Optimalisation of the Treatment of Acute Neonatal Hyperammonaemia

Recruiting

• Conditions: Hyperammonemic Encephalopathy
• Interventions: Diagnostic Test: user-friendly and widely applicable algorithm to tailor the treatment of acute neonatal hyperammonemia

• Registration Number: NCT05754372
• Lead Sponsor: University Hospital, Ghent

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-01-03
• Study Start: 2023-01-15
• Study First Submitted QC: 2023-03-01
• Study First Posted: 2023-03-03
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-11
• Last Update Posted: 2024-04-15
• Primary Completion: 2025-12-31
• Study Completion: 2026-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

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

Exclusion Criteria

• older than 1 year

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Hyperammonaemia	user-friendly and widely applicable algorithm to tailor the treatment of acute neonatal hyperammonemia	-

Primary Outcome Measures

Name	Time	Method
serum ammonium levels	1 year	serum ammonium levels

Secondary Outcome Measures

Name	Time	Method
Removal of treatment sodium benzoate, L-arginine	1 year	serum and dialysate concentration (mg/dL) of sodium benzoate and L-arginine

Trial Locations

Locations (1)

Ghent University Hospital - Department of Paediatric Nephrology; 🇧🇪 Ghent, Belgium