Multicentric Retrospective Study on the Use of Cinacalcet in an Off-label Phosphocalcic Context
试验速览
- 阶段
- 不适用
- 入组人数
- 25
- 试验地点
- 10
- 主要终点
- Serum PTH concentration
研究概览
简要总结
Currently, the indications used for MA (Marketing Authorization) Cinacalcet in France are hyperparathyroidism (hyperPTH) in adults, whether primary (for patients in whom parathyroidectomy is theoretically indicated but in whom it is contraindicated or not is not clinically appropriate) or secondary to a chronic kidney disease, and parathyroid carcinomas.
In pediatric patients, data on its use are restricted due to its recent marketing authorization (2017) and limited to dialysis patients suffering from secondary hyperPTH.
Nevertheless, some patients with phosphocalcic pathologies without renal insufficiency must be treated off-label by cinacalcet in the presence of severe hyperPTH, without any other chronic treatment available to date.
The objective of this study is therefore to evaluate the use in France of cinacalcet in phosphocalcic pathologies without renal insufficiency, in order to obtain efficacy and safety data in order to improve our knowledge on the management of these orphan diseases.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- — 至 99 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with phosphocalcic pathology without end-stage renal failure
- •Patients followed in one of the hospitals of the centers of reference and competence of the diseases of the metabolism of calcium and phosphate
- •For children under 18: patient and parent (s) / parent having been informed of the study and having expressed their on-opposition
- •For adults: patient / legal guardian of the patient under guardianship having been informed of the study and expressing his or her non opposition
排除标准
- •Patient suffering from parathyroid cancer,
- •Patient suffering from primary hyperPTH in whom parathyroidectomy would theoretically be indicated but contraindicated or clinically inappropriate,
- •Patient suffering from hyperPTH secondary to end-stage renal failure
- •No social security support
结局指标
主要结局
Serum PTH concentration
时间窗: 3 months after treatment initiation
次要结局
- Serum PTH concentration(3 years after treatment initiation)