Esta página se tradujo automáticamente y no se garantiza la precisión de la traducción. por favor refiérase a versión inglesa para un texto fuente.

Red de estudio del síndrome nefrótico (NEPTUNE)

5 de noviembre de 2026 actualizado por: Matthias Kretzler, University of Michigan

Red de estudio del síndrome nefrótico bajo la red de investigación clínica de enfermedades raras

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.

Descripción general del estudio

Estado

Reclutamiento

Condiciones

Intervención / Tratamiento

Descripción detallada

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.

Tipo de estudio

De observación

Inscripción (Estimado)

1200

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Estudio Contacto

  • Nombre: Chrysta C. Lienczewski, BS
  • Número de teléfono: 734-615-5021
  • Correo electrónico: NEPTUNE-Study@umich.edu

Copia de seguridad de contactos de estudio

Ubicaciones de estudio

  • Canadá
    • Ontario
      • Richmond Hill, Ontario, Canadá, L4C 4Z3
        • Terminado
        • York Central Hospital
      • Scarborough Village, Ontario, Canadá, M1H 3G4
        • Terminado
        • Scarborough Hospital
      • Toronto, Ontario, Canadá, M4N 3M5
        • Reclutamiento
        • Sunnybrook Hospital
        • Investigador principal:
          • Michelle Hladunewich, MD
        • Contacto:
      • Toronto, Ontario, Canadá, L5M 2N1
        • Reclutamiento
        • Credit Valley Hospital
        • Sub-Investigador:
          • Phillip Boll, MD
        • Contacto:
      • Toronto, Ontario, Canadá, M5G2C4
        • Reclutamiento
        • University Health Network
        • Contacto:
        • Contacto:
        • Investigador principal:
          • Daniel Cattran, MD
        • Sub-Investigador:
          • Heather Reich, MD
        • Sub-Investigador:
          • Michelle Hladunewich, MD
  • Estados Unidos
    • California
      • Los Angeles, California, Estados Unidos, 90227
        • Reclutamiento
        • University of Southern California-Children's Hospital
        • Investigador principal:
          • Kevin Lemley, MD, PhD
        • Contacto:
      • Palo Alto, California, Estados Unidos, 94304
        • Reclutamiento
        • Stanford University School of Medicine
        • Contacto:
        • Investigador principal:
          • Richard Lafayette, MD
      • San Francisco, California, Estados Unidos, 94158
        • Aún no reclutando
        • University of California San Francisco Benioff Children's Hospitals
        • Investigador principal:
          • Paul Brakeman, MD, PhD
        • Contacto:
      • Torrance, California, Estados Unidos, 90502
        • Reclutamiento
        • Lundquist Biomedical Research Institute at Harbor UCLA Medical Center
        • Contacto:
        • Investigador principal:
          • Sharon Adler, MD
    • Colorado
      • Aurora, Colorado, Estados Unidos, 80045
      • Aurora, Colorado, Estados Unidos, 80045
        • Aún no reclutando
        • University of Colorado Anschutz School of Medicine
        • Investigador principal:
          • Amber Podoll, MD
        • Contacto:
    • Florida
      • Miami, Florida, Estados Unidos, 33136
        • Reclutamiento
        • University of Miami Miller School of Medicine
        • Contacto:
          • Carlos Bidot
          • Número de teléfono: 305-243-8973
          • Correo electrónico: cbidot2@miami.edu
        • Investigador principal:
          • Alessia Fornoni, MD. PhD
    • Georgia
      • Atlanta, Georgia, Estados Unidos, 30322
        • Reclutamiento
        • Emory University and Children's Healthcare of Atlanta
        • Investigador principal:
          • Laurence Greenbaum, MD, PhD
        • Contacto:
    • Illinois
      • Chicago, Illinois, Estados Unidos, 60680
        • Reclutamiento
        • John Stroger Cook County Hospital
        • Contacto:
        • Investigador principal:
          • Ambarish Athavale, MD
    • Kansas
      • Kansas City, Kansas, Estados Unidos, 66160
        • Reclutamiento
        • University of Kansas Medical Center
        • Contacto:
        • Investigador principal:
          • Ellen McCarthy, MD
    • Maryland
      • Baltimore, Maryland, Estados Unidos, 21287
        • Reclutamiento
        • Johns Hopkins Medical Institute
        • Contacto:
          • Sara Boynton
          • Número de teléfono: 443-287-9051
          • Correo electrónico: sboynto3@jhmi.edu
        • Investigador principal:
          • Alicia Neu, MD
        • Sub-Investigador:
          • Michael Choi, MD
      • Bethesda, Maryland, Estados Unidos, 20892
        • Terminado
        • Kidney Disease Section, NIDDK, NIH
    • Michigan
      • Ann Arbor, Michigan, Estados Unidos, 48109
        • Reclutamiento
        • University of Michigan Medical Center
        • Contacto:
          • Chrysta C. Lienczewski, BS - adult
          • Número de teléfono: 734-615-5021
          • Correo electrónico: boridley@med.umich.edu
        • Investigador principal:
          • Matthias Kretzler, MD
        • Contacto:
          • Amanda Williams, BS - adult
          • Número de teléfono: 734-615-5017
          • Correo electrónico: amwi@umich.edu
      • Ann Arbor, Michigan, Estados Unidos, 48109
        • Reclutamiento
        • CS Mott Children's Hospital, University of Michigan
        • Sub-Investigador:
          • Debbie Gipson, MD, MS
        • Contacto:
          • Hailey Desmond, MS
          • Número de teléfono: 734-232-4851
          • Correo electrónico: heturner@umich.edu
    • Minnesota
      • Rochester, Minnesota, Estados Unidos, 55905
        • Terminado
        • Mayo Clinic
    • Missouri
      • Kansas City, Missouri, Estados Unidos, 64108
        • Reclutamiento
        • Children's Mercy Hospital
        • Investigador principal:
          • Tarak Srivastava, MD
        • Contacto:
      • St Louis, Missouri, Estados Unidos, 63110
        • Aún no reclutando
        • Washington University - St Louis
        • Contacto:
        • Investigador principal:
          • Vikas Dharnidharka, MD, PhD
    • New York
      • New Hyde Park, New York, Estados Unidos, 11040
        • Reclutamiento
        • Cohen Children's Hospital
        • Investigador principal:
          • Christine Sethna, MD
        • Contacto:
      • New York, New York, Estados Unidos, 10032
        • Reclutamiento
        • Columbia University Medical Center
        • Investigador principal:
          • Andrew Bomback, MD, MPH
        • Contacto:
      • New York, New York, Estados Unidos, 10010
        • Reclutamiento
        • New York University Medical Center
        • Contacto:
        • Investigador principal:
          • Olga Zhadnova, MD
        • Investigador principal:
          • Howard Trachtman, MD
        • Contacto:
      • New York, New York, Estados Unidos, 10016
        • Reclutamiento
        • Bellevue Hospital
        • Contacto:
        • Investigador principal:
          • Laura Barisoni, MD
        • Sub-Investigador:
          • Olga Zhadnova, MD
      • New York, New York, Estados Unidos, 10016
        • Reclutamiento
        • New York University Veterans Administration
        • Contacto:
        • Investigador principal:
          • Laura Barisoni, MD
        • Sub-Investigador:
          • Olga Zhadnova, MD
      • The Bronx, New York, Estados Unidos, 11040
        • Reclutamiento
        • Montefiore Medical Center
        • Contacto:
        • Sub-Investigador:
          • Robert Woroniecki, MD
        • Investigador principal:
          • Frederick Kaskel, MD
    • North Carolina
      • Chapel Hill, North Carolina, Estados Unidos, 27599
        • Reclutamiento
        • University of North Carolina at Chapel Hill
        • Contacto:
        • Investigador principal:
          • Patrick H. Nachman, MD
        • Sub-Investigador:
          • Susan L. Hogan, PhD, MPH
        • Sub-Investigador:
          • Keisha Gibson, MD
        • Contacto:
      • Charlotte, North Carolina, Estados Unidos, 28203
        • Reclutamiento
        • Atrium Health Levine Children's Hospital
        • Investigador principal:
          • Susan Massengill, MD
        • Contacto:
      • Winston-Salem, North Carolina, Estados Unidos, 27157
        • Retirado
        • Wake Forest School of Medicine
    • Ohio
      • Cleveland, Ohio, Estados Unidos, 44195
        • Reclutamiento
        • Cleveland Clinic
        • Sub-Investigador:
          • Katherine Dell, MD
        • Contacto:
        • Contacto:
          • Villarreal
      • Cleveland, Ohio, Estados Unidos, 44106
        • Terminado
        • University Hospital Rainbow Babies & Children's Hospital
      • Cleveland, Ohio, Estados Unidos, 44109
        • Terminado
        • MetroHealth Hospital at Case Western Medical Center
      • Columbus, Ohio, Estados Unidos, 43210
        • Reclutamiento
        • The Ohio State University Wexner Medical Center
        • Contacto:
        • Investigador principal:
          • Salem Almaani, MD
    • Pennsylvania
      • Philadelphia, Pennsylvania, Estados Unidos, 19104
        • Reclutamiento
        • University of Pennsylvania
        • Contacto:
        • Investigador principal:
          • Lawrence Holzman, MD
        • Contacto:
      • Philadelphia, Pennsylvania, Estados Unidos, 19104
        • Reclutamiento
        • Children's Hospital of Philadelphia
        • Sub-Investigador:
          • Kevin Meyers, MD
        • Contacto:
      • Philadelphia, Pennsylvania, Estados Unidos, 19140
        • Reclutamiento
        • Temple University
        • Contacto:
        • Investigador principal:
          • Crystal Gadegbeku, MD
        • Investigador principal:
          • Iris Lee, MD
    • South Carolina
      • Charleston, South Carolina, Estados Unidos, 29425
        • Reclutamiento
        • Medical University of South Carolina
        • Investigador principal:
          • David Selewski, MD, MS
    • Texas
      • Dallas, Texas, Estados Unidos, 75390
        • Reclutamiento
        • University of Texas-Southwestern
        • Investigador principal:
          • Kamal Sambandam, MD
        • Contacto:
      • Houston, Texas, Estados Unidos, 77030
        • Reclutamiento
        • Texas Children's Hospital - Baylor College of Medicine
        • Contacto:
        • Investigador principal:
          • Shweta S. Shah, MD
    • Washington
      • Seattle, Washington, Estados Unidos, 98195
      • Seattle, Washington, Estados Unidos, 98145
        • Reclutamiento
        • Seattle Children's Hospital
        • Sub-Investigador:
          • Sangeeta Hingorani, MD
        • Contacto:
      • Spokane, Washington, Estados Unidos, 99204
        • Reclutamiento
        • Providence Medical Research Center
        • Contacto:
        • Sub-Investigador:
          • Katherine Tuttle, MD
        • Contacto:

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

No mayor que 80 años (Niño, Adulto, Adulto Mayor)

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Todos

Método de muestreo

Muestra no probabilística

Población de estudio

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.

Descripción

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)

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Modelos observacionales: COHORT
  • Perspectivas temporales: PROSPECTIVE

Cohortes e Intervenciones

Grupo / Cohorte
Intervención / Tratamiento
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.
Procedimiento: 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.
Procedimiento: 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.
Procedimiento: 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.

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Event rate of change in urinary protein excretion and renal function.
Periodo de tiempo: 60 months

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

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Quality of Life:
Periodo de tiempo: 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
Periodo de tiempo: 60 months

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

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

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

University of Michigan

Colaboradores

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

Investigadores

  • Investigador principal: Matthias Kretzler, MD, University of Michigan

Publicaciones y enlaces útiles

La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.

Publicaciones Generales

Enlaces Útiles

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

1 de abril de 2010

Finalización primaria (Estimado)

31 de diciembre de 2030

Finalización del estudio (Estimado)

31 de diciembre de 2030

Fechas de registro del estudio

Enviado por primera vez

29 de julio de 2010

Primero enviado que cumplió con los criterios de control de calidad

23 de septiembre de 2010

Publicado por primera vez (Estimado)

24 de septiembre de 2010

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

10 de noviembre de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

5 de noviembre de 2026

Última verificación

1 de noviembre de 2026

Más información

Términos relacionados con este estudio

Palabras clave

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

  • 6801
  • 1U54DK083912 (Subvención/contrato del NIH de EE. UU.)
  • 1R01DK141114-01A1 (Subvención/contrato del NIH de EE. UU.)

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

Suscribir