Nephrotic Syndrome Study Network (NEPTUNE)
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ClinicalTrials.gov Identifier: NCT01209000 |
Recruitment Status :
Recruiting
First Posted : September 24, 2010
Last Update Posted : February 1, 2023
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Tracking Information | |||||||||
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First Submitted Date | July 29, 2010 | ||||||||
First Posted Date | September 24, 2010 | ||||||||
Last Update Posted Date | February 1, 2023 | ||||||||
Study Start Date | April 2010 | ||||||||
Estimated Primary Completion Date | June 30, 2024 (Final data collection date for primary outcome measure) | ||||||||
Current Primary Outcome Measures |
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Original Primary Outcome Measures | Same as current | ||||||||
Change History | |||||||||
Current Secondary Outcome Measures |
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Original Secondary Outcome Measures | Same as current | ||||||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||||
Descriptive Information | |||||||||
Brief Title | Nephrotic Syndrome Study Network | ||||||||
Official Title | Nephrotic Syndrome Study Network Under the Rare Diseases Clinical Research Network | ||||||||
Brief Summary | 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. |
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Detailed Description | 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. |
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Study Type | Observational | ||||||||
Study Design | Observational Model: Cohort Time Perspective: Prospective |
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Target Follow-Up Duration | Not Provided | ||||||||
Biospecimen | Retention: Samples With DNA Description: Renal tissue core (from clinically indicated kidney biopsy procedure) Blood products Urine products DNA/RNA specimens (declining consent does not forego participant eligibility)
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Sampling Method | Non-Probability Sample | ||||||||
Study 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. |
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Condition |
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Intervention | 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.
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Study Groups/Cohorts |
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Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||||
Recruitment Status | Recruiting | ||||||||
Estimated Enrollment |
1200 | ||||||||
Original Estimated Enrollment |
450 | ||||||||
Estimated Study Completion Date | June 30, 2024 | ||||||||
Estimated Primary Completion Date | June 30, 2024 (Final data collection date for primary outcome measure) | ||||||||
Eligibility Criteria | 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:
Cohort B (non-biopsy, cNEPTUNE) Inclusion Criteria:
Exclusion Criteria (Cohort A&B):
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Sex/Gender |
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Ages | up to 80 Years (Child, Adult, Older Adult) | ||||||||
Accepts Healthy Volunteers | No | ||||||||
Contacts |
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Listed Location Countries | Canada, United States | ||||||||
Removed Location Countries | |||||||||
Administrative Information | |||||||||
NCT Number | NCT01209000 | ||||||||
Other Study ID Numbers | 6801 1U54DK083912 ( U.S. NIH Grant/Contract ) |
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Has Data Monitoring Committee | Yes | ||||||||
U.S. FDA-regulated Product | Not Provided | ||||||||
IPD Sharing Statement | Not Provided | ||||||||
Current Responsible Party | Matthias Kretzler, University of Michigan | ||||||||
Original Responsible Party | Matthias Kretzler, MD, University of Michigan | ||||||||
Current Study Sponsor | University of Michigan | ||||||||
Original Study Sponsor | Same as current | ||||||||
Collaborators |
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Investigators |
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PRS Account | University of Michigan | ||||||||
Verification Date | January 2023 |