- ICH GCP
- Registro degli studi clinici negli Stati Uniti
- Sperimentazione clinica NCT07931170
Pediatric Longitudinal Glomerular Disease: a PNRC Multi-Institutional Cohort Study (PLUGGED IN)
To review and collect information from a patients medical record in order to study and research how to help other children with glomerular diseases.
Panoramica dello studio
Stato
Condizioni
- Malattia a cambiamento minimo
- Nefropatia membranosa
- C3 Glomerulopatia
- Sindrome di Alporto
- Nefropatia da IgA (IgAN)
- Vasculite da IgA
- Nefrite lupica (LN)
- Sindrome nefrotica idiopatica (INS)
- Idiopathic Immune Complex Glomerulonephritis
- Focal and Segmental Glomerulosclerosis
- Anti-Neutrophilic Cytoplasmic Antibody-GN (ANCA)
- Infection-related or Post-infectious Glomerulonephritis
Descrizione dettagliata
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.
Tipo di studio
Iscrizione (Stimato)
Contatti e Sedi
Contatto studio
- Nome: Amy Dutcher, MCR
- Numero di telefono: 6145547621
- Email: adutcher@PNRConsortium.org
Backup dei contatti dello studio
- Nome: Teri Crumb, MSN, RN, CCRC
- Numero di telefono: 616-607-4430
- Email: teri@pnrconsortium.org
Luoghi di studio
-
-
Massachusetts
-
Boston, Massachusetts, Stati Uniti, 02115
- Reclutamento
- Boston Children's Hospital
-
Contatto:
- Natalie Pursel
- Numero di telefono: 617-355-6762
- Email: getpluggedin@pnrconsortium.org
-
Contatto:
- Swethapriya Damagatla
- Numero di telefono: 617-355-6762
- Email: getpluggedin@pnrconsortium.org
-
Investigatore principale:
- Ankana Daga, MD
-
-
Ohio
-
Cincinnati, Ohio, Stati Uniti, 45229
- Reclutamento
- Cincinnati Children's Hospital Medical Center
-
Contatto:
- Libby Siry, CRP
- Numero di telefono: 513.517.1439
- Email: getpluggedin@pnrconsortium.org
-
Contatto:
- Nina Kanis, CRC
- Numero di telefono: 513.517.1439
- Email: getpluggedin@pnrconsortium.org
-
Investigatore principale:
- Elif Erkan, MD
-
-
Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Metodo di campionamento
Popolazione di studio
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)
Descrizione
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)
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Modelli osservazionali: COHORT
- Prospettive temporali: OTHER
Coorti e interventi
Gruppo / Coorte |
|---|
|
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 |
Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Proteinuria, and change over time thereof (urine protein-creatinine ratio)
Lasso di tempo: 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
|
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Percent change in estimated glomerular filtration rate (eGFR) from baseline to follow up
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
|
Collaboratori e investigatori
Collaboratori
Investigatori
- Investigatore principale: Joseph Gaut, MD, PhD, Washington University School of Medicine
- Investigatore principale: Carla Nester, MD, University of Iowa
- Investigatore principale: William Smoyer, MD, Nationwide Children's Hospital
Pubblicazioni e link utili
Pubblicazioni generali
- 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.
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Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
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Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
- Malattie urogenitali
- Malattie vascolari
- Malattia cardiovascolare
- Processi patologici
- Malattie urogenitali maschili
- Malattie urologiche
- Malattie urogenitali femminili
- Malattie urogenitali femminili e complicanze della gravidanza
- Malattie del tessuto connettivo
- Malattie autoimmuni
- Malattie del sistema immunitario
- Emorragia
- Manifestazioni cutanee
- Ipersensibilità
- Malattie ematologiche
- Malattie della pelle
- Anomalie congenite
- Disturbi della coagulazione del sangue
- Malattie della pelle, vascolari
- Disturbi emostatici
- Disturbi emorragici
- Anomalie urogenitali
- Vasculite
- Glomerulonefrite
- Lupus Eritematoso, Sistemico
- Porpora
- Malattie del collagene
- Nefrosi
- Malattie del complesso immunitario
- Malattie e anomalie congenite, ereditarie e neonatali
- Condizioni patologiche, segni e sintomi
- Malattie della pelle e del tessuto connettivo
- Segni e sintomi
- Malattie emiche e linfatiche
- Nefrite
- Nefrite da lupus
- Malattie renali
- Glomerulonefrite, IGA
- Glomerulonefrite, membranosa
- Glomerulosclerosi, segmentale focale
- Nefrite, ereditaria
- Nefrosi, lipoide
- Vasculite da IgA
Altri numeri di identificazione dello studio
- PNRC 2026-003
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