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临床试验/NCT05754372
NCT05754372招募中不适用

Optimalisation of the Treatment of Acute Neonatal Hyperammonaemia

University Hospital, Ghent1 个研究点 分布在 1 个国家目标入组 10 人开始时间: 2023年1月15日最近更新:
适应症

试验速览

阶段
不适用
状态
招募中
入组人数
10
试验地点
1
主要终点
serum ammonium levels

研究概览

简要总结

Acute neonatal hyperammonemia is associated with poor neurological outcomes and high mortality. A user-friendly and widely applicable algorithm - based on kinetics - to tailor the treatment of acute neonatal hyperammonemia. A single compartmental model was calibrated assuming a distribution volume equal to the patient's total body water (V), as calculated using Wells' formula, and dialyzer clearance as derived from the measured ammonia time-concentration curves during 11 dialysis sessions in four patients (3.2 +/- 0.4 kg). Based on these kinetic simulations, dialysis protocols could be derived for clinical use with different body weights, start concentrations, dialysis machines/dialyzers and dialysis settings (e.g., blood flow QB). By a single measurement of ammonia concentration at the dialyzer inlet and outlet, dialyzer clearance (K) can be calculated as K = QB[(Cinlet - Coutlet)/Cinlet]. The time (T) needed to decrease the ammonia concentration from a predialysis start concentration Cstart to a desired target concentration Ctarget is then equal to T = (-V/K)LN(Ctarget/Cstart). By implementing these formulae in a simple spreadsheet, medical staff can draw an institution-specific flowchart for patient-tailored treatment of hyperammonemia.

The aim of this study is to validate these formula with a prospective study.

研究设计

研究类型
Observational
观察模型
Cohort
时间视角
Prospective

入排标准

年龄范围
— 至 1 Year(Child)
性别
All
接受健康志愿者

入选标准

  • severe acute neonatal hyperammonaemia for which acute hemodialysis is indicated
  • inborn error of metabolism
  • below 1 year of age

排除标准

  • older than 1 year

结局指标

主要结局

serum ammonium levels

时间窗: 1 year

serum ammonium levels

次要结局

  • Removal of treatment sodium benzoate, L-arginine(1 year)

研究者

申办方类型
Other
责任方
Sponsor

研究点 (1)

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