Nephrotic Syndrome Study Network



Status:Recruiting
Conditions:Renal Impairment / Chronic Kidney Disease, Nephrology
Therapuetic Areas:Nephrology / Urology
Healthy:No
Age Range:Any - 80
Updated:10/26/2018
Start Date:April 2010
End Date:June 30, 2019
Contact:Chrysta C. Lienczewski, BS
Email:NEPTUNE-Study@umich.edu
Phone:1-877-9-NEPTUNE

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Nephrotic Syndrome Study Network Under the Rare Diseases Clinical Research Network

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.

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.

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)
We found this trial at
31
sites
10 Trench Street
Richmond Hill, Ontario L4C 4Z3
Phone: 905-508-5911
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3550 Jerome Avenue
Bronx, New York 10467
(718) 920-4321
Principal Investigator: Frederick Kaskel, MD
Phone: 718-655-1120
Montefiore Medical Center As the academic medical center and University Hospital for Albert Einstein College...
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Bronx, NY
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Chapel Hill, North Carolina 27599
(919) 962-2211
Principal Investigator: Patrick H. Nachman, MD
Phone: 919-923-1382
University of North Carolina at Chapel Hill Carolina’s vibrant people and programs attest to the...
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9500 Euclid Avenue
Cleveland, Ohio 44106
216.444.2200
Phone: 216-778-4321
Cleveland Clinic Cleveland Clinic is committed to principles as presented in the United Nations Global...
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Cleveland, OH
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2401 Gillham Rd
Kansas City, Missouri 64108
(816) 234-3000
Principal Investigator: Tarak Srivastava, MD
Phone: 816-234-3891
Children's Mercy Hospital Children's Mercy Hospitals and Clinics continues redefining pediatric medicine throughout the Midwest...
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1601 Northwest 12th Avenue
Miami, Florida 33136
(305) 243-6545
Principal Investigator: Alessia Fornoni, MD. PhD
Phone: 305-243-8973
University of Miami Miller School of Medicine The University of Miami Leonard M. Miller School...
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Phone: 484-358-0315
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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Seattle, Washington 98104
(206) 543-2100
Principal Investigator: Peter J. Nelson, MD
Phone: 203-221-3938
Univ of Washington Founded in 1861 by a private gift of 10 acres in what...
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Ann Arbor, Michigan 48109
Phone: 734-232-4851
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Ann Arbor, Michigan 48109
Principal Investigator: Matthias Kretzler, MD
Phone: 734-615-5021
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Atlanta, Georgia 30322
Principal Investigator: Laurence Greenbaum, MD, PhD
Phone: 404-712-9998
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Baltimore, Maryland 21287
Principal Investigator: Alicia Neu, MD
Phone: 443-287-9051
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Bethesda, Maryland 20892
Principal Investigator: Jeffrey P. Kopp, MD
Phone: 301-451-6994
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Chicago, Illinois 60680
Principal Investigator: Ambarish Athavale, MD
Phone: 312-864-4614
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Cleveland, Ohio 44106
Phone: 216-778-4321
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Cleveland, Ohio 44109
Principal Investigator: John Sedor, MI
Phone: 216-778-4321
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Dallas, Texas 75390
Principal Investigator: Kamal Sambandam, MD
Phone: 214-645-8263
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Los Angeles, California 90227
Principal Investigator: Kevin Lemley, MD, PhD
Phone: 323-361-7299
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New Hyde Park, New York 11040
Principal Investigator: Christine Sethna, MD
Phone: 718-470-3499
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New York, New York 10021
Principal Investigator: Olga Zhadnova, MD
Phone: 212-686-7500
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New York, New York 10016
Principal Investigator: Laura Barisoni, MD
Phone: 212-686-7500
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New York, New York 10016
Principal Investigator: Laura Barisoni, MD
Phone: 212-686-7500
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630 W 168th St
New York, New York
212-305-2862
Principal Investigator: Andrew Bomback, MD, MPH
Phone: 212-305-6842
Columbia University Medical Center Situated on a 20-acre campus in Northern Manhattan and accounting for...
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Palo Alto, California 94304
Principal Investigator: Richard Lafayette, MD
Phone: 650-736-1822
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3451 Walnut St
Philadelphia, Pennsylvania 19104
1 (215) 898-5000
Principal Investigator: Lawrence Holzman, MD
Phone: 484-358-0315
Univ of Pennsylvania Penn has a long and proud tradition of intellectual rigor and pursuit...
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1801 N Broad St
Philadelphia, Pennsylvania 19122
(215) 204-7000
Principal Investigator: Crystal Gadegbeku, MD
Phone: 215-707-4712
Temple University Temple University is many things to many people. A place to pursue life's...
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Rochester, Minnesota 55905
Principal Investigator: Fernando Fervenza, MD, PhD
Phone: 507-255-0231
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4800 Sand Point Way NE
Seattle, Washington 98105
(206) 987-2000
Phone: 206-884-1372
Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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Spokane, Washington 99204
Phone: 509-474-4327
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Torrance, California 90502
Principal Investigator: Sharon Adler, MD
Phone: 310-222-4104
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Winston-Salem, North Carolina 27157
Principal Investigator: Jen-Jar Lin, MD
Phone: 336-716-4246
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