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Идёт набор NCT03949972

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

— Наблюдательное Glomerulosclerosis, Focal Segmental Minimal Change Disease Idiopathic Nephrotic Syndrome

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают
В протоколе указаны: Biosampling.
Кому может быть актуально
Состояния в реестре: Glomerulosclerosis, Focal Segmental, Minimal Change Disease, Idiopathic Nephrotic Syndrome. Базовые параметры: Без ограничений · Все.
Что важно проверить
Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится
Германия
Следующий шаг
Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Обзор

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.

Подробное описание

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).

Вмешательства

  • Другое Biosampling
    Biosampling at initial visit and follow-up visits

Первичные конечные точки

  • Average Annual Change in estimated glomerular filtration rate (eGFR) [Срок оценки: 5-15 years]
  • Incidence of End-stage Renal Disease (ESRD) [Срок оценки: 5-15 years]
  • Incidence of Death [Срок оценки: 5-15 years]
  • Incidence of Kidney Transplantation [Срок оценки: 5-15 years]
  • Changes in Quality of Life (adults patients) [Срок оценки: 5-15 years]
  • Changes in Quality of Life (pediatric patients) [Срок оценки: 5-15 years]

Критерии участия

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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения
Когортное

Центры проведения

Германия · 12 центров
  • University Hospital of Cologne — Cologne
  • Uniklinik RWTH Aachen — Aachen
  • Charité University Hospital — Berlin
  • Kindernierenzentrum Bonn — Bonn
  • Kindernephrologie Dachau — Dachau
  • University Hospital Erlangen — Erlangen
  • University Hospital Essen — Essen
  • University Hospital Heidelberg — Heidelberg
  • … и ещё 4 центра

Публикации

  • Osterholt T, Todorova P, Kuhne L, Ehren R, Weber LT, Grundmann F, Benzing T, Brinkkotter PT, Volker LA. Repetitive administration of rituximab can achieve and maintain clinical remission in patients with MCD or FSGS. Sci Rep. 2023 Apr 28;13(1):6980. doi: 10.1038/s41598-023-32576-7. PMID 37117201

Идентификаторы

NCT: NCT03949972 · 005

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗