Clinical trial NCT03949972: The FOrMe Registry (The German Focal Segmental Glomerulosclerosis and Minimal Change Disease Registry)
Official title: The FOrMe Registry (The German Focal Segmental Glomerulosclerosis and Minimal Change Disease Registry)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Glomerulosclerosis, Focal Segmental
Minimal Change Disease
Idiopathic Nephrotic Syndrome
Keywords: Focal and Segmental Glomerulosclerosis
Focal & Segmental Glomerulosclerosis
FSGS
Minimal change disease
MCD
Nephrotic Syndrome
Lead sponsor: University of Cologne
Sponsor class: OTHER
Collaborators: name: German Research Foundation
name: Medical Faculty and the Faculty of Natural Sciences of the University of Cologne
name: Cluster of Excellence on Cellular Stress Responses in Ageing-Associated Diseases
name: Cologne Center for Genomics
First posted: 2019-05-14
Enrollment: 500
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.

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).

Eligibility 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

Primary outcomes:
measure: Average Annual Change in estimated glomerular filtration rate (eGFR)
timeFrame: 5-15 years
measure: Incidence of End-stage Renal Disease (ESRD)
timeFrame: 5-15 years
measure: Incidence of Death
timeFrame: 5-15 years
measure: Incidence of Kidney Transplantation
timeFrame: 5-15 years
measure: Changes in Quality of Life (adults patients)
timeFrame: 5-15 years
measure: Changes in Quality of Life (pediatric patients)
timeFrame: 5-15 years
Secondary outcomes:
Other outcomes:
Locations:
city: Cologne
country: Germany
facility: University Hospital of Cologne
state: NRW
city: Berlin
country: Germany
facility: Charité University Hospital
city: Bonn
country: Germany
facility: Kindernierenzentrum Bonn
city: Dachau
country: Germany
facility: Kindernephrologie Dachau
city: Erlangen
country: Germany
facility: University Hospital Erlangen
city: Essen
country: Germany
facility: University Hospital Essen
city: Greifswald
country: Germany
facility: University Hospital Greifswald
city: Heidelberg
country: Germany
facility: University Hospital Heidelberg
city: Leipzig
country: Germany
facility: Klinikum St. Georg
city: Marburg
country: Germany
facility: University Hospital Marburg
city: Münster
country: Germany
facility: University Hospital Münster
Study officials:
affiliation: University Hospital of Cologne, Cologne, Germany
name: Paul T Brinkkoetter, MD
role: STUDY_DIRECTOR
References: