The FOrMe Registry (The German Focal Segmental Glomerulosclerosis and Minimal Change Disease Registry)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 500
- 试验地点
- 12
- 主要终点
- Incidence of Death
研究概览
简要总结
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.
详细描述
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).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
Incidence of Death
时间窗: 5-15 years
Documented patient death due to any cause
Average Annual Change in estimated glomerular filtration rate (eGFR)
时间窗: 5-15 years
Outcome measure: eGFR loss in ml/min/year. Higher values are considered worse outcome.
Incidence of End-stage Renal Disease (ESRD)
时间窗: 5-15 years
Documented initiation of chronic renal replacement therapy regardless of type.
Incidence of Kidney Transplantation
时间窗: 5-15 years
Documented kidney transplantation regardless of type (living donor, cadaveric donor)
Changes in Quality of Life (adults patients)
时间窗: 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.
Changes in Quality of Life (pediatric patients)
时间窗: 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.
次要结局
未报告次要终点
研究者
Prof. Dr. Paul Brinkkoetter
Consultant in Nephrology, Scientific Coordinator KFO 329
University of Cologne