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The FOrMe Registry (The German Focal Segmental Glomerulosclerosis and Minimal Change Disease Registry) (FOrMe)

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ClinicalTrials.gov Identifier: NCT03949972
Recruitment Status : Recruiting
First Posted : May 14, 2019
Last Update Posted : August 22, 2019
Sponsor:
Collaborators:
German Research Foundation
Medical Faculty and the Faculty of Natural Sciences of the University of Cologne
Cluster of Excellence on Cellular Stress Responses in Ageing-Associated Diseases
Cologne Center for Genomics
Information provided by (Responsible Party):
Prof. Dr. Paul Brinkkoetter, University of Cologne

Brief Summary:
In a monocentric, later multicentric prospective approach the FOrMe registry (The German Focal Segmental Glomerulosclerosis and Minimal Change Disease Registry) aims to generate a longitudinal cohort of 150 pediatric cases of idiopathic nephrotic syndrome and 350 adult cases of biopsy-proven Minimal Change Disease (MCD) or Focal and Segmental Glomerular Sclerosis (FSGS) over 10 years. The registry will provide a repository for biomaterials such as blood samples, DNA, urine, feces, and tissue biopsies that will be accessible to collaborators to facilitate future research on pathogenesis, diagnostics, and treatment.

Condition or disease Intervention/treatment
Glomerulosclerosis, Focal Segmental Minimal Change Disease Idiopathic Nephrotic Syndrome Other: Biosampling

Detailed Description:

Idiopathic Nephrotic Syndrome is characterized by proteinuria, volume retention, hyperlipidemia, hypoalbuminemia. As Minimal Change Disease (MCD) represents by far the most prevalent underlying diagnosis in children older than 1 year, a kidney biopsy is usually deferred in these cases. In adolescence and adults, a kidney biopsy is crucial for the diagnosis because MCD and FSGS account for only 10-15 and 12-35 percent of all cases of nephrotic syndrome respectively. Pathomechanisms as well as optimal treatment remain elusive as systematic trials are scarce and hampered by low incidence and heterogenicity of the clinical presentation. To bridge this informational gap, the investigators identified the need for a German registry of pediatric and adult patients with idiopathic nephrotic syndrome (in children) and biopsy-proven MCD/FSGS (in adults).

The registry will record clinical data of participants regarding basic demographics, initial presentation, hereditary traits, disease course and treatment modalities as well as quality of life, concomitant diseases, and comedication. During the initial visit and to a lesser intent on follow-up visits, biomaterials (blood, urine, DNA, feces, tissue) will be collected and stored in a state-of-the art biobank. This material will be available to collaborators to support research on idiopathic nephrotic syndrome and MCD/FSGS. By the time of completion, the registry will provide data on clinical courses and outcome of approximately 500 patients that can easily be correlated with biomaterials giving insight into risk factors, prognostic parameters, and association with comorbidities.

Tissue sections of all patients that undergo kidney biopsy (all adult and some pediatric patients) will be digitalized, annotated, and analyzed by a panel of nephropathologists. Histopathologic features will be individually assessed and scored according to a set of descriptors that was developed and is used by the American NEPTUNE (Nephrotic Syndrome Study Network).


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Study Type : Observational [Patient Registry]
Estimated Enrollment : 500 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 15 Years
Official Title: The FOrMe Registry (The German Focal Segmental Glomerulosclerosis and Minimal Change Disease Registry)
Actual Study Start Date : April 1, 2018
Estimated Primary Completion Date : March 31, 2028
Estimated Study Completion Date : March 31, 2033


Group/Cohort Intervention/treatment
A - Pediatric Cohort
Pediatric patients until 18 years of age presenting with or diagnosed with idiopathic nephrotic syndrome or a biopsy-proven diagnosis of MCD or FSGS.
Other: Biosampling
Biosampling at initial visit and follow-up visits

B -Adult Cohort
Adult patients 18 years and above with a biopsy-proven diagnosis of primary or secondary FSGS or MCD.
Other: Biosampling
Biosampling at initial visit and follow-up visits




Primary Outcome Measures :
  1. Average Annual Change in estimated glomerular filtration rate (eGFR) [ Time Frame: 5-15 years ]
    Outcome measure: eGFR loss in ml/min/year. Higher values are considered worse outcome.

  2. Incidence of End-stage Renal Disease (ESRD) [ Time Frame: 5-15 years ]
    Documented initiation of chronic renal replacement therapy regardless of type.

  3. Incidence of Death [ Time Frame: 5-15 years ]
    Documented patient death due to any cause

  4. Incidence of Kidney Transplantation [ Time Frame: 5-15 years ]
    Documented kidney transplantation regardless of type (living donor, cadaveric donor)

  5. Changes in Quality of Life (adults patients) [ Time Frame: 5-15 years ]

    Patient-reported outcome will be assessed using Quality of Life questionnaires at regular intervals using the SF-36 questionnaire.

    For reference see https://www.rand.org/health-care/surveys_tools/mos/36-item-short-form/scoring.html The SF-36 questionnaire measures eight health concepts: physical functioning, bodily pain, role limitations due to physical health problems, role limitations due to personal or emotional problems, emotional well-being, social functioning, energy/fatigue, and general health perceptions. All items are scored so that a high score defines a more favorable health state. Lowest and highest possible scores are 0 and 100. Scores represent the percentage of total possible score achieved.

    Original publication: Ware, J.E., Jr., & Sherbourne, C.D. "The MOS 36-Item Short-Form Health Survey (SF-36): I. Conceptual Framework and Item Selection,". Medical Care, 30:473-483, 1992.


  6. Changes in Quality of Life (pediatric patients) [ Time Frame: 5-15 years ]

    Patient-reported outcome will be assessed using Quality of Life questionnaires at regular intervals using the PedsQL questionnaire.

    For reference see https://www.pedsql.org/score.html The PedSQL questionnaire measures four health concepts: Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning. Items are reversed scored and linearly transformed to a 0-100 scale, so that higher scores indicate better HRQOL (Health-Related Quality of Life). To create Scale Scores, the mean is computed as the sum of the items over the number of items answered (this accounts for missing data). To create the Total Scale Score, the mean is computed as the sum of all the items over the number of items answered on all the Scales.



Biospecimen Retention:   Samples With DNA
Blood (plasma, serum), urine, DNA, feces


Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Pediatric patients until 18 years of age presenting with or diagnosed with idiopathic nephrotic syndrome or a biopsy-proven diagnosis of MCD or FSGS. Candidate participants need to be willing to provide written informed consent.

Adult patients 18 years and above with a biopsy-proven diagnosis of primary or secondary FSGS or MCD. Candidate participants need to be willing to provide written informed consent.

Criteria

Inclusion Criteria (cohort A):

  • written informed consent
  • 17 or less years of age
  • idiopathic nephrotic syndrome

Inclusion Criteria (cohort B):

  • written informed consent
  • older or equal to 18 years of age
  • biopsy-proven primary or secondary FSGS or MCD or biopsy-proven recurrence of disease in kidney transplant.

Exclusion Criteria (both cohorts):

  • Prior kidney transplant without biopsy-proven recurrence
  • A clinical diagnosis of other glomerular disease resulting in secondary MCD or FSGS as judged by the treating physicians.
  • Refusal to provide written informed consent
  • (Anticipated) incompliance with visit schedule

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03949972


Contacts
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Contact: Paul T Brinkkoetter, MD +49-221-478-4480 paul.brinkkoetter@uk-koeln.de
Contact: Linus A Voelker, MD +49-221-478-4480 linus.voelker@uk-koeln.de

Locations
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Germany
University Hospital of Cologne Recruiting
Cologne, NRW, Germany, 50937
Contact: paul brinkkoetter, MD    +49-221 478-4480    paul.brinkkoetter@uk-koeln.de   
Contact: Lutz Weber, MD    +49 221 478-4319    lutz.weber@uk-koeln.de   
Sub-Investigator: Linus Völker, MD         
Sub-Investigator: Paul Brinkkötter, MD         
Sub-Investigator: Rasmus Ehren, MD         
Sub-Investigator: Franziska Grundmann, MD         
Sub-Investigator: Thomas Benzing, MD         
Sponsors and Collaborators
Prof. Dr. Paul Brinkkoetter
German Research Foundation
Medical Faculty and the Faculty of Natural Sciences of the University of Cologne
Cluster of Excellence on Cellular Stress Responses in Ageing-Associated Diseases
Cologne Center for Genomics
Investigators
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Study Director: Paul T Brinkkoetter, MD University Hospital of Cologne, Cologne, Germany

Additional Information:
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Responsible Party: Prof. Dr. Paul Brinkkoetter, Consultant in Nephrology, Scientific Coordinator KFO 329, University of Cologne
ClinicalTrials.gov Identifier: NCT03949972     History of Changes
Other Study ID Numbers: 005
First Posted: May 14, 2019    Key Record Dates
Last Update Posted: August 22, 2019
Last Verified: August 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Biosamples as well as patient data will be available to collaborators after revision of research application by a steering committee.
Time Frame: 5-15 years
Access Criteria: Actively contributing to registry and approval by international steering committee.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Prof. Dr. Paul Brinkkoetter, University of Cologne:
Focal and Segmental Glomerulosclerosis
Focal & Segmental Glomerulosclerosis
FSGS
Minimal change disease
MCD
Nephrotic Syndrome

Additional relevant MeSH terms:
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Nephrotic Syndrome
Nephrosis
Glomerulosclerosis, Focal Segmental
Nephrosis, Lipoid
Kidney Diseases
Urologic Diseases
Glomerulonephritis
Nephritis