinary malformations with vesico-urethral reflux or renal dysplasia)
  • Known systemic disease diagnosis at time of enrollment with a life expectancy less than 6 months
  • Unwillingness or inability to give a comprehensive informed consent
  • Unwillingness to comply with study procedures and visit schedule
  • Institutionalized individuals (e.g., prisoners)

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Observatiemodellen: COHORT
  • Tijdsperspectieven: PROSPECTIVE

Cohorten en interventies

Groep / Cohort
Interventie / Behandeling
FSGS/MCD Cohort (Cohort A)

Focal Segmental Glomerulosclerosis/Minimal Change Disease (FSGS/MCD) Cohort

Participants enrolled in NEPTUNE with a biopsy proven histological diagnosis for FSGS or MCD.

Eligible participants must be scheduled for a clinically indicated renal biopsy.
Procedure: Kidney Biopsy

Patients scheduled to undergo a clinically indicated kidney biopsy will be requested to consent to an additional renal core, to be set aside until all clinical care is complete.
MN Cohort (Cohort A)

Membranous Nephropathy (MN) Cohort

Participants enrolled in NEPTUNE with a biopsy proven histological diagnosis for MN.

Eligible participants must be scheduled for a clinically indicated renal biopsy.
Procedure: Kidney Biopsy

Patients scheduled to undergo a clinically indicated kidney biopsy will be requested to consent to an additional renal core, to be set aside until all clinical care is complete.
Other glomerulopathies cohort

Participants enrolled in NEPTUNE and determined to not have FSGS/MCD or MN will be followed in a third group.

Eligible participants must be scheduled for a clinically indicated renal biopsy.
Procedure: Kidney Biopsy

Patients scheduled to undergo a clinically indicated kidney biopsy will be requested to consent to an additional renal core, to be set aside until all clinical care is complete.
cNEPTUNE (Cohort B)

Participants < 19 years of age, with < 30 days exposure to immunosuppression therapy who are not scheduled for renal biopsy.

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Event rate of change in urinary protein excretion and renal function.
Tijdsspanne: 60 months

Defined as remission, partial remission and non-remission
60 months
Rate of change in renal function.
Tijdsspanne: 60 months

Defined as:

  1. 25 mls/min/1.73m2 reduction in follow-up estimated GFR (using the 4-variable MDRD equation for ages ≥18 years and modified Schwartz for ages <18 years) compared to baseline estimated GFR
  2. 50% decline in follow-up estimated GFR compared to baseline measurement
  3. End stage renal disease defined as estimated GFR ≤10cc/min, initiation of maintenance dialysis or preemptive kidney transplantation.
60 months

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Quality of Life:
Tijdsspanne: 60 months

Patient-reported outcome will be assessed using Quality of Life questionnaires at regular intervals as stipulated in the visit calendar using the SF-36, PedsQL and the Patient Reported Outcome Measurement Information System (PROMIS) (in the age-appropriate groups).
60 months
Malignancies
Tijdsspanne: 60 months

Any cancer diagnosis of the skin, hematopoietic system, or solid organ after enrollment in NEPTUNE
60 months
Infections, Serious and Systemic
Tijdsspanne: 60 months

Infections including one of the following:

  1. Documented diagnosis of infection of the skin or subcutaneous tissue (e.g. cellulitis), vascular system, peritoneum, or any vital organ requiring the use of parenteral antibiotics and/or oral antibiotics alone or in combination for a treatment interval of ≥72 hours.
  2. Hospitalization for treatment of infection
60 months
Thromboembolic Events
Tijdsspanne: 60 months

Documented diagnosis of one of the following:

  1. Embolic cerebrovascular accident
  2. Deep venous thrombosis
  3. Renal vein thrombosis or
  4. Pulmonary embolus
60 months
Hospitalization
Tijdsspanne: 60 months

Documented hospital admission, including observation for ≥24 hours.
60 months
Emergency Department/ Observation Unit Visit
Tijdsspanne: 60 months

Documented visit to an emergency department or observation unit that does not lead to hospitalization and is less than 24 hours.
60 months
Acute Kidney Injury
Tijdsspanne: 60 months

Documented diagnosis of acute kidney injury as defined by the AKIN (Mehta et al., Critical Care 2007, 11:R31) and/or renal failure requiring renal replacement therapy <3 months.
60 months
Death
Tijdsspanne: 60 months
  1. Documentation of death that is secondary to infection or sepsis.
  2. Cardiovascular/Cerebrovascular-related Death: Sudden death; Myocardial infarction; Congestive heart failure; Primary intractable serious arrhythmia; Peripheral vascular disease; Ischemic cerebrovascular accident; Hemorrhagic cerebrovascular accident; Thromboembolic event
  3. Documentation of death secondary to cancer
  4. Other Death: Documentation of death that does not fall into the above categories.
60 months
New Onset Diabetes
Tijdsspanne: 60 months

Diagnosis of diabetes as indicated by 1 or more of the following not present at NEPTUNE Enrollment:

  1. Documented diagnosis of diabetes in medical record
  2. Casual (non-fasting) blood glucose > 200 mg/dL c) Fasting blood glucose > 126 mg/dL d) 2 hour glucose > 200 after oral glucose tolerance test e) chronic use (>6 mos) hypoglycemic therapy outside of pregnancy f) Hemogloblin A1C >= 6.5%
60 months

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

University of Michigan

Medewerkers

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
The NephCure Foundation

Onderzoekers

  • Hoofdonderzoeker: Matthias Kretzler, MD, University of Michigan

Publicaties en nuttige links

De persoon die verantwoordelijk is voor het invoeren van informatie over het onderzoek stelt deze publicaties vrijwillig ter beschikking. Dit kan gaan over alles wat met het onderzoek te maken heeft.

Algemene publicaties

Nuttige links

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

1 april 2010

Primaire voltooiing (Geschat)

31 december 2030

Studie voltooiing (Geschat)

31 december 2030

Studieregistratiedata

Eerst ingediend

29 juli 2010

Eerst ingediend dat voldeed aan de QC-criteria

23 september 2010

Eerst geplaatst (Geschat)

24 september 2010

Updates van studierecords

Laatste update geplaatst (Werkelijk)

10 november 2026

Laatste update ingediend die voldeed aan QC-criteria

5 november 2026

Laatst geverifieerd

1 november 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • 6801
  • 1U54DK083912 (Subsidie/contract van de Amerikaanse NIH)
  • 1R01DK141114-01A1 (Subsidie/contract van de Amerikaanse NIH)

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

Abonneren