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

Minimal Change Disease Clinical Trial

— FOrMe  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03949972
• Other study ID #: 005
• Status: Recruiting
• Start date: April 1, 2018
• Est. completion date: March 31, 2033

Study information

• Verified date: May 2024
• Source: University of Cologne
• Contact: Paul T Brinkkoetter, MD
• Phone: +49-221-478-4480
• Email: paul.brinkkoetter[@]uk-koeln.de
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 500
• Est. completion date: March 31, 2033
• Est. primary completion date: March 31, 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Glomerulosclerosis, Focal Segmental
• Idiopathic Nephrotic Syndrome
• Minimal Change Disease
• Nephrosis
• Nephrosis, Lipoid
• Nephrotic Syndrome

Intervention

Other:
• Biosampling
Biosampling at initial visit and follow-up visits

Locations

Country	Name	City	State
Germany	Charité University Hospital	Berlin
Germany	Kindernierenzentrum Bonn	Bonn
Germany	University Hospital of Cologne	Cologne	NRW
Germany	Kindernephrologie Dachau	Dachau
Germany	University Hospital Erlangen	Erlangen
Germany	University Hospital Essen	Essen
Germany	University Hospital Greifswald	Greifswald
Germany	University Hospital Heidelberg	Heidelberg
Germany	Klinikum St. Georg	Leipzig
Germany	University Hospital Marburg	Marburg
Germany	University Hospital Münster	Münster

Sponsors (5)

Lead Sponsor	Collaborator
Prof. Dr. Paul Brinkkoetter	Cluster of Excellence on Cellular Stress Responses in Ageing-Associated Diseases, Cologne Center for Genomics, German Research Foundation, Medical Faculty and the Faculty of Natural Sciences of the University of Cologne

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Average Annual Change in estimated glomerular filtration rate (eGFR)	Outcome measure: eGFR loss in ml/min/year. Higher values are considered worse outcome.	5-15 years
Primary	Incidence of End-stage Renal Disease (ESRD)	Documented initiation of chronic renal replacement therapy regardless of type.	5-15 years
Primary	Incidence of Death	Documented patient death due to any cause	5-15 years
Primary	Incidence of Kidney Transplantation	Documented kidney transplantation regardless of type (living donor, cadaveric donor)	5-15 years
Primary	Changes in Quality of Life (adults patients)	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.	5-15 years
Primary	Changes in Quality of Life (pediatric patients)	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.	5-15 years

External Links

•   CRU 329 Homepage