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Nephrotic Syndrome Study Network

ConditionsMinimal Change Disease (MCD)Membranous NephropathyGlomerulosclerosis, Focal Segmental
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeUp to 80
SponsorUniversity of Michigan

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Age <19 years of age

Initial presentation with <30 days immunosuppression therapy

Disqualifiers

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)

Trial population

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.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • Kidney Biopsy

    Procedure/Surgery

    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.

Treatment groups

1,200 Participants
are divided into 4 treatment groups
Group A: FSGS/MCD Cohort (Cohort A)1 intervention
Group B: MN Cohort (Cohort A)1 intervention
Group C: Other glomerulopathies cohort1 intervention
Group D: cNEPTUNE (Cohort B)0 interventions

Trial outcomes

Primary outcomes

1

Event rate of change in urinary protein excretion and renal function.

Defined as remission, partial remission and non-remission

Time frame
60 months
2

Rate of change in renal function.

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.

Time frame
60 months

Secondary outcomes

1

Quality of Life:

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).

Time frame
60 months
2

Malignancies

Any cancer diagnosis of the skin, hematopoietic system, or solid organ after enrollment in NEPTUNE

Time frame
60 months
3

Infections, Serious and Systemic

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

Time frame
60 months
4

Thromboembolic Events

Documented diagnosis of one of the following: 1. Embolic cerebrovascular accident 2. Deep venous thrombosis 3. Renal vein thrombosis or 4. Pulmonary embolus

Time frame
60 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

University of Michigan

Lead sponsor

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Collaborator

The NephCure Foundation

Collaborator

Source ClinicalTrials.gov
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