Clinical Trial on Minimal Change Disease, Membranous Nephropathy, C3 Glomerulopathy, Alport Syndrome, IgA Nephropathy (IgAN), IgA Vasculitis - Clinical Trials Registry

Study Overview

Status

Recruiting

Conditions

Detailed Description

This study is designed to look at the natural history of glomerular diseases in children and adolescents in order to direct the research to find cures. This study will also possibly help identify or locate participants that may be eligible for clinical trials.

Mixed retrospective-prospective cohort study (longitudinal, observational, non-interventional study) to research the natural history of glomerular disease in children and adolescents in the US.

Objective: Evaluate the natural history of glomerular disease in children in the United States.

Aim 1: To investigate and determine important relationships among patient/disease characteristics, natural history, treatments, histopathology, and outcomes among pediatric patients with glomerular disease, both across and within pre-specified disease cohorts (see below), by means of multicenter collection, storage, and analysis of longitudinal data.

Aim 2: To provide a platform for and facilitate, with participant consent, rapid assessment of potential participant eligibility for planned and/or ongoing investigator-initiated and/or industry-sponsored clinical trials of pediatric glomerular disease.

20 initial sites were selected to recruit participants with glomerular disease. Patients diagnosed starting in July 2023, as well as newly diagnosed patients can be enrolled. Participants will be followed until 21 years of age.

Data Collection at 6 month timepoints +/- 30 days. Collection starts with demographic information, medical history, and family medical history. Also collected at enrollment and at each time point are standard of care labs, hospitalizations, renal biopsy images, pathology reports and specialized or genetic testing. Participants will be asked to answer quality of life and social determinate of health questionnaires at enrollment and annually. These are optional and will not affect eligibility.

Enrollment expected to be 500 participants per year.

Study Type

Observational

Enrollment (Estimated)

1000

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Name: Amy Dutcher, MCR
Phone Number: 6145547621
Email: adutcher@PNRConsortium.org

Study Contact Backup

Name: Teri Crumb, MSN, RN, CCRC
Phone Number: 616-607-4430
Email: teri@pnrconsortium.org

Study Locations

United States
- Massachusetts
  - Boston, Massachusetts, United States, 02115
    - Recruiting
    - Boston Children's Hospital
    - Contact:
      
      Natalie Pursel
      
      Phone Number: 617-355-6762
      
      Email: getpluggedin@pnrconsortium.org
    - Contact:
      
      Swethapriya Damagatla
      
      Phone Number: 617-355-6762
      
      Email: getpluggedin@pnrconsortium.org
    - Principal Investigator:
      
      Ankana Daga, MD
- Ohio
  - Cincinnati, Ohio, United States, 45229
    - Recruiting
    - Cincinnati Children's Hospital Medical Center
    - Contact:
      
      Libby Siry, CRP
      
      Phone Number: 513.517.1439
      
      Email: getpluggedin@pnrconsortium.org
    - Contact:
      
      Nina Kanis, CRC
      
      Phone Number: 513.517.1439
      
      Email: getpluggedin@pnrconsortium.org
    - Principal Investigator:
      
      Elif Erkan, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

The study population will be less than 18 years of age at time of diagnosis and consent, categorized into the following prespecified disease cohorts:

IgA Nephropathy
IgA Vasculitis
Idiopathic Immune Complex Glomerulonephritis (ex: IC-MPGN, Full house GN without diagnosis of Lupus, etc.)
C3 Glomerulopathy
Membranous Nephropathy
Focal and Segmental Glomerulosclerosis
Minimal Change Disease
Anti-Neutrophilic Cytoplasmic antibody-GN (ANCA)
Lupus Nephritis
Alport Syndrome
Infection-related or post-infectious Glomerulonephritis (with or without biopsy)
Idiopathic Nephrotic Syndrome (with or without biopsy)

Description

Inclusion Criteria:

Children with biopsy-proven glomerular disease or populations with a clinical diagnosis of glomerular disease

Exclusion Criteria:

Patients who declined (or unable to provide informed consent, + informed assent of minors) to participate Patients with End Stage Kidney Disease (Stage 5 CKD, eGFR < 15mL/min/1.73m2)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Observational Models: COHORT
Time Perspectives: OTHER

Number of groups / cohorts

12

Cohorts and Interventions

Group / Cohort
IgA Nephropathy (with biopsy) Subjects diagnosed with IgA Nephropathy less than 18 years of age
IgA Vasculitis (with biopsy) Subjects diagnosed with IgA Vasculitis less than 18 years of age
Idiopathic Immune Complex Glomerulonephritis (with biopsy) Subjects diagnosed with Idiopathic Immune Complex Glomerulonephritis less than 18 years of age
C3 Glomerulopathy (with biopsy) Subjects diagnosed with C3 Glomerulopathy less than 18 years of age
Membranous Nephropathy (with biopsy) Subjects diagnosed with Membranous Nephropathy less than 18 years of age
Focal and Segmental Glomerulosclerosis (with biopsy) Subjects diagnosed with Focal and Segmental Glomerulosclerosis less than 18 years of age
Minimal Change Disease (with biopsy) Subjects diagnosed with Minimal Change Disease less than 18 years of age
Anti-Neutrophilic Cytoplasmic antibody-GN (ANCA) (with biopsy) Subjects diagnosed with Anti-Neutrophilic Cytoplasmic antibody-GN (ANCA) less than 18 years of age
Lupus Nephritis (with biopsy) Subjects diagnosed with Lupus Nephritis less than 18 years of age
Alport Syndrome (with biopsy) Subjects diagnosed with Alport Syndrome less than 18 years of age
Infection-related or post-infectious Glomerulonephritis (with or without biopsy) Subjects diagnosed with Infection-related or post-infectious Glomerulonephritis (with or without biopsy) less than 18 years of age
Idiopathic Nephrotic Syndrome (with or without biopsy) Subjects diagnosed with Idiopathic Nephrotic Syndrome (with or without biopsy) less than 18 years of age

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proteinuria, and change over time thereof (urine protein-creatinine ratio) Time Frame: Baseline and follow up timepoints (every 6 months) post-diagnosis to be reviewed at end of years 1 and 2 of data collection	To characterize the longitudinal trajectory and progression of proteinuria, measured by urine protein-creatinine ratio (UPCR), following diagnosis and to identify baseline demographic and clinical factors associated with changes in proteinuria over time.	Baseline and follow up timepoints (every 6 months) post-diagnosis to be reviewed at end of years 1 and 2 of data collection

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent change in estimated glomerular filtration rate (eGFR) from baseline to follow up Time Frame: Two years after study initiation	To characterize longitudinal changes in kidney function as measured by estimated glomerular filtration rate (eGFR) and to identify baseline demographic and clinical factors associated with changes in kidney function over time.	Two years after study initiation
eGFR, and change (from baseline) overtime Time Frame: Two years after study initiation	Measures how much a patient's kidney filtering capacity has increased or decreased over time compared to their initial starting measurement	Two years after study initiation
Growth trajectory, derived from height and weight Time Frame: Two years after study initiation	Describe longitudinal growth patterns (e.g., height, weight, BMI z -scores) and identify disease-related factors associated with impaired growth trajectories. Growth trajectory will be defined as change in height and weight over time. We will calculate height-for-weight Z score, weight-for-height Z score and BMI Z-score over time.	Two years after study initiation
Developmental trajectory derived from QOL survey of caregiver Time Frame: Two years after study initiation	Characterizing developmental progression, measured over time and identify clinical factors associated with delays or deviations from expected developmental milestones. Time to attainment of key developmental milestones (derived from QOL survey of caregiver) will be summarized using mean and standard deviation or median and interquartile range. The proportion of patients with delayed development at baseline (first assessment after diagnosis) and follow up will also be reported.	Two years after study initiation
Educational attainment trajectory and milestones, derived from QOL of caregiver Time Frame: Two years after study initiation	Evaluation of educational outcomes over follow-up and examine associations between disease severity and academic achievement. Time to attainment of key educational milestones will be summarized using mean and standard deviation or median and interquartile range. The proportion of patients with delayed educational attainment at baseline (first assessment after diagnosis) and follow up will also be reported.	Two years after study initiation
Caregiver effect: Missed days of school and work for caregiver Time Frame: Two years after study initiation	To quantify school absenteeism and examine its relationship with disease burden and clinical progression. Missed school days will be summarized using descriptive statistics (mean and standard deviation or median and interquartile range as appropriate)	Two years after study initiation
Unplanned Hospitalization(s) Time Frame: Two years after study initiation	To estimate the frequency and timing of unplanned hospitalizations and identify factors associated with unplanned hospitalizations. Unplanned Hospitalization will be summarized using descriptive statistics. The association between clinical factors and number of unplanned hospitalizations will be examined with a multivariable negative binomial regression model	Two years after study initiation
Infections (Urine, ear, skin/tissue, URI and others) Time Frame: Two years after study initiation	To characterize the incidence and types of infections during follow -up and evaluate clinical factors associated with increased infection risk. The incidence and type of infection will be reported with the corresponding 95% CI at each follow up time point. The association between clinical factors and infection will be evaluated using multivariable mixed models with random effects for patient and site	Two years after study initiation
Histopathological Response based on serial biopsy reports and images Time Frame: Two years after study initiation	To describe longitudinal changes in histopathological findings and evaluate associations between pathological features and disease progression. Histopathological Response will each be summarized in the analytic population during the exposure period using descriptive statistics. Distributions will be compared across age groups where appropriate, using Chi -Square or Fisher's exact test, for categorical variables, and Kruskal-Wallis for continuous variables	Two years after study initiation
Renal failure (including dialysis-dependence) Time Frame: Two years after study initiation	To determine the incidence and timing of progression to renal failure (including dialysis-dependence) and to identify prognostic factors associated with risk of progression. The incidence of renal failure will be summarized with the corresponding 95% confidence interval for each disease group. The association between clinical characteristics and time to renal failure will be evaluated using Fine and Gray subdistribution hazard model with prespecified covariates (see above); accounting for mortality (not related to renal failure) and renal transplantation as competing risks.	Two years after study initiation
Renal Transplantation Time Frame: Two years after study initiation	To characterize the incidence and timing of renal transplantation and describe clinical characteristics associated with progression to transplant. The incidence of renal transplantation will be summarized with the corresponding 95% confidence interval for each disease group. Association between clinical characteristics and time to renal transplantation will be evaluated with Fine and Gray sub distribution hazard model with prespecified covariates; accounting for mortality (not related to renal transplantation) and renal failure as competing risks. Time to renal transplantation will be defined as time from diagnosis of GD to the first occurrence of renal transplantation and survivors who do not receive renal transplantation will be censored at the last day of follow up or end of study, whichever occurs first.	Two years after study initiation
Death Time Frame: Two years after study initiation	To estimate overall survival and identify baseline demographic and clinical factors associated with risk of death during follow-up. Association between clinical characteristics and mortality will be evaluated using a multivariable Cox proportional hazards regression with prespecified baseline and time-varying covariates (see below). Survival time will be defined as time from diagnosis of GD to death and survivors will be censored at the last day of follow up or end of study, whichever occurs first.	Two years after study initiation

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Pediatric Nephrology Research Consortium

Collaborators

Washington University School of Medicine

Novartis

Johns Hopkins All Children's Hospital

Investigators

Principal Investigator: Joseph Gaut, MD, PhD, Washington University School of Medicine
Principal Investigator: Carla Nester, MD, University of Iowa
Principal Investigator: William Smoyer, MD, Nationwide Children's Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

United States Renal Data System. Chronic Kidney Disease: Chapter 5. Kidney disease among children and adolescents. 2024 USRDS Annual Data Report: Epidemiology of kidney disease in the United States. Bethesda, MD: National Institutes of Health, National Institute of Diabetes and Digestive and Kidney Diseases; 2024.
Szklo M and Nieto EJ. Epidemiology: Beyond the Basics (Jones and Bartlett, Sudbury, MA, USA), 2004, pp. 27-29.
International Council for Harmonisation (ICH) E11 (R1) Addendum: Clinical Investigation of Medicinal Products in the Pediatric Population. (18 August 2017) section 2.5
Seo-Mayer P, Ashoor I, Hayde N, Laster M, Sanderson K, Soranno D, Wigfall D, Brown D. Seeking justice, equity, diversity and inclusion in pediatric nephrology. Front Pediatr. 2022 Dec 12;10:1084848. doi: 10.3389/fped.2022.1084848. eCollection 2022.
Harambat J, van Stralen KJ, Kim JJ, Tizard EJ. Epidemiology of chronic kidney disease in children. Pediatr Nephrol. 2012 Mar;27(3):363-73. doi: 10.1007/s00467-011-1939-1. Epub 2011 Jun 29.
Bernard TJ, Armstrong-Wells J, Goldenberg NA. The institution-based prospective inception cohort study: design, implementation, and quality assurance in pediatric thrombosis and stroke research. Semin Thromb Hemost. 2013 Feb;39(1):10-4. doi: 10.1055/s-0032-1329551. Epub 2012 Dec 26.
Armstrong-Wells J, Goldenberg NA. Institution-based prospective inception cohort studies in neonatal rare disease research. Semin Fetal Neonatal Med. 2011 Dec;16(6):355-8. doi: 10.1016/j.siny.2011.07.004. Epub 2011 Aug 5.
Pitcher D, Braddon F, Hendry B, Mercer A, Barratt J, Steenkamp R, Wong K, Turner AN, Gong W, Gale DP, Saleem MA. Long-Term Outcomes in Nephrotic Syndrome by Kidney Biopsy Diagnosis and Proteinuria. J Am Soc Nephrol. 2025 Apr 17;36(7):1398-1413. doi: 10.1681/ASN.0000000610.
Pitcher D, Braddon F, Hendry B, Mercer A, Osmaston K, Saleem MA, Steenkamp R, Wong K, Turner AN, Wang K, Gale DP, Barratt J. Long-Term Outcomes in IgA Nephropathy. Clin J Am Soc Nephrol. 2023 Jun 1;18(6):727-738. doi: 10.2215/CJN.0000000000000135. Epub 2023 Apr 13.

Helpful Links

<p>Study website with information for patients, families and clinicians, contains list of sites, information and a glossary of terms</p>

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 1, 2026

Primary Completion (Estimated)

December 1, 2031

Study Completion (Estimated)

June 1, 2032

Study Registration Dates

First Submitted

October 21, 2026

First Submitted That Met QC Criteria

November 5, 2026

First Posted (Actual)

November 12, 2026

Study Record Updates

Last Update Posted (Actual)