NCT01209000

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.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1,200

participants targeted

Target at P75+ for all trials

Timeline
38mo left

Started Apr 2010

Longer than P75 for all trials

Geographic Reach
2 countries

44 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress84%
Apr 2010Jun 2029

Study Start

First participant enrolled

April 1, 2010

Completed
4 months until next milestone

First Submitted

Initial submission to the registry

July 29, 2010

Completed
2 months until next milestone

First Posted

Study publicly available on registry

September 24, 2010

Completed
18.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 30, 2029

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 30, 2029

Last Updated

April 24, 2025

Status Verified

April 1, 2025

Enrollment Period

19.3 years

First QC Date

July 29, 2010

Last Update Submit

April 18, 2025

Conditions

Keywords

Focal and Segmental GlomerulosclerosisFocal & Segmental GlomerulosclerosisFocal Segmental GlomerulosclerosisFSGSMinimal change diseaseMCDMembranous NephropathyMNNephrotic SyndromeNeph SyndromeNEPTUNENephCureHalpin

Outcome Measures

Primary Outcomes (2)

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

    Defined as remission, partial remission and non-remission

    60 months

  • 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.

    60 months

Secondary Outcomes (9)

  • Quality of Life:

    60 months

  • Malignancies

    60 months

  • Infections, Serious and Systemic

    60 months

  • Thromboembolic Events

    60 months

  • Hospitalization

    60 months

  • +4 more secondary outcomes

Study Arms (4)

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

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

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

cNEPTUNE (Cohort B)

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

Interventions

Kidney BiopsyPROCEDURE

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.

FSGS/MCD Cohort (Cohort A)MN Cohort (Cohort A)Other glomerulopathies cohort

Eligibility Criteria

AgeUp to 80 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-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.

You may qualify if:

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

You may not qualify if:

  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (44)

University of Southern California-Children's Hospital

Los Angeles, California, 90227, United States

RECRUITING

Stanford University School of Medicine

Palo Alto, California, 94304, United States

RECRUITING

University of California San Francisco Benioff Children's Hospitals

San Francisco, California, 94158, United States

NOT YET RECRUITING

Lundquist Biomedical Research Institute at Harbor UCLA Medical Center

Torrance, California, 90502, United States

RECRUITING

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

NOT YET RECRUITING

University of Colorado Anschutz School of Medicine

Aurora, Colorado, 80045, United States

NOT YET RECRUITING

University of Miami Miller School of Medicine

Miami, Florida, 33136, United States

RECRUITING

Emory University and Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States

RECRUITING

John Stroger Cook County Hospital

Chicago, Illinois, 60680, United States

RECRUITING

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

RECRUITING

Johns Hopkins Medical Institute

Baltimore, Maryland, 21287, United States

RECRUITING

Kidney Disease Section, NIDDK, NIH

Bethesda, Maryland, 20892, United States

COMPLETED

CS Mott Children's Hospital, University of Michigan

Ann Arbor, Michigan, 48109, United States

RECRUITING

University of Michigan Medical Center

Ann Arbor, Michigan, 48109, United States

RECRUITING

Mayo Clinic

Rochester, Minnesota, 55905, United States

COMPLETED

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

RECRUITING

Washington University - St Louis

St Louis, Missouri, 63110, United States

NOT YET RECRUITING

Cohen Children's Hospital

New Hyde Park, New York, 11040, United States

RECRUITING

New York University Medical Center

New York, New York, 10010, United States

RECRUITING

Bellevue Hospital

New York, New York, 10016, United States

RECRUITING

New York University Veterans Administration

New York, New York, 10016, United States

RECRUITING

Columbia University Medical Center

New York, New York, 10032, United States

RECRUITING

Montefiore Medical Center

The Bronx, New York, 11040, United States

RECRUITING

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

RECRUITING

Atrium Health Levine Children's Hospital

Charlotte, North Carolina, 28203, United States

RECRUITING

Wake Forest School of Medicine

Winston-Salem, North Carolina, 27157, United States

WITHDRAWN

University Hospital Rainbow Babies & Children's Hospital

Cleveland, Ohio, 44106, United States

COMPLETED

MetroHealth Hospital at Case Western Medical Center

Cleveland, Ohio, 44109, United States

COMPLETED

Cleveland Clinic

Cleveland, Ohio, 44195, United States

RECRUITING

The Ohio State University Wexner Medical Center

Columbus, Ohio, 43210, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Temple University

Philadelphia, Pennsylvania, 19140, United States

RECRUITING

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

RECRUITING

University of Texas-Southwestern

Dallas, Texas, 75390, United States

RECRUITING

Texas Children's Hospital - Baylor College of Medicine

Houston, Texas, 77030, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98145, United States

RECRUITING

University of Washington

Seattle, Washington, 98195, United States

RECRUITING

Providence Medical Research Center

Spokane, Washington, 99204, United States

RECRUITING

York Central Hospital

Richmond Hill, Ontario, L4C 4Z3, Canada

COMPLETED

Scarborough Hospital

Scarborough Village, Ontario, M1H 3G4, Canada

COMPLETED

Credit Valley Hospital

Toronto, Ontario, L5M 2N1, Canada

RECRUITING

Sunnybrook Hospital

Toronto, Ontario, M4N 3M5, Canada

RECRUITING

University Health Network

Toronto, Ontario, M5G2C4, Canada

RECRUITING

Related Publications (4)

  • Wang CS, Troost JP, Wang Y, Greenbaum LA, Gibson K, Trachtman H, Srivastava T, Reidy K, Kaskel F, Sethna CB, Meyers K, Dell KM, Tran CL, Hingorani S, Lemley KV, Lin JJ, Gipson DS. Determinants of medication adherence in childhood nephrotic syndrome and associations of adherence with clinical outcomes. Pediatr Nephrol. 2022 Jul;37(7):1585-1595. doi: 10.1007/s00467-021-05176-8. Epub 2021 Nov 18.

  • Solagna F, Tezze C, Lindenmeyer MT, Lu S, Wu G, Liu S, Zhao Y, Mitchell R, Meyer C, Omairi S, Kilic T, Paolini A, Ritvos O, Pasternack A, Matsakas A, Kylies D, Wiesch JSZ, Turner JE, Wanner N, Nair V, Eichinger F, Menon R, Martin IV, Klinkhammer BM, Hoxha E, Cohen CD, Tharaux PL, Boor P, Ostendorf T, Kretzler M, Sandri M, Kretz O, Puelles VG, Patel K, Huber TB. Pro-cachectic factors link experimental and human chronic kidney disease to skeletal muscle wasting programs. J Clin Invest. 2021 Jun 1;131(11):e135821. doi: 10.1172/JCI135821.

  • Kang H, Kim DR, Jung YH, Baek CW, Park YH, In Oh J, Kim WJ, Choi GJ. Pre-warming the Streamlined Liner of the Pharynx Airway (SLIPA) improves fitting to the laryngeal structure: a randomized, double-blind study. BMC Anesthesiol. 2015 Nov 20;15:167. doi: 10.1186/s12871-015-0151-4.

  • Hogan MC, Lieske JC, Lienczewski CC, Nesbitt LL, Wickman LT, Heyer CM, Harris PC, Ward CJ, Sundsbak JL, Manganelli L, Ju W, Kopp JB, Nelson PJ, Adler SG, Reich HN, Holzmann LB, Kretzler M, Bitzer M. Strategy and rationale for urine collection protocols employed in the NEPTUNE study. BMC Nephrol. 2015 Nov 17;16:190. doi: 10.1186/s12882-015-0185-3.

Related Links

Biospecimen

Retention: SAMPLES WITH DNA

Renal tissue core (from clinically indicated kidney biopsy procedure) Blood products Urine products DNA/RNA specimens (declining consent does not forego participant eligibility)

MeSH Terms

Conditions

Nephrosis, LipoidGlomerulonephritis, MembranousGlomerulosclerosis, Focal SegmentalMacular dystrophy, corneal type 1Nephrotic Syndrome

Condition Hierarchy (Ancestors)

NephrosisKidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesGlomerulonephritisNephritisAutoimmune DiseasesImmune System Diseases

Study Officials

  • Matthias Kretzler, MD

    University of Michigan

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Chrysta C. Lienczewski, BS

CONTACT

Amanda Williams, BS

CONTACT

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

July 29, 2010

First Posted

September 24, 2010

Study Start

April 1, 2010

Primary Completion (Estimated)

June 30, 2029

Study Completion (Estimated)

June 30, 2029

Last Updated

April 24, 2025

Record last verified: 2025-04

Locations